This global ITP registry study is FDA approved and developed in partnership with the National Organization for Rare Disorders (NORD) to follow ITP patients of all ages, in all phases of disease to learn more about the natural history of ITP. The goal of the registry is to learn more about the clinical characteristics of ITP, the incidence of co-morbidities, treatment patterns, adverse outcomes, how quality of life is impacted, how family and socioeconomics is affected, and how ITP patients respond to COVID-19 and vaccinations. 

Note: The registry is in English only, currently.

USA, Canada, International (global)

https://www.pdsa.org/registry.html

Recruiting

Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), PH20 administered subcutaneously. This is a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.

Argentina, Australia, Chile, Georgia, Ireland, Israel, Italy, Japan, Republic of Korea, New Zealand, Norway, Poland, Portugal, Romania, Russian Federation, Spain, Thailand, Turkey, and USA

https://www.clinicaltrials.gov/ct2/show/NCT04687072?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=14#contacts

Recruiting

Principia Biopharma is conducting a phase three randomized, double-blind study of rilzabrutinib (previously referred to as PRN1008), and oral BTK inhibitor. They are looking to enroll patients with persistent or chronic ITP who are at least 12 years of age. Eligible patients must have an average platelet count of <30,000/μL on two counts (with no count higher than <35,000/μL) at least 5 days apart in the 14 days before treatment begins. The double-blind treatment period is 12-24 weeks depending on platelet response, followed by a 28-week open label period. Patients who respond to rilzabrutinib may enter a long term-extension. After the last dose of rilzabrutinib there will be a 4-week safety follow-up visit.

Argentina, Austria, Australia, Chile, France, Germany, Hungary, Israel, Italy, Norway, Poland, Russian Confederation, Singapore, South Korea, Spain, Thailand, Ukraine, UK, and the USA.

https://clinicaltrials.gov/ct2/show/NCT04562766

Recruiting

This phase 3 multicenter, open-label, long-term clinical trial is designed to determine the safety and efficacy of Efgartigimod (ARGX-113) PH20 given subcutaneously to adults (over 18 years) with primary ITP. Efgartigimod is a first-in-class investigational antibody fragment to target the neonatal Fc receptor (FcRn). Eligible participants must be at least 18 years of age.

Australia, Bulgaria, China, Italy, Japan, Poland, Portugal, Romania, Russian Federation, Thailand, Turkey, USA

https://www.clinicaltrials.gov/ct2/show/NCT04812925?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=16

Status: Recruiting

Principia Biopharma is conducting a study of their oral drug PRN1008 in patients with ITP who are refractory or relapsed with no available and approved therapeutic options and have a platelet count <30,000/μL on two counts no sooner than 7 days apart in the 15 days before treatment begins. Preclinical findings show that PRN1008 decreases the activity of B cells and other inflammatory-causing immune cells, and in a mouse model of ITP show that PRN1008 can reduce platelet loss.

AUSTRALIA

https://clinicaltrials.gov/ct2/show/NCT03395210?term=PRN1008&rank=1

Recruiting

Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), PH20 administered subcutaneously. This is a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.

Argentina, Australia, Chile, Georgia, Ireland, Israel, Italy, Japan, Republic of Korea, New Zealand, Norway, Poland, Portugal, Romania, Russian Federation, Spain, Thailand, Turkey, and USA

https://www.clinicaltrials.gov/ct2/show/NCT04687072?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=14#contacts

Recruiting

Pfizer is conducting an interventional phase two, open-label, one-arm, multicenter study to determine the safety and efficacy of a drug, PF-06835375, in adults with moderate-severe bleeding due to ITP. Specifically, the study will determine if the drug can increase platelet counts to reduce bleeding risks, without a significant side effect profile. PF-06835375 is a chemokine (CXCR5) receptor antagonist that works as an immune system regulator and has been reported as a critical chemokine regulator in several autoimmune diseases. Its mechanism of action is not fully understood yet. The drug would be provided through a subcutaneous injection every month for a total of three months. Eligible participants include adults (between the ages of 18-70 years) with primary persistent or chronic ITP (over 3 months since diagnosis) who have a platelet count less than 50,000/μL and have not experienced severe bleeding within 1 month. Individuals who have had a splenectomy within three months of the study initiation period will be excluded from participating.

Australia, Canada, Czechia, Hungary, Poland, USA

https://www.clinicaltrials.gov/ct2/show/NCT05070845?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=15

Status: Recruiting

Principia Biopharma is conducting a phase three randomized, double-blind study of rilzabrutinib (previously referred to as PRN1008), and oral BTK inhibitor. They are looking to enroll patients with persistent or chronic ITP who are at least 12 years of age. Eligible patients must have an average platelet count of <30,000/μL on two counts (with no count higher than <35,000/μL) at least 5 days apart in the 14 days before treatment begins. The double-blind treatment period is 12-24 weeks depending on platelet response, followed by a 28-week open label period. Patients who respond to rilzabrutinib may enter a long term-extension. After the last dose of rilzabrutinib there will be a 4-week safety follow-up visit.

Argentina, Austria, Australia, Chile, France, Germany, Hungary, Israel, Italy, Norway, Poland, Russian Confederation, Singapore, South Korea, Spain, Thailand, Ukraine, UK, and the USA.

https://clinicaltrials.gov/ct2/show/NCT04562766

Recruiting

This international ITP registry study through the South Eastern Sydney Local Health District and GlaxoSmithLine is looking to prospectively enrol newly diagnosed adult patients with ITP to follow them for a period of two years. This study is recruiting ITP patients over the age of 18 years from one of the international designated participating centres. The goal of this registry is to learn more about the clinical characteristics of ITP, including the prevalence of the disease in various places, the incidence of co-morbidities, treatment patterns, and possible adverse outcomes (resulting from treatment or disease). Multi-site participation of this registry project will be rolled out in stages.

Australia, Korea, Taiwan, Singapore, Malaysia, Japan, Thailand, and Turkey.

https://www.clinicaltrials.gov/ct2/show/NCT01970189?recrs=ab&cond=Immune+Thrombocytopenia&draw=2&rank=47

Recruiting

Principia Biopharma is conducting a phase three randomized, double-blind study of rilzabrutinib (previously referred to as PRN1008), and oral BTK inhibitor. They are looking to enroll patients with persistent or chronic ITP who are at least 12 years of age. Eligible patients must have an average platelet count of <30,000/μL on two counts (with no count higher than <35,000/μL) at least 5 days apart in the 14 days before treatment begins. The double-blind treatment period is 12-24 weeks depending on platelet response, followed by a 28-week open label period. Patients who respond to rilzabrutinib may enter a long term-extension. After the last dose of rilzabrutinib there will be a 4-week safety follow-up visit.

Argentina, Austria, Australia, Chile, France, Germany, Hungary, Israel, Italy, Norway, Poland, Russian Confederation, Singapore, South Korea, Spain, Thailand, Ukraine, UK, and the USA.

https://clinicaltrials.gov/ct2/show/NCT04562766

Recruiting

This phase 3 multicenter, open-label, long-term clinical trial is designed to determine the safety and efficacy of Efgartigimod (ARGX-113) PH20 given subcutaneously to adults (over 18 years) with primary ITP. Efgartigimod is a first-in-class investigational antibody fragment to target the neonatal Fc receptor (FcRn). Eligible participants must be at least 18 years of age.

Australia, Bulgaria, China, Italy, Japan, Poland, Portugal, Romania, Russian Federation, Thailand, Turkey, USA

https://www.clinicaltrials.gov/ct2/show/NCT04812925?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=16

Status: Recruiting

Principia Biopharma is conducting a study of their oral drug PRN1008 in patients with ITP who are refractory or relapsed with no available and approved therapeutic options and have a platelet count <30,000/μL on two counts no sooner than 7 days apart in the 15 days before treatment begins. Preclinical findings show that PRN1008 decreases the activity of B cells and other inflammatory-causing immune cells, and in a mouse model of ITP show that PRN1008 can reduce platelet loss.

BULGARIA

https://clinicaltrials.gov/ct2/show/NCT03395210?term=PRN1008&rank=1

Recruiting

Takeda pharmaceuticals is conducting a multi-centered phase two placebo-controlled double-blinded study to look at the safety, tolerability, and efficacy of a drug called TAK-079, which is a monoclonal antibody (anti-CD38 antibody). The trial is for adult ITP patients whose ITP is persistent or chronic, with a platelet count less than 30,000/µL for four consecutive weeks prior to participating in the trial. TAK-079 would be administered as a subcutaneous injection.

Bulgaria, Croatia, Germany, Italy, Slovenia, Ukraine, and USA.

https://www.clinicaltrials.gov/ct2/show/NCT04278924?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=11

Recruiting

Principia Biopharma is conducting a study of their oral drug PRN1008 in patients with ITP who are refractory or relapsed with no available and approved therapeutic options and have a platelet count <30,000/μL on two counts no sooner than 7 days apart in the 15 days before treatment begins. Preclinical findings show that PRN1008 decreases the activity of B cells and other inflammatory-causing immune cells, and in a mouse model of ITP show that PRN1008 can reduce platelet loss.

CANADA

https://clinicaltrials.gov/ct2/show/NCT03395210?term=PRN1008&rank=1

Recruiting

This global ITP registry study is FDA approved and developed in partnership with the National Organization for Rare Disorders (NORD) to follow ITP patients of all ages, in all phases of disease to learn more about the natural history of ITP. The goal of the registry is to learn more about the clinical characteristics of ITP, the incidence of co-morbidities, treatment patterns, adverse outcomes, how quality of life is impacted, how family and socioeconomics is affected, and how ITP patients respond to COVID-19 and vaccinations.  Note: The registry is in English only, currently.

USA, Canada, International (all places).

https://www.pdsa.org/registry.html

Recruiting

This is a randomized double-blind pilot study to determine the clinical utility of using Oseltamirvir compared to placebo in the treatment of ITP. Oseltamivir is an attractive drug for ITP since it specifically targets a pathophysiologic mechanism that appears to be important for the development of ITP and has a benign side effect profile compared to standard ITP therapy. Eligibility includes adults with ITP who have not had a sustained response to other available treatments.

Canada

https://clinicaltrials.gov/ct2/show/NCT03520049?recrs=a&cond=ITP&draw=4

Status: Recruiting

Pfizer is conducting an interventional phase two, open-label, one-arm, multicenter study to determine the safety and efficacy of a drug, PF-06835375, in adults with moderate-severe bleeding due to ITP. Specifically, the study will determine if the drug can increase platelet counts to reduce bleeding risks, without a significant side effect profile. PF-06835375 is a chemokine (CXCR5) receptor antagonist that works as an immune system regulator and has been reported as a critical chemokine regulator in several autoimmune diseases. Its mechanism of action is not fully understood yet. The drug would be provided through a subcutaneous injection every month for a total of three months. Eligible participants include adults (between the ages of 18-70 years) with primary persistent or chronic ITP (over 3 months since diagnosis) who have a platelet count less than 50,000/μL and have not experienced severe bleeding within 1 month. Individuals who have had a splenectomy within three months of the study initiation period will be excluded from participating.

Australia, Canada, Czechia, Hungary, Poland, USA

https://www.clinicaltrials.gov/ct2/show/NCT05070845?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=15

Status: Recruiting

Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), PH20 administered subcutaneously. This is a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.

Argentina, Australia, Chile, Georgia, Ireland, Israel, Italy, Japan, Republic of Korea, New Zealand, Norway, Poland, Portugal, Romania, Russian Federation, Spain, Thailand, Turkey, and USA

https://www.clinicaltrials.gov/ct2/show/NCT04687072?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=14#contacts

Recruiting

Principia Biopharma is conducting a phase three randomized, double-blind study of rilzabrutinib (previously referred to as PRN1008), and oral BTK inhibitor. They are looking to enroll patients with persistent or chronic ITP who are at least 12 years of age. Eligible patients must have an average platelet count of <30,000/μL on two counts (with no count higher than <35,000/μL) at least 5 days apart in the 14 days before treatment begins. The double-blind treatment period is 12-24 weeks depending on platelet response, followed by a 28-week open label period. Patients who respond to rilzabrutinib may enter a long term-extension. After the last dose of rilzabrutinib there will be a 4-week safety follow-up visit.

Argentina, Austria, Australia, Chile, France, Germany, Hungary, Israel, Italy, Norway, Poland, Russian Confederation, Singapore, South Korea, Spain, Thailand, Ukraine, UK, and the USA.

https://clinicaltrials.gov/ct2/show/NCT04562766

Recruiting

Harbour BioMed (Guangzhou) Co. Ltd. Is conducting a phase 2/3 randomized, double-blind, placebo-controlled, clinical study to evaluate the efficacy and safety of HBM9161, a human anti-FcRN monoclonal antibody that removes IgG to reduce platelet destruction and increase the overall platelet count. This drug is administered through a subcutaneous injection and in this study would be provided to participants with ITP weekly. The study will be conducted in a Phase 2/3 operational seamless design. Eligible participants include adults with ITP who are 18 years of age or older. Participants must have chronic ITP and an average platelet count under 30,000 µL with no prior bleeding at least four weeks before enrollment. There are a number of exclusions including having a known autoimmune disease in addition to ITP, or a known secondary ITP.

China

https://www.clinicaltrials.gov/ct2/show/NCT04428255?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=39

Recruiting

This study through Shandong University is an open-label randomized phase 2 study involving multiple sites looking at the immunomodulation effect of using high-dose dexamethasone alone vs in combination with Diammonium Glycyrrhizinate, a Chinese Herbal Medicinal salt that is believed to have anti-inflammatory, antioxidant, and hepatoprotective properties. Inclusion criteria is restricted to participants who have not received ITP treatment in past and are between the ages of 18-80 years. Participants must also have a platelet count less than 30,000 µL. Individuals who have secondary ITP or an immune deficiency (severe) will not be eligible to participate. Individuals with common comorbidities such as inflammatory bowel, hypertension, diabetes, and pregnancy will also be excluded from participation.

China

https://www.clinicaltrials.gov/ct2/show/NCT05023915?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=29

Actively recruiting

This phase 3 multicenter, open-label, long-term clinical trial is designed to determine the safety and efficacy of Efgartigimod (ARGX-113) PH20 given subcutaneously to adults (over 18 years) with primary ITP. Efgartigimod is a first-in-class investigational antibody fragment to target the neonatal Fc receptor (FcRn). Eligible participants must be at least 18 years of age.

Australia, Bulgaria, China, Italy, Japan, Poland, Portugal, Romania, Russian Federation, Thailand, Turkey, USA

https://www.clinicaltrials.gov/ct2/show/NCT04812925?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=16

Status: Recruiting

This phase two open-label study is designed to assess the safety and efficacy of using Zanubrutibin, a selective next-generation covalent BTK inhibitor, to treat immune thrombocytopenia. Zanubrutinib was developed by a company in China to be a stronger BTK inhibitor with less side effects. Eligible participants include adult patients with confirmed persistent or chronic ITP who have a platelet count less than 30,000µL and demonstrate corticosteroid resistance and failure to respond to at least one approved second-line therapy. The study is looking to enroll 30 participants.

China

https://www.clinicaltrials.gov/ct2/show/NCT05214391?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=25

Recruiting

The Peking University’s People’s Hospital is conducting a prospective observational study of switching Eltrombopag and rhTPO in primary ITP. This is a non-interventional study. Adult ITP patients between the age of 18-75 years who are steroid resistant and who receive rhTPO and then switched to eltrombopag will be enrolled into the study. Treatments are terminated if the platelet count increases above 100,000 uL. One of the goals of the study is to assess if there is cross-resistance between the two drugs for the treatment of ITP and to investigate the efficacy and safety of switching eltrombopag and RhTPO in adults with ITP.

China

https://www.clinicaltrials.gov/ct2/show/NCT04214951?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=34

Active, but no longer recruiting

Peking University People's Hospital is conducting a small prospective study looking at the effectiveness of using Zanubrutinib in adults with ITP between 18-70 years of age. They are looking to recruit 10 patients who live in China. Zanubrutinib is BTK kinase inhibitor that is currently used to treat refractory mantle cell lymphoma. Participants must have confirmed primary and refractory ITP (i.e., not responding to other ITP therapies), have a platelet count less than 30,000/μL or with bleeding symptoms. Exclusions include current treatment with chemotherapy or anticoagulants (“blood thinners”), infection with HIV or hepatitis B/C, or having cancer or other severe medical conditions. Pregnant (or nursing mothers) will be excluded from participating.

China

https://www.clinicaltrials.gov/ct2/show/NCT05279872?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=40

Status: Recruiting

Beijing InnoCare Pharma Tech Co., Ltd is conducting a phase II randomized, open, multi-center trial evaluating the efficacy of Orelabrutinib, a Bruton’s tyrosine kinase inhibitor, in adult ITP. The trial will begin with a phase IIa trial, consisting of a screening, treatment, and safety follow up period. Individuals between 18-80 years old with persistent or chronic ITP (>3 months from diagnosis) and have failed to respond to 1+ first-line ITP therapies are eligible to enrol. Individuals who had hemorrhage 4 weeks prior to screening, history of blood disorders other than ITP, cancer, organ transplantation, or have an active infection will be excluded. The selection criteria for the phase IIb trial will be released after obtaining a better understanding of the efficacy, risks, and benefits of the therapy upon completion of the phase IIa trial.

China

https://www.clinicaltrials.gov/ct2/show/NCT05232149?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=15

Recruiting

The First Affiliated Hospital of Soochow University is conducting a phase II trial investigating the use of an immunotherapy, known as anti-BCMA CAR-T cell therapy, for relapsed and refractory ITP. This type of therapy uses a genetically modified version of a patient’s T-cells (type of immune cell) to better fight cells that are responsible for producing autoantibodies. Eligible participants include refractory and relapsed ITP patients aged 18-65. Participants are ineligible to enrol if they have secondary ITP, history of thrombosis, cardiovascular disease, cancer, are pregnant, or have received CAR-T cell infusion in the 3 months prior to enrolment.

China

https://www.clinicaltrials.gov/ct2/show/NCT05315778?recrs=a&cond=Immune+Thrombocytopenia&draw=2

Recruiting

This single centre prospective study conducted through Qilu Hospital in affiliation with Shandong University focuses on assessing the efficacy and safety of atorvastatin (also known as Lipitor) in the treatment of adults with ITP between the ages of 18-80 years, who do not have bleeding associated with a low platelet count under 30K microliters. Participants can expect to receive atorvastatin for one year if assigned to the experimental group.

China

https://www.clinicaltrials.gov/ct2/show/NCT03692754?recrs=ab&cond=Immune+Thrombocytopenia&draw=2&rank=19

On-Hold

The Institute of Hematology & Blood Diseases Hospital in China is conducting a single-centre, prospective study to evaluate the efficacy and safety of avatrombopag in adult ITP patients who do not respond well to eltrombopag or recombinant human thrombopoietin (rhTPO). Avatrombopag is a thrombopoietin receptor agonist used to stimulate the bone marrow to produce more platelets. Enrollment is restricted to 20 adults who are over the age of 18 years and have been diagnosed with chronic (primary) ITP with platelet counts less than 30,000 µL.

China

https://www.clinicaltrials.gov/ct2/show/NCT04890041?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=28

Status: Recruiting

This phase 4 study by the Institute of Hematology & Blood Diseases Hospital is a prospective, open-label, single center clinical trial to evaluate the efficacy and safety of avatrombopag in Chinese adult patients (over 18 years) with ITP (Primary or Secondary). Participants must have autoantibody failure due to intolerance or resistance to eltrombopag or hetrombopag therapy. Participants must have platelet counts below 30,000 µL and must not have used Rituximab in the last three months or have active thyroid disease.

China

https://www.clinicaltrials.gov/ct2/show/NCT04993885?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=41

Recruiting

Peking University People’s Hospital is conducting a phase II trial evaluating Baricitinib, a Janus kinase inhibitor, in steroid-resistant or relapsed ITP. This single-arm, open-label study is recruiting participants aged 18-70 with primary ITP, a platelet count <30,000/µl for over 6 months and have failed at least one ITP treatment. Participants with secondary ITP, active or previous malignancy, active infection, are pregnant, or experienced a thrombotic event in the previous 24 weeks will be unable to enrol in the study.

China

https://www.clinicaltrials.gov/ct2/show/NCT05446831?recrs=a&cond=Immune+Thrombocytopenia&draw=2

Recruiting

This study is a multicenter randomized, double-blind trial conducted through Wuhan Union Hospital, China, where participants are divided into two groups to analyze the efficacy and safety of using recombinant TPO agents (rhTPO) and eltrombopag to treat primary ITP in adult patients. In one group, participants can expect to receive a subcutaneous rhTPO agonist injection or an oral placebo tablet. In the second group, participants can expect to receive oral eltrombopag or a placebo injection. This study will only include adult ITP patients between the ages of 18 years – 75 years in good health.

China

https://www.clinicaltrials.gov/ct2/show/NCT03771378?cond=Immune+Thrombocytopenia&draw=2&rank=34

Unknown status

This study takes place within the Institute of Hematology & Blood Diseases Hospital and is a prospective, open-label, nonrandomized, multi-center trial designed to compare the efficacy and safety of using eltrombopag with low-dose rituximab vs the best available therapy (therapy not consisting of eltrombopag coupled with rituximab) for adults between 18-65 years of age who have autoantibody failure due to intolerance or resistance to first-line therapy options. Participants with secondary ITP will be included. Participants will be excluded from participating if they have active secondary thyroid disease or have a psychiatric disorder clinically diagnosed. Participants who have had rituximab in the last six months, or who have had any thromboses (clots) or are at a higher risk for thrombosis will also be excluded from participation.

China

https://www.clinicaltrials.gov/ct2/show/NCT04915482?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=42

Recruiting

This phase 4 multicenter open-label trial is designed to compare the efficacy of using eltrombopag alone vs in combination with Rituximab in adult patients aged 18-60 years who have not responded to first-line therapies or splenectomy, including those who have relapsed ITP to determine if there is a benefit of combining therapies, and to determine the efficacy in previously treated ITP patients.

China

https://www.clinicaltrials.gov/ct2/show/NCT04518475?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=21

Recruiting

Qilu Hospital of Shandong University in China is conducting a phase two multicentre randomized controlled study to determine if adding diacerin, a slow-acting anthraquinone medicine also known as diacetylrhein used to treat joint diseases such as osteoarthritis, can elicit a good response in combination with eltrombopag. Study leads speculate that the addition of diacerein to eltrombopag may offer a sensitizer effect to maximize efficacy. Eligibility is restricted to adult participants with primary ITP who are 18 years of age or older and do not respond well (or at all) to eltrombopag, or who have relapsed following discontinuation of eltrombobag.

China

https://www.clinicaltrials.gov/ct2/show/NCT04917679

Recruiting

The Peking University People’s Hospital is conducting this phase two prospective multicenter clinical trial to investigate the efficacy and safety of using eltrombopag plus rhTPO vs eltrombopag alone for adult ITP patients (18-80 years) who are corticosteroid resistant or have relapsed ITP during the COVID-19 pandemic. Since they increase the number of platelets through different mechanisms, and previous studies demonstrated that they might exert synergic effect. The investigators hypothesized that the combination of these two agents could be a promising option for ITP treatment.

China

https://www.clinicaltrials.gov/ct2/show/NCT04516837?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=22

Recruiting

This prospective, case-controlled, study for adult ITP patients between the ages of 18-60 years who requiring splenectomy who have failed to respond to multiple first and second line therapies. The goal is to assess their immune system and to determine how this changes post-splenectomy. Patients would be required to have their splenectomy performed as part of the study. Their platelet count will be raised to a safe level for the procedure. Blood for analysis will be collected at various points before and after surgery in addition to analyzing a small amount of tissue from the spleen taken during surgery, which will be matched with healthy donors. Individuals with secondary ITP will be excluded.

China

https://www.clinicaltrials.gov/ct2/show/NCT03998059?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=36

Status: Recruiting

The Peking University People’s Hospital is conducting this single-arm phase one clinical trial to investigate the efficacy and safety of using a combination therapy of a fixed low-dose of eltrombopag coupled with recombinant human TPO for adults with ITP who are between the ages of 18-80 years, and who are resistant to steroids or have relapsed ITP.

China

https://www.clinicaltrials.gov/ct2/show/NCT04518878?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=4

Recruiting

This is a randomized single center prospective study in phase 4 conducted through Qilu Hospital in affiliation with Shandong University. The focus of this study is to assess the efficacy and safety of glycyrrhetinic acid in synergy with high-dose pulse dexamethasone therapy for the treatment of adults with ITP who have a platelet count less than 30K microliters. Participants are required to be between the ages of 18-80 years with newly-diagnosed primary immune thrombocytopenia (ITP). Participants can expect to receive oral compound glycyrrhizin three times a day for a month in combination with high-dose dexamethasone (for four days) if assigned to the experimental group.

China

https://www.clinicaltrials.gov/ct2/show/NCT03998982?recrs=ab&cond=Immune+Thrombocytopenia&draw=2&rank=17

Recruiting

This study through Peking University is a observational and prospective study to explore the diagnostic and therapeutic value of the gut microbiome in newly diagnosed ITP patients who qualify for first line corticosteroid treatment. The goal is to access the predictive value of the baseline gut microbiome for corticosteroid (GC) resistance/relapse and will (1) collect fecal samples from ITP patients at initial diagnosis (baseline) and after first-line GC treatment, (2) detect the composition of gut microbiome and related metabolites using metagenomic sequencing combined with metabolomics, (3) observe the impact of first-line treatment on gut microbiome, (4) explore the significance of gut microbiome in the diagnosis of ITP, and (5) identify the predictive value of baseline gut microbiome for GC resistance/relapse, thus to provide new ideas for clinical diagnosis and treatment of ITP. Participation is restricted to those who are over the age of 18 years and have a confirmed diagnosis of primary ITP.

China

https://www.clinicaltrials.gov/ct2/show/NCT05118126?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=56#contacts

Recruiting

This observational and prospective study developed through Peking University aims to test a novel machine learning algorithm to identify which individuals with ITP are at an increased risk for a critical bleed. The tool used to generate the algorithm is a web-based resource to integrate clinical and laboratory data across multiple centers in China to build a clinical prediction model. The study target is ITP patients who are newly diagnosed; however, eligibility includes all adults over the age of 18 years with a confirmed diagnosed of ITP.

China

https://www.clinicaltrials.gov/ct2/show/NCT05116423?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=18#contacts

Recruiting

A multicentre, randomised, double-blind,4-stages phase III study enrolled 414 patients with chronic, previously treated ITP. Dosage of Hetrombopag olamine could be adjusted to maintain platelet counts 50~250×109/L. Hetrombopag olamine is a non-peptide thrombopoietin (TPO) receptor agonist that attempts to increase platelet production.

China

https://clinicaltrials.gov/ct2/show/NCT03222843?recrs=a&cond=ITP&draw=2&rank=3

Status: Unknown

This is an open, one-armed, prospective study through Peking Union Medical College Hospital that is looking to assess how well a single dose of anti-CD20 monoclonal antibody combined with bortezomib (a type of chemotherapy) works in the treatment of relapsed refractory autoimmune hemolytic anemia. The study is also open to patients who have secondary Evans syndrome. Participants must be adults between the ages of 18-80 years.

China

https://www.clinicaltrials.gov/ct2/show/NCT04083014?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=35

Recruiting

This single-armed prospective study conducted through the Institute of Hematology & Blood Diseases Hospital in China aims to evaluate the safety and efficacy of human umbilical cord-derived mesenchymal stem cells（hUC-MSCs) to treat refractory immune thrombocytopenia. The study also aims to better understand how hUC-MSCs work mechanistically to treat ITP through observing changes in the immune function with human umbilical cord-derived mesenchymal stem cells（hUC-MSCs） after infusion. Eligibility includes adults between the ages of 18-60 years with a platelet count less than 30K microliters who have had a splenectomy more than 3 months prior to being recruited into the study. The investigators will observe the changes of immune function in refractory ITP patients after hUC-MSCs infusion at 7 time points, including one day before hUC-MSCs infusion, 2 weeks, 4 weeks, 8 weeks, 12 weeks, 16weeks and 28 weeks after hUC-MSCs infusion.

China

https://www.clinicaltrials.gov/ct2/show/NCT04014166?recrs=ab&cond=Immune+Thrombocytopenia&draw=2&rank=39

Recruiting

This is a phase two, single-arm, open-label study through Peking University People’s Hospital in China to study the efficacy and safety of acetylcysteine (also called N-acetylcysteine or NAC) and high dose dexamethasone for therapy in adults with newly diagnosed ITP. NAC is a therapy that has traditionally been used to treat cystic fibrosis and other respiratory conditions by reducing mucous secretions. Recently, it’s been studied in patients with other platelet disorders such as thrombotic thrombocytopenic purpura (TTP) and is now being investigated for its potential treatment of ITP. In this study, NAC would be given orally, three times a day, in combination with a four-day high-dose pulse treatment of dexamethasone. Eligibility is restricted to adult patients with newly diagnosed ITP who have never had cancer or a splenectomy, are between the ages of 18-80 years, and have not been treated previously with Rituxan®. Participants must also have a diagnosis of primary ITP and have a platelet count under 30,000μL.

China

https://clinicaltrials.gov/ct2/show/NCT04368598?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=13

Unknown status

Peking University People's Hospital is conducting a randomized control, multicenter, open-label study to look at the clinical benefit of using a combination of Iguratimod and Danazol together vs Danazol alone for the treatment of primary ITP in adults between the ages of 18-70 years. Iguratimod is a non-steroid anti-inflammatory disease-modifying anti-rheumatic drug (DMARD), a newer therapy used to treat patients with Rheumatoid Arthritis. It works by acting on T- and B-cells in the immune system and can inhibit the expression of various inflammatory factors in the body. The study is looking to recruit 100 adult participants who are steroid-resistant or steroid-dependant, i.e., patients who do not have a significant platelet increase in response to steroid treatment, or patients who are unable to wean off steroids without risking a decrease in their platelet count. Participants must have a platelet count less than 30,000/μL. Participants will be excluded it they have congestive heart failure, an arrhythmia, are pregnant or nursing, or have HIV, cancer, lupus, H.pylori, certain metabolic conditions, Hepatitis C (HCV) or other infections causing thrombocytopenia.

China

https://www.clinicaltrials.gov/ct2/show/NCT05281068?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=26

Status: Recruiting

Randomized, open-label, multicentre study to assess the efficacy and safety of the combination of low-dose rituximab and ATRA (All-trans retinoic acid) in patients with steroid-resistant/relapsed ITP.

China

https://clinicaltrials.gov/ct2/show/NCT03304288?cond=itp&cntry=CN&draw=2&rank=47

Recruiting

Randomized, open-label, multicentre study to assess the efficacy and safety of the combination of low-dose rituximab and ATRA (All-trans retinoic acid) in patients with steroid-resistant/relapsed ITP.

China

https://clinicaltrials.gov/ct2/show/NCT03304288?cond=ITP&cntry=CN&rank=48

Recruiting

This phase II trial, designed by Peking University People’s Hospital, is a randomized, open-label, multicenter study to assess the combination treatment of tacrolimus and danazol compared to danazol alone in steroid-resistant or relapsed ITP. Individuals with primary ITP between 18-70 years old and have relapsed or did not achieve a response with corticosteroids are eligible to enrol. Participants must have a platelet count less than 30x109/L at the time of enrollment. Individuals with secondary ITP, cancer, are pregnant, or were previously treated with danazol will not be ineligible to participate in this study.

China

https://www.clinicaltrials.gov/ct2/show/NCT05471050?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=2

Recruiting

Peking University People's Hospital is conducting a phase two, randomized, open-label, multicenter study to compare the efficacy and safety of using high-dose dexamethasone in combination with Tacrolimus, an immunosuppressive, compared to using high-dose dexamethasone alone as a first line therapy for adults with newly diagnosed primary ITP. Eligibility includes adults over the age of 18 years who have a platelet count under 30,000 µL or under 50,000 µL with bleeding symptoms. There are a range of exclusion that can be accessed through the below study link.

China

https://www.clinicaltrials.gov/ct2/show/NCT04747080?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=27

Recruiting

The Peking University People’s Hospital is conducting a phase two single-arm single center study to assess the efficacy and safety of using a combination of terbutaline and danazol in adult patients (over the age of 18 years) who are resistant to steroids or who have relapsed with ITP and have a platelet count less than 30,000. Terbutaline is a beta2-AR agonist that modulates T cell differentiation and effector cell function. It is branded as Bricanyl which is commonly used for asthma in inhalers.

China

https://www.clinicaltrials.gov/ct2/show/NCT04481282?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=7

Recruiting

This is a multisite, double-blind, randomized, placebo-controlled, and parallel study designed to evaluate the clinical efficacy and safety of Huaiqihuang granule for treatment to children with chronic primary immune thrombocytopenia. Huaiqihuang granule, a Chinese herbal medicine, has been found to increase nephrin and podocin production which leads to an increase in kidney function.

China

https://clinicaltrials.gov/ct2/show/NCT03201848?recrs=a&cond=ITP&draw=1&rank=25

Recruiting

Hutchison Medipharma Limited is conducting a study to assess the Safety, tolerability, pharmacokinetics and preliminary efficacy of HMPL-523, a syk inhibitor, in adult patients with immune thrombocytopenia. This is a randomized, double blinded, placebo controlled phase Ib study where participants can expect to receive the sky inhibitor for up to eight weeks. Participants must be refractory to other therapies and be between ages 18 years – 75 years of age, and have either persistent or chronic ITP.

China

https://www.clinicaltrials.gov/ct2/show/NCT03951623?cond=Immune+Thrombocytopenia&draw=2&rank=35

Recruiting

Novartis Pharmaceuticals and a number of participating medical centres/universities in China are conducting this multi-center single-arm study alongside the Institute of Hematology & Blood Diseases Hospital to evaluate the efficacy and safety of eltrombopag to treat thrombocytopenia associated with chronic hepatitis B virus infection. This study includes two parts. In the first part short-term efficacy, safety and tolerability of eltrombopag is evaluated. Any subjects who benefit from eltrombopag treatment (platelet count ≥ 50×109/L) will then be offered the option to enter part two designed to evaluate the long-term efficacy and safety of the drug. This study is open to patients with primary ITP who are over the age of 18 years and were diagnosed with an HBV-infection for at least 6 months prior to the study. They also have to have a platelet count of less than 30K microlitres <30 ×109/L.

China

https://www.clinicaltrials.gov/ct2/show/NCT03664518?titles=To+evaluate+the+efficacy+and+safety+of+eltrombopag+for+thrombocytopenia+with+chronic+HBV+infection&draw=2&rank=1

Recruiting

Swedish Orphan Biovitrum (SOBI) is conducting a phase 4, multicenter, observational study on adult ITP patients (over 18 years of age). Patients eligible for participation will, as part of their routine medical care, be receiving avatrombopag (Doptelet) for the treatment of ITP. The scope of the study is to collect both retrospective and prospective data. The prospective part of the study will be to collect data on usage, effectiveness, safety, patient- and clinician-reported outcomes in addition to health parameters. The retrospective part of the study will consist of collecting information on previous treatments, reason for treatment switch, healthcare resource use and, if applicable, avatrombopag treatment prior to enrollment. The retrospective data collection will be based on the information available in the patient's medical records. Eligible participants must have a documented diagnosis of ITP and have been treated with (or at enrollment prescribed) avatrombopag for ITP. Participants with secondary ITP, such as lupus or Evan syndrome will not be included in this study.

Croatia, Germany, Czechia, Ireland, Netherlands, Norway, Spain, Switzerland, United Kingdom

https://www.clinicaltrials.gov/ct2/show/NCT04943042?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=42

Actively Recruiting

Takeda pharmaceuticals is conducting a multi-centered phase two placebo-controlled double-blinded study to look at the safety, tolerability, and efficacy of a drug called TAK-079, which is a monoclonal antibody (anti-CD38 antibody). The trial is for adult ITP patients whose ITP is persistent or chronic, with a platelet count less than 30,000/µL for four consecutive weeks prior to participating in the trial. TAK-079 would be administered as a subcutaneous injection.

Bulgaria, Croatia, Germany, Italy, Slovenia, Ukraine, and USA.

https://www.clinicaltrials.gov/ct2/show/NCT04278924?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=11

Recruiting

Principia Biopharma is conducting a study of their oral drug PRN1008 in patients with ITP who are refractory or relapsed with no available and approved therapeutic options and have a platelet count <30,000/μL on two counts no sooner than 7 days apart in the 15 days before treatment begins. Preclinical findings show that PRN1008 decreases the activity of B cells and other inflammatory-causing immune cells, and in a mouse model of ITP show that PRN1008 can reduce platelet loss.

CZECH REPUBLIC

https://clinicaltrials.gov/ct2/show/NCT03395210?term=PRN1008&rank=1

Recruiting

Swedish Orphan Biovitrum (SOBI) is conducting a phase 4, multicenter, observational study on adult ITP patients (over 18 years of age). Patients eligible for participation will, as part of their routine medical care, be receiving avatrombopag (Doptelet) for the treatment of ITP. The scope of the study is to collect both retrospective and prospective data. The prospective part of the study will be to collect data on usage, effectiveness, safety, patient- and clinician-reported outcomes in addition to health parameters. The retrospective part of the study will consist of collecting information on previous treatments, reason for treatment switch, healthcare resource use and, if applicable, avatrombopag treatment prior to enrollment. The retrospective data collection will be based on the information available in the patient's medical records. Eligible participants must have a documented diagnosis of ITP and have been treated with (or at enrollment prescribed) avatrombopag for ITP. Participants with secondary ITP, such as lupus or Evan syndrome will not be included in this study.

Croatia, Germany, Czechia, Ireland, Netherlands, Norway, Spain, Switzerland, United Kingdom

https://www.clinicaltrials.gov/ct2/show/NCT04943042?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=42

Actively Recruiting

Pfizer is conducting an interventional phase two, open-label, one-arm, multicenter study to determine the safety and efficacy of a drug, PF-06835375, in adults with moderate-severe bleeding due to ITP. Specifically, the study will determine if the drug can increase platelet counts to reduce bleeding risks, without a significant side effect profile. PF-06835375 is a chemokine (CXCR5) receptor antagonist that works as an immune system regulator and has been reported as a critical chemokine regulator in several autoimmune diseases. Its mechanism of action is not fully understood yet. The drug would be provided through a subcutaneous injection every month for a total of three months. Eligible participants include adults (between the ages of 18-70 years) with primary persistent or chronic ITP (over 3 months since diagnosis) who have a platelet count less than 50,000/μL and have not experienced severe bleeding within 1 month. Individuals who have had a splenectomy within three months of the study initiation period will be excluded from participating.

Australia, Canada, Czechia, Hungary, Poland, USA

https://www.clinicaltrials.gov/ct2/show/NCT05070845?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=15

Status: Recruiting

Ostfold Hospital Trust is conducting a phase two multicenter clinical, open-label, (total) dose-escalating study with a safety run tacked on to explore the clinical activity, total dosage, and safety of daratumumab in adult ITP patients who have not responded adequately (or relapsed after) corticosteroids and at least one second-line therapy including rituximab and/or TPO-RA. Daratumumab is a human monoclonal antibody) that is received through a subcutaneous injection. This study aims to investigate the efficacy, the optimal number of treatments, and the safety  daratumumab in steroid-refractory or steroid-dependent ITP patients who fail to respond to at least one previous second-line therapy, including rituximab and/ or TPO agonist. The first three patients enrolled will be included in the safety run-in phase. The next nine patients will be included in cohort one and will receive eight weekly injections. If the response rate is less than 100%, the next nine patients will be included in cohort two and will receive eight weekly injections followed by two bi-weekly injections. Eligibility includes adult ITP patients 18 years of age or older with a platelet count less than 30,000 µL. Participants must also have previously failed to achieve response to (or has relapsed after) corticosteroid therapy and at least one second-line therapy. 

Denmark, France, Norway

https://www.clinicaltrials.gov/ct2/show/NCT04703621?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=48

Recruiting

Sohag University (Egypt) is conducting a study to investigate genetic differences in the FOXP3 gene and levels of interleukin 10 in ITP vs. non-ITP participants. It has been shown that the FOXP3 gene is related to the function of regulatory T-cells, an immune cell with important roles in autoimmune diseases. Similarly, interleukin 10 is a cytokine that regulates immune function and are released by regulatory T-cells. The study is recruiting patients with primary ITP that are 18+ years old. Participants with secondary ITP, other autoimmune disorders, or cancer will be ineligible to enrol in the study.

Egypt

https://www.clinicaltrials.gov/ct2/show/NCT05410249?recrs=a&cond=Immune+Thrombocytopenia&draw=2

Recruiting

This is a prospective, observational study conducted through Assiut University to investigate a potential relationship between the COVID-19 vaccine and the development of ITP. Participants aged 17-60 who received a COVID-19 vaccine at Assiut University Hospital (Egypt) between December 2021 to April 2023 are eligible to participate in the study. Those with previously diagnosed ITP, other causes of thrombocytopenia, or other autoimmune disorders will be excluded from the study.

Egypt

https://www.clinicaltrials.gov/ct2/show/NCT05455983?recrs=a&cond=Immune+Thrombocytopenia&draw=2

Recruiting

Assiut University is looking at whether treatment for H.pylori increases the overall platelet count in individuals who are infected with H. Pylori, a bacteria in the digestive tract that has been associated in some cases with ITP. Both adults and children over the age of one year are eligible to participate. Participants must have chronic ITP and be resistant to steroids and IVIg.

Egypt

https://clinicaltrials.gov/ct2/show/NCT03172676?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=14

Unknown status

This study plans to examine the quality of life of patient with chronic idiopathic thrombocytopenic purpura according to the type of treatment received.

France

https://clinicaltrials.gov/ct2/show/NCT03123497?recrs=a&cond=ITP&draw=2&rank=11

Unknown status

CARMEN is a clinical registry of all incident adult Immune thrombocytopenia (ITP) and Autoimmune Hemolytic anemia (AIHA) patients in the Midi-Pyrenees region (South of France). It is aimed at describing ITP and AIHA clinical features, assessing the risk-benefit ratio of second-line treatments (SLTs) and adherence to guidelines for ITP and AIHA management.

France

https://clinicaltrials.gov/ct2/show/NCT02877706?recrs=a&cond=ITP&draw=1&rank=38

Recruiting

This study is conducted in part by GlaxoSmithKline and is designed for adults who have Lupus (SLE) and/or ITP and are 18 years of age or older and may have been part of the ITP RITUX-PLUS clinical trial study or the Believe clinical trial (for SLE). The aim of this clinical trial is to determine if the use of anti-BAFF, an antibody, can restore a normal threshold of tolerance. This work would help to determine if 'double hit' therapy may help to reset the immune system toward a more tolerogenic profile, comparing the poly-reactivity and autoreactivity of immature (central tolerance) and naïve B cells (peripheral tolerance) in the blood along the RITUX-PLUS STUDY (NCT03154385 now completed) and the BLISS BELIEVE study (NCT03312907 now completed) after treatment (B-cell reconstitution time). No additional inclusion or exclusion criteria.

France

https://www.clinicaltrials.gov/ct2/show/NCT03975361?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=30

Recruiting

Swedish Orphan Biovitrum AB International (SOBI) pharmaceuticals is conducting a randomized, double-blind, placebo-controlled study with an open-label extension phase to evaluate the efficacy and safety of avatrombopag for the treatment of thrombocytopenia in pediatric subjects. The study is for participants between 1-17 years of age with a diagnosis of primary ITP for at least 6 months duration who have had an insufficient response to a previous treatment. Participants must have an average of two platelet counts less than 30×109/L with no single count greater than 35×109/L during the screening period. Avatrombopag is a thrombopoietin receptor agonist currently available for use in adults only with ITP.

France, Germany, Hungary, Poland, Russia, Turkey Ukraine, UK, and USA

https://clinicaltrials.gov/ct2/show/NCT04516967

Recruiting

This prospective case-based study is being conducted through the University Hospital of Bordeaux, in France in collaboration with the Ministry for Health and Solidarity. The goal is to investigate the molecular mechanisms underlying ITP, specifically those contributing to abnormalities of megakaryopoiesis (the process of platelet production).

France

https://www.clinicaltrials.gov/ct2/show/NCT04005638?recrs=ab&cond=Immune+Thrombocytopenia&draw=2&rank=46

Recruiting

This single center experimental study is based out of University Hospital in Bordeaux, France and is designed to assess the contribution of anti-platelet antibodies identified with monoclonal antibody immobilization of platelet antigens (MAIPA) to determine the autoimmune nature of a thrombocytopenia at diagnosis. The gold standard analysis for the determination of platelet antibodies, is the MAIPA assay. This trial is open to adults over the age of 18 years who have never been treated for their ITP and who have a platelet count less than 100 µL.

France

https://www.clinicaltrials.gov/ct2/show/NCT04800458?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=51

Recruiting

University Hospital, Bordeaux, is conducting a non-randomized diet-based study on patients who have systemic lupus erythematosus (SLE) to establish what role (if any) phytoestrogens play in the etiology of SLE and flare-ups of SLE. The study is an observational, monocentric, preliminary study aiming at determining if estrogenic isoflavones can be risk factors for SLE. The intervention will be the collection of extra blood and urine samples on SLE subjects and on autoimmune and healthy counterparts.

France

https://www.clinicaltrials.gov/ct2/show/NCT03421184?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=38

Recruiting

Toulouse University Hospital is a conducting a pharmacoepidemiological study designed to look at all adult ITP patient’s who are treated for their persistent (≥3 months) primary ITP in France to assess the clinical benefit-to-risk ratio of available second-line therapies. Participants will be recruited using the French Health Insurance System (SNIIRAM) database which covers the entire French population. Participants must be over the age of 18 years. They must also be registered in the database between 2009-2012.

France

https://www.clinicaltrials.gov/ct2/show/NCT03429660?recrs=ab&cond=Immune+Thrombocytopenia&draw=2&rank=37

Recruiting

TPO-RAs have been thought to play only a supporting role in ITP management. The purpose of this study is to demonstrate that a substantial proportion of ITP patients may achieve a prolonged response after TPO-RA discontinuation.

France

https://clinicaltrials.gov/ct2/show/NCT03119974?recrs=a&cond=ITP&rank=1

Unknown status

Monocytes/macrophages play a double role by phagocyting platelets recognized by autoantibodies and by maintaining the autoimmune response via their antigen-presenting cell functions. Fcgamma receptors (FcγR), that are represented by activating receptors (FcγRI, FcγRIIa, FcγRIII) and an inhibiting one (FcγRIIb), are involved in the regulation of macrophages and have been reported to be dysregulated in autoimmune diseases such as rheumatoid arthritis and systemic lupus erythematous.

The aim of this study is to compare the expression of FcγR in patients with ITP on circulating monocytes and on splenic macrophages.

France

https://clinicaltrials.gov/ct2/show/NCT02821572?recrs=a&cond=ITP&rank=4

Unknown

Principia Biopharma is conducting a phase three randomized, double-blind study of rilzabrutinib (previously referred to as PRN1008), and oral BTK inhibitor. They are looking to enroll patients with persistent or chronic ITP who are at least 12 years of age. Eligible patients must have an average platelet count of <30,000/μL on two counts (with no count higher than <35,000/μL) at least 5 days apart in the 14 days before treatment begins. The double-blind treatment period is 12-24 weeks depending on platelet response, followed by a 28-week open label period. Patients who respond to rilzabrutinib may enter a long term-extension. After the last dose of rilzabrutinib there will be a 4-week safety follow-up visit.

Argentina, Austria, Australia, Chile, France, Germany, Hungary, Israel, Italy, Norway, Poland, Russian Confederation, Singapore, South Korea, Spain, Thailand, Ukraine, UK, and the USA.

https://clinicaltrials.gov/ct2/show/NCT04562766

Recruiting

Ostfold Hospital Trust is conducting a phase two multicenter clinical, open-label, (total) dose-escalating study with a safety run tacked on to explore the clinical activity, total dosage, and safety of daratumumab in adult ITP patients who have not responded adequately (or relapsed after) corticosteroids and at least one second-line therapy including rituximab and/or TPO-RA. Daratumumab is a human monoclonal antibody) that is received through a subcutaneous injection. This study aims to investigate the efficacy, the optimal number of treatments, and the safety  daratumumab in steroid-refractory or steroid-dependent ITP patients who fail to respond to at least one previous second-line therapy, including rituximab and/ or TPO agonist. The first three patients enrolled will be included in the safety run-in phase. The next nine patients will be included in cohort one and will receive eight weekly injections. If the response rate is less than 100%, the next nine patients will be included in cohort two and will receive eight weekly injections followed by two bi-weekly injections. Eligibility includes adult ITP patients 18 years of age or older with a platelet count less than 30,000 µL. Participants must also have previously failed to achieve response to (or has relapsed after) corticosteroid therapy and at least one second-line therapy. 

Denmark, France, Norway

https://www.clinicaltrials.gov/ct2/show/NCT04703621?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=48

Recruiting

The Centre Hospitalier Universitaire de Saint Etienne has developed a clinical test that is designed to pinpoint the specific drug causing of drug-induced thrombocytopenia, using flow cytometry. Their Induction Test in vitro TIM (TITIM) is reported by the group to be simple, fast, inexpensive, and can easily be used in hospital laboratories. This study aims to validate this clinical test.

France

https://www.clinicaltrials.gov/ct2/show/NCT02862353?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=42

Recruiting

Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), PH20 administered subcutaneously. This is a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.

Argentina, Australia, Chile, Georgia, Ireland, Israel, Italy, Japan, Republic of Korea, New Zealand, Norway, Poland, Portugal, Romania, Russian Federation, Spain, Thailand, Turkey, and USA

https://www.clinicaltrials.gov/ct2/show/NCT04687072?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=14#contacts

Recruiting

Novartis Pharmaceuticals is conducting a phase two, multicentered, randomized, open label study to look at the safety and efficacy of using eltrombopag in combination with short-term pulse high dose of dexamethasone therapy in adult ITP patients who are newly diagnosed. The aim of the study is to compare the effectiveness of using this treatment approach compared to short-term high dose dexamethasone monotherapy alone as a first line treatment in this ITP patient population. Participants must be at least 18 years of age, and must have a platelet count less than 30 x 109/L

Germany

https://clinicaltrials.gov/ct2/show/NCT04346654?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=24

Recruiting

Swedish Orphan Biovitrum (SOBI) is conducting a phase 4, multicenter, observational study on adult ITP patients (over 18 years of age). Patients eligible for participation will, as part of their routine medical care, be receiving avatrombopag (Doptelet) for the treatment of ITP. The scope of the study is to collect both retrospective and prospective data. The prospective part of the study will be to collect data on usage, effectiveness, safety, patient- and clinician-reported outcomes in addition to health parameters. The retrospective part of the study will consist of collecting information on previous treatments, reason for treatment switch, healthcare resource use and, if applicable, avatrombopag treatment prior to enrollment. The retrospective data collection will be based on the information available in the patient's medical records. Eligible participants must have a documented diagnosis of ITP and have been treated with (or at enrollment prescribed) avatrombopag for ITP. Participants with secondary ITP, such as lupus or Evan syndrome will not be included in this study.

Croatia, Germany, Czechia, Ireland, Netherlands, Norway, Spain, Switzerland, United Kingdom

https://www.clinicaltrials.gov/ct2/show/NCT04943042?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=42

Actively Recruiting

Swedish Orphan Biovitrum AB International (SOBI) pharmaceuticals is conducting a randomized, double-blind, placebo-controlled study with an open-label extension phase to evaluate the efficacy and safety of avatrombopag for the treatment of thrombocytopenia in pediatric subjects. The study is for participants between 1-17 years of age with a diagnosis of primary ITP for at least 6 months duration who have had an insufficient response to a previous treatment. Participants must have an average of two platelet counts less than 30×109/L with no single count greater than 35×109/L during the screening period. Avatrombopag is a thrombopoietin receptor agonist currently available for use in adults only with ITP.

France, Germany, Hungary, Poland, Russia, Turkey Ukraine, UK, and USA

https://clinicaltrials.gov/ct2/show/NCT04516967

Recruiting

This phase two open-label multicenter study by Novartis Pharmaceuticals is designed to evaluate the efficacy and safety of iptacopan (LNP023) in participants with persistent or chronic ITP or with primary cold agglutinin disease. Iptacopan is a complement factor B inhibitor that targets the complement pathway of the immune system. ITP participants would be recruited into ‘cohort 1’ of the trial. Eligible participants include adult participants over 18 years of age who have had ITP for at least three months and have received at least one line of therapy previously and still have a low platelet count. Women of childbearing age are excluded from participation, in addition to those who have a confirmed diagnosed of secondary ITP.

Germany, Spain, and United Kingdom

https://www.clinicaltrials.gov/ct2/show/NCT05086744?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=49#contacts

Recruiting

Principia Biopharma is conducting a phase three randomized, double-blind study of rilzabrutinib (previously referred to as PRN1008), and oral BTK inhibitor. They are looking to enroll patients with persistent or chronic ITP who are at least 12 years of age. Eligible patients must have an average platelet count of <30,000/μL on two counts (with no count higher than <35,000/μL) at least 5 days apart in the 14 days before treatment begins. The double-blind treatment period is 12-24 weeks depending on platelet response, followed by a 28-week open label period. Patients who respond to rilzabrutinib may enter a long term-extension. After the last dose of rilzabrutinib there will be a 4-week safety follow-up visit.

Argentina, Austria, Australia, Chile, France, Germany, Hungary, Israel, Italy, Norway, Poland, Russian Confederation, Singapore, South Korea, Spain, Thailand, Ukraine, UK, and the USA.

https://clinicaltrials.gov/ct2/show/NCT04562766

Recruiting

Takeda pharmaceuticals is conducting a multi-centered phase two placebo-controlled double-blinded study to look at the safety, tolerability, and efficacy of a drug called TAK-079, which is a monoclonal antibody (anti-CD38 antibody). The trial is for adult ITP patients whose ITP is persistent or chronic, with a platelet count less than 30,000/µL for four consecutive weeks prior to participating in the trial. TAK-079 would be administered as a subcutaneous injection.

Bulgaria, Croatia, Germany, Italy, Slovenia, Ukraine, and USA.

https://www.clinicaltrials.gov/ct2/show/NCT04278924?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=11

Recruiting

Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), PH20 administered subcutaneously. This is a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.

Argentina, Australia, Chile, Georgia, Ireland, Israel, Italy, Japan, Republic of Korea, New Zealand, Norway, Poland, Portugal, Romania, Russian Federation, Spain, Thailand, Turkey, and USA

https://www.clinicaltrials.gov/ct2/show/NCT04687072?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=14#contacts

Recruiting

This is a phase two double-blind, randomized, multicenter study through Oscotec Inc. designed to evaluate the efficacy and safety of SKI-0-703, an oral SYK inhibitor. This study is targeting adult ITP patients who have persistent or chronic ITP and have failed to respond adequately to other approved therapies, or who have relapsed following exposure to other approved ITP treatments. SYK inhibitors play important roles in signalling within the immune system, particularly in the adaptive immune system. Inhibition of SYK leads to reduced antibody mediated destruction of platelets. Eligibility includes individuals diagnosed with ITP for at least three months who have a platelet count less than 30,000 µL and are at least 18 years of age.

USA, Greece, Republic of Korea, Poland, Spain

https://www.clinicaltrials.gov/ct2/show/NCT04056195?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=2

Recruiting

The Humanity & Health Medical Group Limited is overseeing a pilot study to assess whether twelve weeks of using eltrombopag and pulsed dexamethasone together can be used as ‘first-line’ therapy to evoke a sustained platelet count response in adults with ITP who have platelet counts below 30K microliters (or below 50K microliters with bleeding events). To be eligible to participate you must be an adult over 18 years of age, and you must not have tried any previous ITP therapies with the exception of a platelet transfusion.

Hong Kong

https://www.clinicaltrials.gov/ct2/show/NCT03830749?cond=Immune+Thrombocytopenia&draw=2&rank=44

On Hold

This is a non-randomized parallel pilot study conducting through the Chinese University of Hong Kong looking at the safety and efficacy of using fecal microbiota transplantation (FMT) for the treatment of ITP. FMT has been used previously in the treatment of other autoimmune disorders and has in some cases been shown to reduce symptoms of the disorder. FMT is an innovative investigational treatment that has demonstrated in randomized, controlled clinical trials to be effective in resolving infections caused by recurrent C. difficile that does not respond to antibiotics. FMT works by transferring fecal microbiota cells from a healthy donor into the colon of the receiving participant. This study is open to individuals with ITP of all ages.

Hong Kong

https://clinicaltrials.gov/ct2/show/NCT04014413?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=44

Recruiting

Swedish Orphan Biovitrum AB International (SOBI) pharmaceuticals is conducting a randomized, double-blind, placebo-controlled study with an open-label extension phase to evaluate the efficacy and safety of avatrombopag for the treatment of thrombocytopenia in pediatric subjects. The study is for participants between 1-17 years of age with a diagnosis of primary ITP for at least 6 months duration who have had an insufficient response to a previous treatment. Participants must have an average of two platelet counts less than 30×109/L with no single count greater than 35×109/L during the screening period. Avatrombopag is a thrombopoietin receptor agonist currently available for use in adults only with ITP.

France, Germany, Hungary, Poland, Russia, Turkey Ukraine, UK, and USA

https://clinicaltrials.gov/ct2/show/NCT04516967

Recruiting

Pfizer is conducting an interventional phase two, open-label, one-arm, multicenter study to determine the safety and efficacy of a drug, PF-06835375, in adults with moderate-severe bleeding due to ITP. Specifically, the study will determine if the drug can increase platelet counts to reduce bleeding risks, without a significant side effect profile. PF-06835375 is a chemokine (CXCR5) receptor antagonist that works as an immune system regulator and has been reported as a critical chemokine regulator in several autoimmune diseases. Its mechanism of action is not fully understood yet. The drug would be provided through a subcutaneous injection every month for a total of three months. Eligible participants include adults (between the ages of 18-70 years) with primary persistent or chronic ITP (over 3 months since diagnosis) who have a platelet count less than 50,000/μL and have not experienced severe bleeding within 1 month. Individuals who have had a splenectomy within three months of the study initiation period will be excluded from participating.

Australia, Canada, Czechia, Hungary, Poland, USA

https://www.clinicaltrials.gov/ct2/show/NCT05070845?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=15

Status: Recruiting

Principia Biopharma is conducting a phase three randomized, double-blind study of rilzabrutinib (previously referred to as PRN1008), and oral BTK inhibitor. They are looking to enroll patients with persistent or chronic ITP who are at least 12 years of age. Eligible patients must have an average platelet count of <30,000/μL on two counts (with no count higher than <35,000/μL) at least 5 days apart in the 14 days before treatment begins. The double-blind treatment period is 12-24 weeks depending on platelet response, followed by a 28-week open label period. Patients who respond to rilzabrutinib may enter a long term-extension. After the last dose of rilzabrutinib there will be a 4-week safety follow-up visit.

Argentina, Austria, Australia, Chile, France, Germany, Hungary, Israel, Italy, Norway, Poland, Russian Confederation, Singapore, South Korea, Spain, Thailand, Ukraine, UK, and the USA.

https://clinicaltrials.gov/ct2/show/NCT04562766

Recruiting

Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), PH20 administered subcutaneously. This is a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.

Argentina, Australia, Chile, Georgia, Ireland, Israel, Italy, Japan, Republic of Korea, New Zealand, Norway, Poland, Portugal, Romania, Russian Federation, Spain, Thailand, Turkey, and USA

https://www.clinicaltrials.gov/ct2/show/NCT04687072?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=14#contacts

Recruiting

Swedish Orphan Biovitrum (SOBI) is conducting a phase 4, multicenter, observational study on adult ITP patients (over 18 years of age). Patients eligible for participation will, as part of their routine medical care, be receiving avatrombopag (Doptelet) for the treatment of ITP. The scope of the study is to collect both retrospective and prospective data. The prospective part of the study will be to collect data on usage, effectiveness, safety, patient- and clinician-reported outcomes in addition to health parameters. The retrospective part of the study will consist of collecting information on previous treatments, reason for treatment switch, healthcare resource use and, if applicable, avatrombopag treatment prior to enrollment. The retrospective data collection will be based on the information available in the patient's medical records. Eligible participants must have a documented diagnosis of ITP and have been treated with (or at enrollment prescribed) avatrombopag for ITP. Participants with secondary ITP, such as lupus or Evan syndrome will not be included in this study.

Croatia, Germany, Czechia, Ireland, Netherlands, Norway, Spain, Switzerland, United Kingdom

https://www.clinicaltrials.gov/ct2/show/NCT04943042?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=42

Actively Recruiting

Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), PH20 administered subcutaneously. This is a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.

Argentina, Australia, Chile, Georgia, Ireland, Israel, Italy, Japan, Republic of Korea, New Zealand, Norway, Poland, Portugal, Romania, Russian Federation, Spain, Thailand, Turkey, and USA

https://www.clinicaltrials.gov/ct2/show/NCT04687072?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=14#contacts

Recruiting

Principia Biopharma is conducting a phase three randomized, double-blind study of rilzabrutinib (previously referred to as PRN1008), and oral BTK inhibitor. They are looking to enroll patients with persistent or chronic ITP who are at least 12 years of age. Eligible patients must have an average platelet count of <30,000/μL on two counts (with no count higher than <35,000/μL) at least 5 days apart in the 14 days before treatment begins. The double-blind treatment period is 12-24 weeks depending on platelet response, followed by a 28-week open label period. Patients who respond to rilzabrutinib may enter a long term-extension. After the last dose of rilzabrutinib there will be a 4-week safety follow-up visit.

Argentina, Austria, Australia, Chile, France, Germany, Hungary, Israel, Italy, Norway, Poland, Russian Confederation, Singapore, South Korea, Spain, Thailand, Ukraine, UK, and the USA.

https://clinicaltrials.gov/ct2/show/NCT04562766

Recruiting

Primary immune thrombocytopenia (ITP) is a hemorrhagic disorder. Spontaneous recovery within 12 months occurs in the majority of pediatric patients. Nevertheless, in 20–30% of children the disease is chronic. The impact extends to the patients' families, whose everyday life, in terms of interpersonal relationships and financial status, is adversely affected. This study investigated the ability of a narrative instrument to improve the quality of life of pediatric chronic ITP patients and their families and quantified the familial burden imposed by the illness.

Italy

https://www.frontiersin.org/articles/10.3389/fped.2019.00163/full

Complete

This phase 3 multicenter, open-label, long-term clinical trial is designed to determine the safety and efficacy of Efgartigimod (ARGX-113) PH20 given subcutaneously to adults (over 18 years) with primary ITP. Efgartigimod is a first-in-class investigational antibody fragment to target the neonatal Fc receptor (FcRn). Eligible participants must be at least 18 years of age.

Australia, Bulgaria, China, Italy, Japan, Poland, Portugal, Romania, Russian Federation, Thailand, Turkey, USA

https://www.clinicaltrials.gov/ct2/show/NCT04812925?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=16

Status: Recruiting

Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), PH20 administered subcutaneously. This is a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.

Argentina, Australia, Chile, Georgia, Ireland, Israel, Italy, Japan, Republic of Korea, New Zealand, Norway, Poland, Portugal, Romania, Russian Federation, Spain, Thailand, Turkey, and USA

https://www.clinicaltrials.gov/ct2/show/NCT04687072?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=14#contacts

Recruiting

The Hematology Project Foundation, part of the Gruppo Italiano Malattie EMatologiche dell'Adulto is creating an adult ITP patient registry for research purposes. Participants must be 18 years of age or older. The primary objective of their prospective research interests include assessing the number of adverse outcomes reported in ITP adult patients and to look at safety and efficacy of different treatment outcomes.

Italy

https://www.clinicaltrials.gov/ct2/show/NCT03465020?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=35

Recruiting

This is a multicenter, observational study designed through Gruppo Italiano Malattie EMatologiche dell'Adulto to assess the incidence of (1) ITP patients developing COVID-19 and (2) patients with COVID-19 developing ITP. Data will be collected from anonymized case report forms from various ITP centers in Italy. The trial is recruiting patients who are 18+ years old with confirmed ITP (persistent or chronic) and were diagnosed with COVID-19. Patients with COVID-19 who developed ITP during active illness or within 6 months after recovery are also eligible to participate in this study. 

Italy

https://www.clinicaltrials.gov/ct2/show/NCT04735588?recrs=a&cond=Immune+Thrombocytopenia&draw=2

Recruiting

Gruppo Italiano Malattie EMatologiche dell'Adulto is conducting this study to determine if the use of TPO agonists (specifically eltrombopag and romiplostim) can cause a sustained or persistent remission in adults patients with either persistent or chronic ITP who have failed to respond to at least one other therapy.

Italy

https://www.clinicaltrials.gov/ct2/show/NCT02298075?recrs=ab&cond=Immune+Thrombocytopenia&draw=2&rank=3

Unknown

Principia Biopharma is conducting a phase three randomized, double-blind study of rilzabrutinib (previously referred to as PRN1008), and oral BTK inhibitor. They are looking to enroll patients with persistent or chronic ITP who are at least 12 years of age. Eligible patients must have an average platelet count of <30,000/μL on two counts (with no count higher than <35,000/μL) at least 5 days apart in the 14 days before treatment begins. The double-blind treatment period is 12-24 weeks depending on platelet response, followed by a 28-week open label period. Patients who respond to rilzabrutinib may enter a long term-extension. After the last dose of rilzabrutinib there will be a 4-week safety follow-up visit.

Argentina, Austria, Australia, Chile, France, Germany, Hungary, Israel, Italy, Norway, Poland, Russian Confederation, Singapore, South Korea, Spain, Thailand, Ukraine, UK, and the USA.

https://clinicaltrials.gov/ct2/show/NCT04562766

Recruiting

Takeda pharmaceuticals is conducting a multi-centered phase two placebo-controlled double-blinded study to look at the safety, tolerability, and efficacy of a drug called TAK-079, which is a monoclonal antibody (anti-CD38 antibody). The trial is for adult ITP patients whose ITP is persistent or chronic, with a platelet count less than 30,000/µL for four consecutive weeks prior to participating in the trial. TAK-079 would be administered as a subcutaneous injection.

Bulgaria, Croatia, Germany, Italy, Slovenia, Ukraine, and USA.

https://www.clinicaltrials.gov/ct2/show/NCT04278924?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=11

Recruiting

This phase 3 multicenter, open-label, long-term clinical trial is designed to determine the safety and efficacy of Efgartigimod (ARGX-113) PH20 given subcutaneously to adults (over 18 years) with primary ITP. Efgartigimod is a first-in-class investigational antibody fragment to target the neonatal Fc receptor (FcRn). Eligible participants must be at least 18 years of age.

Australia, Bulgaria, China, Italy, Japan, Poland, Portugal, Romania, Russian Federation, Thailand, Turkey, USA

https://www.clinicaltrials.gov/ct2/show/NCT04812925?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=16

Status: Recruiting

Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), PH20 administered subcutaneously. This is a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.

Argentina, Australia, Chile, Georgia, Ireland, Israel, Italy, Japan, Republic of Korea, New Zealand, Norway, Poland, Portugal, Romania, Russian Federation, Spain, Thailand, Turkey, and USA

https://www.clinicaltrials.gov/ct2/show/NCT04687072?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=14#contacts

Recruiting

Sobi, Inc is conducting a phase III, multicenter, open-label clinical trial in Japan to evaluate avatrombopag, a thrombopoietin receptor agonist, in chronic adult ITP. Participants must be 18+ years old with chronic ITP (at least 12 months since diagnosis) with a platelet count <30x10⁹/L and were unresponsive to previous ITP therapy. Participants with secondary ITP, inherited thrombocytopenia, are pregnant, or have a history of thrombosis, cardiovascular disease, liver disease, or cancer will be ineligible for this study. Individuals who have received IVIG/anti-D/corticosteroids/other thrombopoietin receptor agonists 1 week prior to the first platelet count or splenectomy/rituximab 12 weeks prior to the first platelet count will also be excluded from this study.

Japan

https://www.clinicaltrials.gov/ct2/show/NCT05369208?recrs=a&cond=Immune+Thrombocytopenia&draw=2

Recruiting

This international ITP registry study through the South Eastern Sydney Local Health District and GlaxoSmithLine is looking to prospectively enrol newly diagnosed adult patients with ITP to follow them for a period of two years. This study is recruiting ITP patients over the age of 18 years from one of the international designated participating centres. The goal of this registry is to learn more about the clinical characteristics of ITP, including the prevalence of the disease in various places, the incidence of co-morbidities, treatment patterns, and possible adverse outcomes (resulting from treatment or disease). Multi-site participation of this registry project will be rolled out in stages.

Australia, Korea, Taiwan, Singapore, Malaysia, Japan, Thailand, and Turkey.

https://www.clinicaltrials.gov/ct2/show/NCT01970189?recrs=ab&cond=Immune+Thrombocytopenia&draw=2&rank=47

Recruiting

Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), PH20 administered subcutaneously. This is a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.

Argentina, Australia, Chile, Georgia, Ireland, Israel, Italy, Japan, Republic of Korea, New Zealand, Norway, Poland, Portugal, Romania, Russian Federation, Spain, Thailand, Turkey, and USA

https://www.clinicaltrials.gov/ct2/show/NCT04687072?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=14#contacts

Recruiting

This international ITP registry study through the South Eastern Sydney Local Health District and GlaxoSmithLine is looking to prospectively enrol newly diagnosed adult patients with ITP to follow them for a period of two years. This study is recruiting ITP patients over the age of 18 years from one of the international designated participating centres. The goal of this registry is to learn more about the clinical characteristics of ITP, including the prevalence of the disease in various places, the incidence of co-morbidities, treatment patterns, and possible adverse outcomes (resulting from treatment or disease). Multi-site participation of this registry project will be rolled out in stages.

Australia, Korea, Taiwan, Singapore, Malaysia, Japan, Thailand, and Turkey.

https://www.clinicaltrials.gov/ct2/show/NCT01970189?recrs=ab&cond=Immune+Thrombocytopenia&draw=2&rank=47

Recruiting

Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), PH20 administered subcutaneously. This is a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.

Argentina, Australia, Chile, Georgia, Ireland, Israel, Italy, Japan, Republic of Korea, New Zealand, Norway, Poland, Portugal, Romania, Russian Federation, Spain, Thailand, Turkey, and USA

https://www.clinicaltrials.gov/ct2/show/NCT04687072?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=14#contacts

Recruiting

The Servicio de Hematología, Hospital Universitario in Mexico is conducting a n open-label study looking at the benefit of using corticosteroids together with second line therapies as front-line treatment in adults with primary ITP. Specifically, they are looking to determine if the response rate and duration of therapy outweighs using corticosteroids alone. Participants will receive a 1-4 day pulse dose of steroid, and then four weeks of Rituximab and romiplostim. Participants will be evaluated at 4 weeks and 6 months. In this study, patients must be between 16-90 years and have a platelet count less than 30,000 µL. Exclusion: Previous splenectomy.

Mexico

https://www.clinicaltrials.gov/ct2/show/NCT04588194?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=25

Recruiting

Principia Biopharma is conducting a study of their oral drug PRN1008 in patients with ITP who are refractory or relapsed with no available and approved therapeutic options and have a platelet count <30,000/μL on two counts no sooner than 7 days apart in the 15 days before treatment begins. Preclinical findings show that PRN1008 decreases the activity of B cells and other inflammatory-causing immune cells, and in a mouse model of ITP show that PRN1008 can reduce platelet loss.

NETHERLANDS

https://clinicaltrials.gov/ct2/show/NCT03395210?term=PRN1008&rank=1

Recruiting

Swedish Orphan Biovitrum (SOBI) is conducting a phase 4, multicenter, observational study on adult ITP patients (over 18 years of age). Patients eligible for participation will, as part of their routine medical care, be receiving avatrombopag (Doptelet) for the treatment of ITP. The scope of the study is to collect both retrospective and prospective data. The prospective part of the study will be to collect data on usage, effectiveness, safety, patient- and clinician-reported outcomes in addition to health parameters. The retrospective part of the study will consist of collecting information on previous treatments, reason for treatment switch, healthcare resource use and, if applicable, avatrombopag treatment prior to enrollment. The retrospective data collection will be based on the information available in the patient's medical records. Eligible participants must have a documented diagnosis of ITP and have been treated with (or at enrollment prescribed) avatrombopag for ITP. Participants with secondary ITP, such as lupus or Evan syndrome will not be included in this study.

Croatia, Germany, Czechia, Ireland, Netherlands, Norway, Spain, Switzerland, United Kingdom

https://www.clinicaltrials.gov/ct2/show/NCT04943042?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=42

Actively Recruiting

Pfizer is conducting a phase one, randomized, double-blind, placebo controlled human trial to evaluate the safety, tolerability, and pharmacokinetics of PF-06755347, a recombinant fragment crystallisable (rFc), in healthy adult male patients between 18-40 years of age. Male participants must have no evidence or history of clinically significant hematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, psychiatric, neurologic, or allergic disease (including drug allergies, but excluding untreated, asymptomatic, seasonal allergies at the time of dosing). Participants with a history of autoimmune disorders and other conditions that compromise or impair the immune system are also excluded from participating. It is hoped that this therapy could be used to treat ITP if tolerable and safe in healthy controls.

New Zealand

https://clinicaltrials.gov/ct2/show/NCT03275740?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=37

Recruiting

Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), PH20 administered subcutaneously. This is a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.

Argentina, Australia, Chile, Georgia, Ireland, Israel, Italy, Japan, Republic of Korea, New Zealand, Norway, Poland, Portugal, Romania, Russian Federation, Spain, Thailand, Turkey, and USA

https://www.clinicaltrials.gov/ct2/show/NCT04687072?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=14#contacts

Recruiting

Principia Biopharma is conducting a study of their oral drug PRN1008 in patients with ITP who are refractory or relapsed with no available and approved therapeutic options and have a platelet count <30,000/μL on two counts no sooner than 7 days apart in the 15 days before treatment begins. Preclinical findings show that PRN1008 decreases the activity of B cells and other inflammatory-causing immune cells, and in a mouse model of ITP show that PRN1008 can reduce platelet loss.

NORWAY

https://clinicaltrials.gov/ct2/show/NCT03395210?term=PRN1008&rank=1

Recruiting

Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), PH20 administered subcutaneously. This is a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.

Argentina, Australia, Chile, Georgia, Ireland, Israel, Italy, Japan, Republic of Korea, New Zealand, Norway, Poland, Portugal, Romania, Russian Federation, Spain, Thailand, Turkey, and USA

https://www.clinicaltrials.gov/ct2/show/NCT04687072?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=14#contacts

Recruiting

Swedish Orphan Biovitrum (SOBI) is conducting a phase 4, multicenter, observational study on adult ITP patients (over 18 years of age). Patients eligible for participation will, as part of their routine medical care, be receiving avatrombopag (Doptelet) for the treatment of ITP. The scope of the study is to collect both retrospective and prospective data. The prospective part of the study will be to collect data on usage, effectiveness, safety, patient- and clinician-reported outcomes in addition to health parameters. The retrospective part of the study will consist of collecting information on previous treatments, reason for treatment switch, healthcare resource use and, if applicable, avatrombopag treatment prior to enrollment. The retrospective data collection will be based on the information available in the patient's medical records. Eligible participants must have a documented diagnosis of ITP and have been treated with (or at enrollment prescribed) avatrombopag for ITP. Participants with secondary ITP, such as lupus or Evan syndrome will not be included in this study.

Croatia, Germany, Czechia, Ireland, Netherlands, Norway, Spain, Switzerland, United Kingdom

https://www.clinicaltrials.gov/ct2/show/NCT04943042?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=42

Actively Recruiting

Principia Biopharma is conducting a phase three randomized, double-blind study of rilzabrutinib (previously referred to as PRN1008), and oral BTK inhibitor. They are looking to enroll patients with persistent or chronic ITP who are at least 12 years of age. Eligible patients must have an average platelet count of <30,000/μL on two counts (with no count higher than <35,000/μL) at least 5 days apart in the 14 days before treatment begins. The double-blind treatment period is 12-24 weeks depending on platelet response, followed by a 28-week open label period. Patients who respond to rilzabrutinib may enter a long term-extension. After the last dose of rilzabrutinib there will be a 4-week safety follow-up visit.

Argentina, Austria, Australia, Chile, France, Germany, Hungary, Israel, Italy, Norway, Poland, Russian Confederation, Singapore, South Korea, Spain, Thailand, Ukraine, UK, and the USA.

https://clinicaltrials.gov/ct2/show/NCT04562766

Recruiting

Ostfold Hospital Trust is conducting a phase two multicenter clinical, open-label, (total) dose-escalating study with a safety run tacked on to explore the clinical activity, total dosage, and safety of daratumumab in adult ITP patients who have not responded adequately (or relapsed after) corticosteroids and at least one second-line therapy including rituximab and/or TPO-RA. Daratumumab is a human monoclonal antibody) that is received through a subcutaneous injection. This study aims to investigate the efficacy, the optimal number of treatments, and the safety  daratumumab in steroid-refractory or steroid-dependent ITP patients who fail to respond to at least one previous second-line therapy, including rituximab and/ or TPO agonist. The first three patients enrolled will be included in the safety run-in phase. The next nine patients will be included in cohort one and will receive eight weekly injections. If the response rate is less than 100%, the next nine patients will be included in cohort two and will receive eight weekly injections followed by two bi-weekly injections. Eligibility includes adult ITP patients 18 years of age or older with a platelet count less than 30,000 µL. Participants must also have previously failed to achieve response to (or has relapsed after) corticosteroid therapy and at least one second-line therapy. 

Denmark, France, Norway

https://www.clinicaltrials.gov/ct2/show/NCT04703621?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=48

Recruiting

This multi-center randomized two-phase story through Ostfold Hospital Trust is looking to
determine if maintenance therapy with low-dose rituximab is superior to placebo in prolonging responses among ITP patients who achieved an initial response with rituximab. The study also seeks to establish if the response to rituximab is greater in combination with dexamethasone, and what the true overall response rate and complete response rate is to rituximab. Health related quality of life will also be measured. This study is open to adults over the age of 18 years with newly diagnosed or persistent ITP only who have a platelet count less than 30K microliters.

Norway

https://www.clinicaltrials.gov/ct2/show/NCT03010202?recrs=ab&cond=Immune+Thrombocytopenia&draw=2&rank=45

Recruiting

This phase 3 multicenter, open-label, long-term clinical trial is designed to determine the safety and efficacy of Efgartigimod (ARGX-113) PH20 given subcutaneously to adults (over 18 years) with primary ITP. Efgartigimod is a first-in-class investigational antibody fragment to target the neonatal Fc receptor (FcRn). Eligible participants must be at least 18 years of age.

Australia, Bulgaria, China, Italy, Japan, Poland, Portugal, Romania, Russian Federation, Thailand, Turkey, USA

https://www.clinicaltrials.gov/ct2/show/NCT04812925?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=16

Status: Recruiting

Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), PH20 administered subcutaneously. This is a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.

Argentina, Australia, Chile, Georgia, Ireland, Israel, Italy, Japan, Republic of Korea, New Zealand, Norway, Poland, Portugal, Romania, Russian Federation, Spain, Thailand, Turkey, and USA

https://www.clinicaltrials.gov/ct2/show/NCT04687072?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=14#contacts

Recruiting

Swedish Orphan Biovitrum AB International (SOBI) pharmaceuticals is conducting a randomized, double-blind, placebo-controlled study with an open-label extension phase to evaluate the efficacy and safety of avatrombopag for the treatment of thrombocytopenia in pediatric subjects. The study is for participants between 1-17 years of age with a diagnosis of primary ITP for at least 6 months duration who have had an insufficient response to a previous treatment. Participants must have an average of two platelet counts less than 30×109/L with no single count greater than 35×109/L during the screening period. Avatrombopag is a thrombopoietin receptor agonist currently available for use in adults only with ITP.

France, Germany, Hungary, Poland, Russia, Turkey Ukraine, UK, and USA

https://clinicaltrials.gov/ct2/show/NCT04516967

Recruiting

Pfizer is conducting an interventional phase two, open-label, one-arm, multicenter study to determine the safety and efficacy of a drug, PF-06835375, in adults with moderate-severe bleeding due to ITP. Specifically, the study will determine if the drug can increase platelet counts to reduce bleeding risks, without a significant side effect profile. PF-06835375 is a chemokine (CXCR5) receptor antagonist that works as an immune system regulator and has been reported as a critical chemokine regulator in several autoimmune diseases. Its mechanism of action is not fully understood yet. The drug would be provided through a subcutaneous injection every month for a total of three months. Eligible participants include adults (between the ages of 18-70 years) with primary persistent or chronic ITP (over 3 months since diagnosis) who have a platelet count less than 50,000/μL and have not experienced severe bleeding within 1 month. Individuals who have had a splenectomy within three months of the study initiation period will be excluded from participating.

Australia, Canada, Czechia, Hungary, Poland, USA

https://www.clinicaltrials.gov/ct2/show/NCT05070845?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=15

Status: Recruiting

Principia Biopharma is conducting a phase three randomized, double-blind study of rilzabrutinib (previously referred to as PRN1008), and oral BTK inhibitor. They are looking to enroll patients with persistent or chronic ITP who are at least 12 years of age. Eligible patients must have an average platelet count of <30,000/μL on two counts (with no count higher than <35,000/μL) at least 5 days apart in the 14 days before treatment begins. The double-blind treatment period is 12-24 weeks depending on platelet response, followed by a 28-week open label period. Patients who respond to rilzabrutinib may enter a long term-extension. After the last dose of rilzabrutinib there will be a 4-week safety follow-up visit.

Argentina, Austria, Australia, Chile, France, Germany, Hungary, Israel, Italy, Norway, Poland, Russian Confederation, Singapore, South Korea, Spain, Thailand, Ukraine, UK, and the USA.

https://clinicaltrials.gov/ct2/show/NCT04562766

Recruiting

This is a phase two double-blind, randomized, multicenter study through Oscotec Inc. designed to evaluate the efficacy and safety of SKI-0-703, an oral SYK inhibitor. This study is targeting adult ITP patients who have persistent or chronic ITP and have failed to respond adequately to other approved therapies, or who have relapsed following exposure to other approved ITP treatments. SYK inhibitors play important roles in signalling within the immune system, particularly in the adaptive immune system. Inhibition of SYK leads to reduced antibody mediated destruction of platelets. Eligibility includes individuals diagnosed with ITP for at least three months who have a platelet count less than 30,000 µL and are at least 18 years of age.

USA, Greece, Republic of Korea, Poland, Spain

https://www.clinicaltrials.gov/ct2/show/NCT04056195?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=2

Recruiting

This phase 3 multicenter, open-label, long-term clinical trial is designed to determine the safety and efficacy of Efgartigimod (ARGX-113) PH20 given subcutaneously to adults (over 18 years) with primary ITP. Efgartigimod is a first-in-class investigational antibody fragment to target the neonatal Fc receptor (FcRn). Eligible participants must be at least 18 years of age.

Australia, Bulgaria, China, Italy, Japan, Poland, Portugal, Romania, Russian Federation, Thailand, Turkey, USA

https://www.clinicaltrials.gov/ct2/show/NCT04812925?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=16

Status: Recruiting

Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), PH20 administered subcutaneously. This is a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.

Argentina, Australia, Chile, Georgia, Ireland, Israel, Italy, Japan, Republic of Korea, New Zealand, Norway, Poland, Portugal, Romania, Russian Federation, Spain, Thailand, Turkey, and USA

https://www.clinicaltrials.gov/ct2/show/NCT04687072?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=14#contacts

Recruiting

Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), PH20 administered subcutaneously. This is a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.

Argentina, Australia, Chile, Georgia, Ireland, Israel, Italy, Japan, Republic of Korea, New Zealand, Norway, Poland, Portugal, Romania, Russian Federation, Spain, Thailand, Turkey, and USA

https://www.clinicaltrials.gov/ct2/show/NCT04687072?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=14#contacts

Recruiting

This phase 3 multicenter, open-label, long-term clinical trial is designed to determine the safety and efficacy of Efgartigimod (ARGX-113) PH20 given subcutaneously to adults (over 18 years) with primary ITP. Efgartigimod is a first-in-class investigational antibody fragment to target the neonatal Fc receptor (FcRn). Eligible participants must be at least 18 years of age.

Australia, Bulgaria, China, Italy, Japan, Poland, Portugal, Romania, Russian Federation, Thailand, Turkey, USA

https://www.clinicaltrials.gov/ct2/show/NCT04812925?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=16

Status: Recruiting

Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), PH20 administered subcutaneously. This is a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.

Argentina, Australia, Chile, Georgia, Ireland, Israel, Italy, Japan, Republic of Korea, New Zealand, Norway, Poland, Portugal, Romania, Russian Federation, Spain, Thailand, Turkey, and USA

https://www.clinicaltrials.gov/ct2/show/NCT04687072?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=14#contacts

Recruiting

Swedish Orphan Biovitrum AB International (SOBI) pharmaceuticals is conducting a randomized, double-blind, placebo-controlled study with an open-label extension phase to evaluate the efficacy and safety of avatrombopag for the treatment of thrombocytopenia in pediatric subjects. The study is for participants between 1-17 years of age with a diagnosis of primary ITP for at least 6 months duration who have had an insufficient response to a previous treatment. Participants must have an average of two platelet counts less than 30×109/L with no single count greater than 35×109/L during the screening period. Avatrombopag is a thrombopoietin receptor agonist currently available for use in adults only with ITP.

France, Germany, Hungary, Poland, Russia, Turkey Ukraine, UK, and USA

https://clinicaltrials.gov/ct2/show/NCT04516967

Recruiting

This is a phase 3 multicenter randomized double-blinded comparative study in collaboration with AP Generium designed to evaluation the clinical safety and efficacy of GNR-069 (a biosimilar to Nplate) vs Nplate in patients with chronic ITP. This study also provides for the evaluation of pharmacokinetic parameters and immunogenicity. Eligibility includes participants with confirmed ITP who are between the ages of 18-75 years.

Russian Federation

https://www.clinicaltrials.gov/ct2/show/NCT05220878?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=44#contacts

Recruiting

Principia Biopharma is conducting a phase three randomized, double-blind study of rilzabrutinib (previously referred to as PRN1008), and oral BTK inhibitor. They are looking to enroll patients with persistent or chronic ITP who are at least 12 years of age. Eligible patients must have an average platelet count of <30,000/μL on two counts (with no count higher than <35,000/μL) at least 5 days apart in the 14 days before treatment begins. The double-blind treatment period is 12-24 weeks depending on platelet response, followed by a 28-week open label period. Patients who respond to rilzabrutinib may enter a long term-extension. After the last dose of rilzabrutinib there will be a 4-week safety follow-up visit.

Argentina, Austria, Australia, Chile, France, Germany, Hungary, Israel, Italy, Norway, Poland, Russian Confederation, Singapore, South Korea, Spain, Thailand, Ukraine, UK, and the USA.

https://clinicaltrials.gov/ct2/show/NCT04562766

Recruiting

Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), PH20 administered subcutaneously. This is a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.

Argentina, Australia, Chile, Georgia, Ireland, Israel, Italy, Japan, Republic of Korea, New Zealand, Norway, Poland, Portugal, Romania, Russian Federation, Spain, Thailand, Turkey, and USA

https://www.clinicaltrials.gov/ct2/show/NCT04687072?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=14#contacts

Recruiting

Principia Biopharma is conducting a phase three randomized, double-blind study of rilzabrutinib (previously referred to as PRN1008), and oral BTK inhibitor. They are looking to enroll patients with persistent or chronic ITP who are at least 12 years of age. Eligible patients must have an average platelet count of <30,000/μL on two counts (with no count higher than <35,000/μL) at least 5 days apart in the 14 days before treatment begins. The double-blind treatment period is 12-24 weeks depending on platelet response, followed by a 28-week open label period. Patients who respond to rilzabrutinib may enter a long term-extension. After the last dose of rilzabrutinib there will be a 4-week safety follow-up visit.

Argentina, Austria, Australia, Chile, France, Germany, Hungary, Israel, Italy, Norway, Poland, Russian Confederation, Singapore, South Korea, Spain, Thailand, Ukraine, UK, and the USA.

https://clinicaltrials.gov/ct2/show/NCT04562766

Recruiting

This international ITP registry study through the South Eastern Sydney Local Health District and GlaxoSmithLine is looking to prospectively enrol newly diagnosed adult patients with ITP to follow them for a period of two years. This study is recruiting ITP patients over the age of 18 years from one of the international designated participating centres. The goal of this registry is to learn more about the clinical characteristics of ITP, including the prevalence of the disease in various places, the incidence of co-morbidities, treatment patterns, and possible adverse outcomes (resulting from treatment or disease). Multi-site participation of this registry project will be rolled out in stages.

Australia, Korea, Taiwan, Singapore, Malaysia, Japan, Thailand, and Turkey.

https://www.clinicaltrials.gov/ct2/show/NCT01970189?recrs=ab&cond=Immune+Thrombocytopenia&draw=2&rank=47

Recruiting

Takeda pharmaceuticals is conducting a multi-centered phase two placebo-controlled double-blinded study to look at the safety, tolerability, and efficacy of a drug called TAK-079, which is a monoclonal antibody (anti-CD38 antibody). The trial is for adult ITP patients whose ITP is persistent or chronic, with a platelet count less than 30,000/µL for four consecutive weeks prior to participating in the trial. TAK-079 would be administered as a subcutaneous injection.

Bulgaria, Croatia, Germany, Italy, Slovenia, Ukraine, and USA.

https://www.clinicaltrials.gov/ct2/show/NCT04278924?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=11

Recruiting

Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), PH20 administered subcutaneously. This is a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.

Argentina, Australia, Chile, Georgia, Ireland, Israel, Italy, Japan, Republic of Korea, New Zealand, Norway, Poland, Portugal, Romania, Russian Federation, Spain, Thailand, Turkey, and USA

https://www.clinicaltrials.gov/ct2/show/NCT04687072?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=14#contacts

Recruiting

Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), PH20 administered subcutaneously. This is a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.

Argentina, Australia, Chile, Georgia, Ireland, Israel, Italy, Japan, Republic of Korea, New Zealand, Norway, Poland, Portugal, Romania, Russian Federation, Spain, Thailand, Turkey, and USA

https://www.clinicaltrials.gov/ct2/show/NCT04687072?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=14#contacts

Recruiting

The purpose of this study is to determine the efficacy of H. pylori eradication for the treatment of chronic or persistent immune thrombocytopenic purpura (ITP) patients with moderate thrombocytopenia. H. pylori can lead to increased inflammation that can exacerbate ITP symptoms.
This is a multi-center, open label, prospective randomized phase IIl study.

Republic of Korea

https://clinicaltrials.gov/ct2/show/NCT03177629?recrs=a&cond=ITP&draw=1&rank=20

Status: Unknown

Principia Biopharma is conducting a phase three randomized, double-blind study of rilzabrutinib (previously referred to as PRN1008), and oral BTK inhibitor. They are looking to enroll patients with persistent or chronic ITP who are at least 12 years of age. Eligible patients must have an average platelet count of <30,000/μL on two counts (with no count higher than <35,000/μL) at least 5 days apart in the 14 days before treatment begins. The double-blind treatment period is 12-24 weeks depending on platelet response, followed by a 28-week open label period. Patients who respond to rilzabrutinib may enter a long term-extension. After the last dose of rilzabrutinib there will be a 4-week safety follow-up visit.

Argentina, Austria, Australia, Chile, France, Germany, Hungary, Israel, Italy, Norway, Poland, Russian Confederation, Singapore, South Korea, Spain, Thailand, Ukraine, UK, and the USA.

https://clinicaltrials.gov/ct2/show/NCT04562766

Recruiting

This international ITP registry study through the South Eastern Sydney Local Health District and GlaxoSmithLine is looking to prospectively enrol newly diagnosed adult patients with ITP to follow them for a period of two years. This study is recruiting ITP patients over the age of 18 years from one of the international designated participating centres. The goal of this registry is to learn more about the clinical characteristics of ITP, including the prevalence of the disease in various places, the incidence of co-morbidities, treatment patterns, and possible adverse outcomes (resulting from treatment or disease). Multi-site participation of this registry project will be rolled out in stages.

Australia, Korea, Taiwan, Singapore, Malaysia, Japan, Thailand, and Turkey.

https://www.clinicaltrials.gov/ct2/show/NCT01970189?recrs=ab&cond=Immune+Thrombocytopenia&draw=2&rank=47

Recruiting

This is a phase two double-blind, randomized, multicenter study through Oscotec Inc. designed to evaluate the efficacy and safety of SKI-0-703, an oral SYK inhibitor. This study is targeting adult ITP patients who have persistent or chronic ITP and have failed to respond adequately to other approved therapies, or who have relapsed following exposure to other approved ITP treatments. SYK inhibitors play important roles in signalling within the immune system, particularly in the adaptive immune system. Inhibition of SYK leads to reduced antibody mediated destruction of platelets. Eligibility includes individuals diagnosed with ITP for at least three months who have a platelet count less than 30,000 µL and are at least 18 years of age.

USA, Greece, Republic of Korea, Poland, Spain

https://www.clinicaltrials.gov/ct2/show/NCT04056195?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=2

Recruiting

Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), PH20 administered subcutaneously. This is a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.

Argentina, Australia, Chile, Georgia, Ireland, Israel, Italy, Japan, Republic of Korea, New Zealand, Norway, Poland, Portugal, Romania, Russian Federation, Spain, Thailand, Turkey, and USA

https://www.clinicaltrials.gov/ct2/show/NCT04687072?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=14#contacts

Recruiting

Swedish Orphan Biovitrum (SOBI) is conducting a phase 4, multicenter, observational study on adult ITP patients (over 18 years of age). Patients eligible for participation will, as part of their routine medical care, be receiving avatrombopag (Doptelet) for the treatment of ITP. The scope of the study is to collect both retrospective and prospective data. The prospective part of the study will be to collect data on usage, effectiveness, safety, patient- and clinician-reported outcomes in addition to health parameters. The retrospective part of the study will consist of collecting information on previous treatments, reason for treatment switch, healthcare resource use and, if applicable, avatrombopag treatment prior to enrollment. The retrospective data collection will be based on the information available in the patient's medical records. Eligible participants must have a documented diagnosis of ITP and have been treated with (or at enrollment prescribed) avatrombopag for ITP. Participants with secondary ITP, such as lupus or Evan syndrome will not be included in this study.

Croatia, Germany, Czechia, Ireland, Netherlands, Norway, Spain, Switzerland, United Kingdom

https://www.clinicaltrials.gov/ct2/show/NCT04943042?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=42

Actively Recruiting

This phase two open-label multicenter study by Novartis Pharmaceuticals is designed to evaluate the efficacy and safety of iptacopan (LNP023) in participants with persistent or chronic ITP or with primary cold agglutinin disease. Iptacopan is a complement factor B inhibitor that targets the complement pathway of the immune system. ITP participants would be recruited into ‘cohort 1’ of the trial. Eligible participants include adult participants over 18 years of age who have had ITP for at least three months and have received at least one line of therapy previously and still have a low platelet count. Women of childbearing age are excluded from participation, in addition to those who have a confirmed diagnosed of secondary ITP.

Germany, Spain, and United Kingdom

https://www.clinicaltrials.gov/ct2/show/NCT05086744?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=49#contacts

Recruiting

Current diagnostic criteria for Immune ThrombocytoPenia (ITP) are mainly based on the presence of low numbers of platelets, excluding other multiple causes of thrombocytopenia, including immunodeficiencies, constitutional or acquired thrombocytopenia, hypersplenism and clonal hematological disorders such as MDS, disorders lymphoproliferative and acute myeloid leukemia (AML), among others. The analysis complementary tests for the diagnosis of ITP include studies basic systematic hematology, together with autoimmune assays and microbiological tests, while the evaluation of bone marrow is limited to elderly patients and/or patients resistant to treatment. In the present study it is planned a broad characterization immunophenotyping of multiple compartments of bone marrow cells and PB platelets from patients with recently diagnosed ITP and investigate their morphological antecedents, in order to identify those patients who show compatible clonal hematopoietic patterns with MDS evident (or at risk of development), as candidates to receive most appropriate therapeutic methods.

Spain

https://clinicaltrials.gov/ct2/show/NCT03469661?recrs=a&cond=ITP&draw=1&rank=15

Active, but no longer recruiting

Principia Biopharma is conducting a phase three randomized, double-blind study of rilzabrutinib (previously referred to as PRN1008), and oral BTK inhibitor. They are looking to enroll patients with persistent or chronic ITP who are at least 12 years of age. Eligible patients must have an average platelet count of <30,000/μL on two counts (with no count higher than <35,000/μL) at least 5 days apart in the 14 days before treatment begins. The double-blind treatment period is 12-24 weeks depending on platelet response, followed by a 28-week open label period. Patients who respond to rilzabrutinib may enter a long term-extension. After the last dose of rilzabrutinib there will be a 4-week safety follow-up visit.

Argentina, Austria, Australia, Chile, France, Germany, Hungary, Israel, Italy, Norway, Poland, Russian Confederation, Singapore, South Korea, Spain, Thailand, Ukraine, UK, and the USA.

https://clinicaltrials.gov/ct2/show/NCT04562766

Recruiting

This is a phase two double-blind, randomized, multicenter study through Oscotec Inc. designed to evaluate the efficacy and safety of SKI-0-703, an oral SYK inhibitor. This study is targeting adult ITP patients who have persistent or chronic ITP and have failed to respond adequately to other approved therapies, or who have relapsed following exposure to other approved ITP treatments. SYK inhibitors play important roles in signalling within the immune system, particularly in the adaptive immune system. Inhibition of SYK leads to reduced antibody mediated destruction of platelets. Eligibility includes individuals diagnosed with ITP for at least three months who have a platelet count less than 30,000 µL and are at least 18 years of age.

USA, Greece, Republic of Korea, Poland, Spain

https://www.clinicaltrials.gov/ct2/show/NCT04056195?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=2

Recruiting

Swedish Orphan Biovitrum (SOBI) is conducting a phase 4, multicenter, observational study on adult ITP patients (over 18 years of age). Patients eligible for participation will, as part of their routine medical care, be receiving avatrombopag (Doptelet) for the treatment of ITP. The scope of the study is to collect both retrospective and prospective data. The prospective part of the study will be to collect data on usage, effectiveness, safety, patient- and clinician-reported outcomes in addition to health parameters. The retrospective part of the study will consist of collecting information on previous treatments, reason for treatment switch, healthcare resource use and, if applicable, avatrombopag treatment prior to enrollment. The retrospective data collection will be based on the information available in the patient's medical records. Eligible participants must have a documented diagnosis of ITP and have been treated with (or at enrollment prescribed) avatrombopag for ITP. Participants with secondary ITP, such as lupus or Evan syndrome will not be included in this study.

Croatia, Germany, Czechia, Ireland, Netherlands, Norway, Spain, Switzerland, United Kingdom

https://www.clinicaltrials.gov/ct2/show/NCT04943042?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=42

Actively Recruiting

This study, conducted by the University Children’s Hospital Basel, is a phase 2 randomized open label trial for young adults (18 – 45 years) with newly diagnosed ITP. This trial aims to compare the effectiveness of using high-dose dexamethasone vs eltrombopag with high-dose dexamethasone. Participants must have a platelet count below 10, 000 µL, and must not have received any treatment for their newly diagnosed ITP. Participants who have a family history of low platelets (thrombocytopenia), a history of additional autoimmune conditions, or a suspected or confirmed diagnosed of secondary ITP, will be excluded from participation.

Switzerland

https://www.clinicaltrials.gov/ct2/show/NCT04812483?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=36

Actively recruiting

Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), PH20 administered subcutaneously. This is a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.

Argentina, Australia, Chile, Georgia, Ireland, Israel, Italy, Japan, Republic of Korea, New Zealand, Norway, Poland, Portugal, Romania, Russian Federation, Spain, Thailand, Turkey, and USA

https://www.clinicaltrials.gov/ct2/show/NCT04687072?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=14#contacts

Recruiting

This international ITP registry study through the South Eastern Sydney Local Health District and GlaxoSmithLine is looking to prospectively enrol newly diagnosed adult patients with ITP to follow them for a period of two years. This study is recruiting ITP patients over the age of 18 years from one of the international designated participating centres. The goal of this registry is to learn more about the clinical characteristics of ITP, including the prevalence of the disease in various places, the incidence of co-morbidities, treatment patterns, and possible adverse outcomes (resulting from treatment or disease). Multi-site participation of this registry project will be rolled out in stages.

Australia, Korea, Taiwan, Singapore, Malaysia, Japan, Thailand, and Turkey.

https://www.clinicaltrials.gov/ct2/show/NCT01970189?recrs=ab&cond=Immune+Thrombocytopenia&draw=2&rank=47

Recruiting

This phase 3 multicenter, open-label, long-term clinical trial is designed to determine the safety and efficacy of Efgartigimod (ARGX-113) PH20 given subcutaneously to adults (over 18 years) with primary ITP. Efgartigimod is a first-in-class investigational antibody fragment to target the neonatal Fc receptor (FcRn). Eligible participants must be at least 18 years of age.

Australia, Bulgaria, China, Italy, Japan, Poland, Portugal, Romania, Russian Federation, Thailand, Turkey, USA

https://www.clinicaltrials.gov/ct2/show/NCT04812925?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=16

Status: Recruiting

Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), PH20 administered subcutaneously. This is a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.

Argentina, Australia, Chile, Georgia, Ireland, Israel, Italy, Japan, Republic of Korea, New Zealand, Norway, Poland, Portugal, Romania, Russian Federation, Spain, Thailand, Turkey, and USA

https://www.clinicaltrials.gov/ct2/show/NCT04687072?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=14#contacts

Recruiting

Principia Biopharma is conducting a phase three randomized, double-blind study of rilzabrutinib (previously referred to as PRN1008), and oral BTK inhibitor. They are looking to enroll patients with persistent or chronic ITP who are at least 12 years of age. Eligible patients must have an average platelet count of <30,000/μL on two counts (with no count higher than <35,000/μL) at least 5 days apart in the 14 days before treatment begins. The double-blind treatment period is 12-24 weeks depending on platelet response, followed by a 28-week open label period. Patients who respond to rilzabrutinib may enter a long term-extension. After the last dose of rilzabrutinib there will be a 4-week safety follow-up visit.

Argentina, Austria, Australia, Chile, France, Germany, Hungary, Israel, Italy, Norway, Poland, Russian Confederation, Singapore, South Korea, Spain, Thailand, Ukraine, UK, and the USA.

https://clinicaltrials.gov/ct2/show/NCT04562766

Recruiting

This international ITP registry study through the South Eastern Sydney Local Health District and GlaxoSmithLine is looking to prospectively enrol newly diagnosed adult patients with ITP to follow them for a period of two years. This study is recruiting ITP patients over the age of 18 years from one of the international designated participating centres. The goal of this registry is to learn more about the clinical characteristics of ITP, including the prevalence of the disease in various places, the incidence of co-morbidities, treatment patterns, and possible adverse outcomes (resulting from treatment or disease). Multi-site participation of this registry project will be rolled out in stages.

Australia, Korea, Taiwan, Singapore, Malaysia, Japan, Thailand, and Turkey.

https://www.clinicaltrials.gov/ct2/show/NCT01970189?recrs=ab&cond=Immune+Thrombocytopenia&draw=2&rank=47

Recruiting

Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), PH20 administered subcutaneously. This is a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.

Argentina, Australia, Chile, Georgia, Ireland, Israel, Italy, Japan, Republic of Korea, New Zealand, Norway, Poland, Portugal, Romania, Russian Federation, Spain, Thailand, Turkey, and USA

https://www.clinicaltrials.gov/ct2/show/NCT04687072?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=14#contacts

Recruiting

This phase 3 multicenter, open-label, long-term clinical trial is designed to determine the safety and efficacy of Efgartigimod (ARGX-113) PH20 given subcutaneously to adults (over 18 years) with primary ITP. Efgartigimod is a first-in-class investigational antibody fragment to target the neonatal Fc receptor (FcRn). Eligible participants must be at least 18 years of age.

Australia, Bulgaria, China, Italy, Japan, Poland, Portugal, Romania, Russian Federation, Thailand, Turkey, USA

https://www.clinicaltrials.gov/ct2/show/NCT04812925?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=16

Status: Recruiting

Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), PH20 administered subcutaneously. This is a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.

Argentina, Australia, Chile, Georgia, Ireland, Israel, Italy, Japan, Republic of Korea, New Zealand, Norway, Poland, Portugal, Romania, Russian Federation, Spain, Thailand, Turkey, and USA

https://www.clinicaltrials.gov/ct2/show/NCT04687072?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=14#contacts

Recruiting

Swedish Orphan Biovitrum AB International (SOBI) pharmaceuticals is conducting a randomized, double-blind, placebo-controlled study with an open-label extension phase to evaluate the efficacy and safety of avatrombopag for the treatment of thrombocytopenia in pediatric subjects. The study is for participants between 1-17 years of age with a diagnosis of primary ITP for at least 6 months duration who have had an insufficient response to a previous treatment. Participants must have an average of two platelet counts less than 30×109/L with no single count greater than 35×109/L during the screening period. Avatrombopag is a thrombopoietin receptor agonist currently available for use in adults only with ITP.

France, Germany, Hungary, Poland, Russia, Turkey Ukraine, UK, and USA

https://clinicaltrials.gov/ct2/show/NCT04516967

Recruiting

This international ITP registry study through the South Eastern Sydney Local Health District and GlaxoSmithLine is looking to prospectively enrol newly diagnosed adult patients with ITP to follow them for a period of two years. This study is recruiting ITP patients over the age of 18 years from one of the international designated participating centres. The goal of this registry is to learn more about the clinical characteristics of ITP, including the prevalence of the disease in various places, the incidence of co-morbidities, treatment patterns, and possible adverse outcomes (resulting from treatment or disease). Multi-site participation of this registry project will be rolled out in stages.

Australia, Korea, Taiwan, Singapore, Malaysia, Japan, Thailand, and Turkey.

https://www.clinicaltrials.gov/ct2/show/NCT01970189?recrs=ab&cond=Immune+Thrombocytopenia&draw=2&rank=47

Recruiting

Principia Biopharma is conducting a study of their oral drug PRN1008 in patients with ITP who are refractory or relapsed with no available and approved therapeutic options and have a platelet count <30,000/μL on two counts no sooner than 7 days apart in the 15 days before treatment begins. Preclinical findings show that PRN1008 decreases the activity of B cells and other inflammatory-causing immune cells, and in a mouse model of ITP show that PRN1008 can reduce platelet loss.

UNITED KINGDOM

https://clinicaltrials.gov/ct2/show/NCT03395210?term=PRN1008&rank=1

Recruiting

Swedish Orphan Biovitrum (SOBI) is conducting a phase 4, multicenter, observational study on adult ITP patients (over 18 years of age). Patients eligible for participation will, as part of their routine medical care, be receiving avatrombopag (Doptelet) for the treatment of ITP. The scope of the study is to collect both retrospective and prospective data. The prospective part of the study will be to collect data on usage, effectiveness, safety, patient- and clinician-reported outcomes in addition to health parameters. The retrospective part of the study will consist of collecting information on previous treatments, reason for treatment switch, healthcare resource use and, if applicable, avatrombopag treatment prior to enrollment. The retrospective data collection will be based on the information available in the patient's medical records. Eligible participants must have a documented diagnosis of ITP and have been treated with (or at enrollment prescribed) avatrombopag for ITP. Participants with secondary ITP, such as lupus or Evan syndrome will not be included in this study.

Croatia, Germany, Czechia, Ireland, Netherlands, Norway, Spain, Switzerland, United Kingdom

https://www.clinicaltrials.gov/ct2/show/NCT04943042?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=42

Actively Recruiting

Swedish Orphan Biovitrum AB International (SOBI) pharmaceuticals is conducting a randomized, double-blind, placebo-controlled study with an open-label extension phase to evaluate the efficacy and safety of avatrombopag for the treatment of thrombocytopenia in pediatric subjects. The study is for participants between 1-17 years of age with a diagnosis of primary ITP for at least 6 months duration who have had an insufficient response to a previous treatment. Participants must have an average of two platelet counts less than 30×109/L with no single count greater than 35×109/L during the screening period. Avatrombopag is a thrombopoietin receptor agonist currently available for use in adults only with ITP.

France, Germany, Hungary, Poland, Russia, Turkey Ukraine, UK, and USA

https://clinicaltrials.gov/ct2/show/NCT04516967

Recruiting

This phase two open-label multicenter study by Novartis Pharmaceuticals is designed to evaluate the efficacy and safety of iptacopan (LNP023) in participants with persistent or chronic ITP or with primary cold agglutinin disease. Iptacopan is a complement factor B inhibitor that targets the complement pathway of the immune system. ITP participants would be recruited into ‘cohort 1’ of the trial. Eligible participants include adult participants over 18 years of age who have had ITP for at least three months and have received at least one line of therapy previously and still have a low platelet count. Women of childbearing age are excluded from participation, in addition to those who have a confirmed diagnosed of secondary ITP.

Germany, Spain, and United Kingdom

https://www.clinicaltrials.gov/ct2/show/NCT05086744?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=49#contacts

Recruiting

Principia Biopharma is conducting a phase three randomized, double-blind study of rilzabrutinib (previously referred to as PRN1008), and oral BTK inhibitor. They are looking to enroll patients with persistent or chronic ITP who are at least 12 years of age. Eligible patients must have an average platelet count of <30,000/μL on two counts (with no count higher than <35,000/μL) at least 5 days apart in the 14 days before treatment begins. The double-blind treatment period is 12-24 weeks depending on platelet response, followed by a 28-week open label period. Patients who respond to rilzabrutinib may enter a long term-extension. After the last dose of rilzabrutinib there will be a 4-week safety follow-up visit.

Argentina, Austria, Australia, Chile, France, Germany, Hungary, Israel, Italy, Norway, Poland, Russian Confederation, Singapore, South Korea, Spain, Thailand, Ukraine, UK, and the USA.

https://clinicaltrials.gov/ct2/show/NCT04562766

Recruiting

Swedish Orphan Biovitrum AB International (SOBI) pharmaceuticals is conducting a randomized, double-blind, placebo-controlled study with an open-label extension phase to evaluate the efficacy and safety of avatrombopag for the treatment of thrombocytopenia in pediatric subjects. The study is for participants between 1-17 years of age with a diagnosis of primary ITP for at least 6 months duration who have had an insufficient response to a previous treatment. Participants must have an average of two platelet counts less than 30×109/L with no single count greater than 35×109/L during the screening period. Avatrombopag is a thrombopoietin receptor agonist currently available for use in adults only with ITP.

France, Germany, Hungary, Poland, Russia, Turkey Ukraine, UK, and USA

https://clinicaltrials.gov/ct2/show/NCT04516967

Recruiting

Biopharma Plasma LLC is recruiting for a phase III trial to assess the safety and efficacy of intravenous immunoglobulin (company: Bioven) in treating chronic primary ITP in adults. Participants must be between 18-65 years old and have chronic primary ITP (>12 months since diagnosis) with a platelet count <30x109/L. Participants taking corticosteroids may be enrolled if the treatment dose has been stable for at least 2 weeks prior to enrolment. Individuals who received transfusions of IVIG in the previous 1 month, are intolerant to plasma preparations, pregnant, had splenectomy, or other blood disorders other than ITP will be unable to participate. Individuals who received other experimental ITP therapies, such as Rituximab, in the prior 3 months will not be able to participate.

Ukraine

https://www.clinicaltrials.gov/ct2/show/NCT05422365?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=7

Recruiting

Principia Biopharma is conducting a phase three randomized, double-blind study of rilzabrutinib (previously referred to as PRN1008), and oral BTK inhibitor. They are looking to enroll patients with persistent or chronic ITP who are at least 12 years of age. Eligible patients must have an average platelet count of <30,000/μL on two counts (with no count higher than <35,000/μL) at least 5 days apart in the 14 days before treatment begins. The double-blind treatment period is 12-24 weeks depending on platelet response, followed by a 28-week open label period. Patients who respond to rilzabrutinib may enter a long term-extension. After the last dose of rilzabrutinib there will be a 4-week safety follow-up visit.

Argentina, Austria, Australia, Chile, France, Germany, Hungary, Israel, Italy, Norway, Poland, Russian Confederation, Singapore, South Korea, Spain, Thailand, Ukraine, UK, and the USA.

https://clinicaltrials.gov/ct2/show/NCT04562766

Recruiting

Takeda pharmaceuticals is conducting a multi-centered phase two placebo-controlled double-blinded study to look at the safety, tolerability, and efficacy of a drug called TAK-079, which is a monoclonal antibody (anti-CD38 antibody). The trial is for adult ITP patients whose ITP is persistent or chronic, with a platelet count less than 30,000/µL for four consecutive weeks prior to participating in the trial. TAK-079 would be administered as a subcutaneous injection.

Bulgaria, Croatia, Germany, Italy, Slovenia, Ukraine, and USA.

https://www.clinicaltrials.gov/ct2/show/NCT04278924?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=11

Recruiting

This phase 3 multicenter, open-label, long-term clinical trial is designed to determine the safety and efficacy of Efgartigimod (ARGX-113) PH20 given subcutaneously to adults (over 18 years) with primary ITP. Efgartigimod is a first-in-class investigational antibody fragment to target the neonatal Fc receptor (FcRn). Eligible participants must be at least 18 years of age.

Australia, Bulgaria, China, Italy, Japan, Poland, Portugal, Romania, Russian Federation, Thailand, Turkey, USA

https://www.clinicaltrials.gov/ct2/show/NCT04812925?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=16

Status: Recruiting

Principia Biopharma is conducting a study of their oral drug PRN1008 in patients with ITP who are refractory or relapsed with no available and approved therapeutic options and have a platelet count <30,000/μL on two counts no sooner than 7 days apart in the 15 days before treatment begins. Preclinical findings show that PRN1008 decreases the activity of B cells and other inflammatory-causing immune cells, and in a mouse model of ITP show that PRN1008 can reduce platelet loss.

USA

https://clinicaltrials.gov/ct2/show/NCT03395210?term=PRN1008&rank=1

Recruiting

Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), PH20 administered subcutaneously. This is a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.

Argentina, Australia, Chile, Georgia, Ireland, Israel, Italy, Japan, Republic of Korea, New Zealand, Norway, Poland, Portugal, Romania, Russian Federation, Spain, Thailand, Turkey, and USA

https://www.clinicaltrials.gov/ct2/show/NCT04687072?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=14#contacts

Recruiting

Swedish Orphan Biovitrum AB International (SOBI) pharmaceuticals is conducting a randomized, double-blind, placebo-controlled study with an open-label extension phase to evaluate the efficacy and safety of avatrombopag for the treatment of thrombocytopenia in pediatric subjects. The study is for participants between 1-17 years of age with a diagnosis of primary ITP for at least 6 months duration who have had an insufficient response to a previous treatment. Participants must have an average of two platelet counts less than 30×109/L with no single count greater than 35×109/L during the screening period. Avatrombopag is a thrombopoietin receptor agonist currently available for use in adults only with ITP.

France, Germany, Hungary, Poland, Russia, Turkey Ukraine, UK, and USA

https://clinicaltrials.gov/ct2/show/NCT04516967

Recruiting

This study is conducted by the New York Medical College and is open for newly diagnosed pediatric ITP patients between 1 year – 21 years of age who have bleeding symptoms and show no evidence of a recent infection. Platelet counts less than 20, 000 µL are required. A bone marrow biopsy is preferred but not required. The aim of the study is to determine the effect of early administration of Rituximab on relapse rates following treatment. To determine impact on quality of life, the kids ITP tools (KIT) will be used according to protocol.

USA

https://www.clinicaltrials.gov/ct2/show/NCT04323748?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=40

Recruiting

Baylor College of Medicine is conducting a prospective, open label, randomized, two-arm, multi-center Phase 3 trial using eltrombopag, a thrombopoietin receptor agonist, in newly diagnosed children, between the ages of 1-18 years. Participants must have a platelet count below 30 X 109/L. Children will be randomized (2:1) into one of two groups, either the treatment group (where they will receive eltrombopag), or the standard group (where they will receive standard first line therapies). The goal of the study is to see which group elicits a greater overall platelet response.

USA

Recruiting

https://www.clinicaltrials.gov/ct2/show/NCT03939637?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=1

Conducted by Rigel Pharmaceuticals, this study is an observational assessment for adult patients with ITP between 18 years – 100 years of age who have not had a successful response to steroids and/or immunoglobulins. This study will focus on evaluating patient characteristics, treatment patterns, and clinical outcomes in addition to the safety of fostamatinib as a second-line therapy. Pregnant women are excluded from participating.

USA

https://www.clinicaltrials.gov/ct2/show/NCT04904276?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=23

Recruiting

Swedish Orphan Biovitrum AB International (SOBI) pharmaceuticals is conducting a phase 4, prospective, multi-center, open-label study to evaluate the safety, platelet count response, and subject reported satisfaction of treatment in adult subjects (18 years of age or older) with chronic ITP after switching to avatrombopag from eltrombopag or romiplostim. This study is for participants who have been undergoing treatment for primary ITP with eltrombopag or romiplostim for at least 90 days. Participants must have had a previous response (at any time) to either eltrombopag or romiplostim, defined as at least 2 platelet counts above 50,000uL. Patients will be treated with avatrombopag for up to 90 days.

Recruiting

USA

https://clinicaltrials.gov/ct2/show/NCT04638829

This global ITP registry study is FDA approved and developed in partnership with the National Organization for Rare Disorders (NORD) to follow ITP patients of all ages, in all phases of disease to learn more about the natural history of ITP. The goal of the registry is to learn more about the clinical characteristics of ITP, the incidence of co-morbidities, treatment patterns, adverse outcomes, how quality of life is impacted, how family and socioeconomics is affected, and how ITP patients respond to COVID-19 and vaccinations.  Note: The registry is in English only, currently.

USA, Canada, International (all places).

https://www.pdsa.org/registry.html

Recruiting

This is a phase one single-arm pilot study taking place through Baylor College of Medicine. The study will focus on post-IVIG treatment in children with ITP in an attempt to reduce the incidence of potential IVIG side-effects. A pilot project is a mini project designed to determine if a larger study is warranted. The pilot project will assess the (1) feasibility of enrollment and the willingness of families to participate in a scheduled medication regimen and (2) adherence of patients and families to the scheduled medication regimen. Children with ITP between the ages of 1 month – 18 years are eligible to participate. Patients with previous serum reactions, such as anaphylaxis will be excluded from enrolling.

USA

https://www.clinicaltrials.gov/ct2/show/NCT04741139?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=5

Recruiting

Pfizer is conducting an interventional phase two, open-label, one-arm, multicenter study to determine the safety and efficacy of a drug, PF-06835375, in adults with moderate-severe bleeding due to ITP. Specifically, the study will determine if the drug can increase platelet counts to reduce bleeding risks, without a significant side effect profile. PF-06835375 is a chemokine (CXCR5) receptor antagonist that works as an immune system regulator and has been reported as a critical chemokine regulator in several autoimmune diseases. Its mechanism of action is not fully understood yet. The drug would be provided through a subcutaneous injection every month for a total of three months. Eligible participants include adults (between the ages of 18-70 years) with primary persistent or chronic ITP (over 3 months since diagnosis) who have a platelet count less than 50,000/μL and have not experienced severe bleeding within 1 month. Individuals who have had a splenectomy within three months of the study initiation period will be excluded from participating.

Australia, Canada, Czechia, Hungary, Poland, USA

https://www.clinicaltrials.gov/ct2/show/NCT05070845?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=15

Status: Recruiting

Principia Biopharma is conducting a phase three randomized, double-blind study of rilzabrutinib (previously referred to as PRN1008), and oral BTK inhibitor. They are looking to enroll patients with persistent or chronic ITP who are at least 12 years of age. Eligible patients must have an average platelet count of <30,000/μL on two counts (with no count higher than <35,000/μL) at least 5 days apart in the 14 days before treatment begins. The double-blind treatment period is 12-24 weeks depending on platelet response, followed by a 28-week open label period. Patients who respond to rilzabrutinib may enter a long term-extension. After the last dose of rilzabrutinib there will be a 4-week safety follow-up visit.

Argentina, Austria, Australia, Chile, France, Germany, Hungary, Israel, Italy, Norway, Poland, Russian Confederation, Singapore, South Korea, Spain, Thailand, Ukraine, UK, and the USA.

https://clinicaltrials.gov/ct2/show/NCT04562766

Recruiting

Takeda pharmaceuticals is conducting a multi-centered phase two placebo-controlled double-blinded study to look at the safety, tolerability, and efficacy of a drug called TAK-079, which is a monoclonal antibody (anti-CD38 antibody). The trial is for adult ITP patients whose ITP is persistent or chronic, with a platelet count less than 30,000/µL for four consecutive weeks prior to participating in the trial. TAK-079 would be administered as a subcutaneous injection.

Bulgaria, Croatia, Germany, Italy, Slovenia, Ukraine, and USA.

https://www.clinicaltrials.gov/ct2/show/NCT04278924?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=11

Recruiting

This is a prospective, open-label, single-arm, multicenter, Phase 4 study that is evaluating the efficacy and safety of PANZYGA, an IVIg product designed to be used as an antibody replacement therapy in ITP chronic ITP patients between the ages of 1 year – 18 years of age in participants who have a platelet count less than 30K microliters.

USA

https://www.clinicaltrials.gov/ct2/show/NCT03866798?cond=Immune+Thrombocytopenia&draw=2&rank=33

Recruiting

This is a phase two double-blind, randomized, multicenter study through Oscotec Inc. designed to evaluate the efficacy and safety of SKI-0-703, an oral SYK inhibitor. This study is targeting adult ITP patients who have persistent or chronic ITP and have failed to respond adequately to other approved therapies, or who have relapsed following exposure to other approved ITP treatments. SYK inhibitors play important roles in signalling within the immune system, particularly in the adaptive immune system. Inhibition of SYK leads to reduced antibody mediated destruction of platelets. Eligibility includes individuals diagnosed with ITP for at least three months who have a platelet count less than 30,000 µL and are at least 18 years of age.

USA, Greece, Republic of Korea, Poland, Spain

https://www.clinicaltrials.gov/ct2/show/NCT04056195?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=2

Recruiting