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

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

Alpine Immune Sciences is conducting a Phase 1b Open-Label Study to Assess the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of a new treatment for immune thrombocytopenia (and other autoimmune cytopenias) called Povetacicept (also known as APLN-303). This study is open to adults with persistent or chronic ITP. Povetacicept is a is a dual B cell cytokine antagonist engineered to inhibit BAFF (BLyS) and APRIL which are clinically validated cytokine pathways that play a key role in B cell development, differentiation, and survival. The drug will be provided to participants every 4 weeks for 6 months, with the possibility of participating in a 6-month study treatment extension period. Adult participants must have shown failure to at least two previous treatments for ITP, including exposure to a TPO-RA, unless otherwise contraindicated or unavailable to them. Participants must also have a documented platelet count of less than 30,000 µL.

Australia, Canada, USA

https://www.clinicaltrials.gov/study/NCT05757570?term=%20Povetacicept%20&rank=2

Actively Recruiting

Novartis Pharmaceuticals is conducting a Phase 3 study involving multiple centers designed to assess the efficacy and safety of using two different doses of ianalumab, a monoclonal antibody. Eligibility is restricted to adults with primary ITP who are over the age of 18 years, and who had a platelet count less than 30,000 µL before starting first line therapy options and meet criteria for treatment.

After completion of the screening period to ensure eligibility, participants will be given (randomly) either ianalumab or placebo as a healthy control to compare responses. All participants will be monitored for efficacy and/or safety.

Participants with current serious bleeding will be excluded, in addition to those who have had a previous splenectomy or ITP treatment within 28 days prior to the start of the study post-screening. Participants will also be excluded if they have used rituximab in past and have an absolute neutrophil count below 1.0 G/L at randomization. Anyone with concurrent coagulation disorders and/or receiving anti-platelet or anticoagulant medication (with an exemption of low dose of acetylsalicylic acid) will also be excluded from participating in this study.

 Australia, Belgium, Czechia, France, Germany, Hong Kong, Hungary, Japan, Malaysia, Norway, Singapore, Spain, Romania, United Kingdom, and United States.

https://www.clinicaltrials.gov/ct2/show/NCT05653349?recrs=a&cond=immune+thrombocytopenia&draw=2&rank=15

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

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

Novartis Pharmaceuticals is conducting a Phase 3 study involving multiple centers designed to assess the efficacy and safety of using two different doses of ianalumab, a monoclonal antibody. Eligibility is restricted to adults with primary ITP who are over the age of 18 years, and who had a platelet count less than 30,000 µL before starting first line therapy options and meet criteria for treatment.

After completion of the screening period to ensure eligibility, participants will be given (randomly) either ianalumab or placebo as a healthy control to compare responses. All participants will be monitored for efficacy and/or safety.

Participants with current serious bleeding will be excluded, in addition to those who have had a previous splenectomy or ITP treatment within 28 days prior to the start of the study post-screening. Participants will also be excluded if they have used rituximab in past and have an absolute neutrophil count below 1.0 G/L at randomization. Anyone with concurrent coagulation disorders and/or receiving anti-platelet or anticoagulant medication (with an exemption of low dose of acetylsalicylic acid) will also be excluded from participating in this study.

 Australia, Belgium, Czechia, France, Germany, Hong Kong, Hungary, Japan, Malaysia, Norway, Singapore, Spain, Romania, United Kingdom, and United States.

https://www.clinicaltrials.gov/ct2/show/NCT05653349?recrs=a&cond=immune+thrombocytopenia&draw=2&rank=15

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, Greece, Italy, Slovenia, Spain, and USA

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

Status: Recruiting

Alpine Immune Sciences is conducting a Phase 1b Open-Label Study to Assess the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of a new treatment for immune thrombocytopenia (and other autoimmune cytopenias) called Povetacicept (also known as APLN-303). This study is open to adults with persistent or chronic ITP. Povetacicept is a is a dual B cell cytokine antagonist engineered to inhibit BAFF (BLyS) and APRIL which are clinically validated cytokine pathways that play a key role in B cell development, differentiation, and survival. The drug will be provided to participants every 4 weeks for 6 months, with the possibility of participating in a 6-month study treatment extension period. Adult participants must have shown failure to at least two previous treatments for ITP, including exposure to a TPO-RA, unless otherwise contraindicated or unavailable to them. Participants must also have a documented platelet count of less than 30,000 µL.

Australia, Canada, USA

https://www.clinicaltrials.gov/study/NCT05757570?term=%20Povetacicept%20&rank=2

Actively 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

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 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

Institute of Hematology & Blood Diseases Hospital is conducting a phase 2 trial to evaluate the safety and efficacy of giving a CM313 injection (which is an anti-Human CD38 Monoclonal Antibody) to treat ITP in adult patients (over 18 years) who are not responsive to steroids, or are steroid-dependent, and may have fail to respond to at least one second-line therapy, including rituximab and/ or TPO agonist. Eligibility is restricted to those who are either in the persistent or chronic phase of ITP (having ITP for more than 3 months) with a platelet count less than 30,000 µL.

China

https://www.clinicaltrials.gov/ct2/show/NCT05694767?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=22

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 Institute of Hematology & Blood Diseases Hospital is conducting a multicenter phase 4 study to compare the efficacy and safety of an optimized recombinant thrombopoietin receptor agonist (rhTPO) treatment compared to using eltrombopag (Promacta/Revolade). Total treatment duration in the study is 6 weeks. The primary objective is to determine the time it takes to achieve a platelet count over 50,000 during 6 weeks of observation following treatment. Participation is restricted to adults with primary ITP between the ages of 18-85 years, who have a baseline platelet count of less than 30,000.

Participants with refractory ITP, or a prior history of arterial or venous thrombosis, or thrombophilia in recent 1 year, will be excluded from the study. In addition, participants who have previously received any platelet increasing drug such as rhTPO, thrombopoietin receptor agonis t(TPO-RA) within 30 days and those who are known non-responders to rhTPO or TPO-RA therapy will also be unable to participate in the study.

China

https://www.clinicaltrials.gov/ct2/show/NCT05583838?recrs=a&cond=Immune+Thrombocytopenia&cntry=CN&draw=2&rank=30

Actively 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 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 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

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

Jiangsu HengRui Medicine Co., Ltd is conducting a two-part, double-blind, randomized, placebo-controlled, and open-label phase 3 study. The purpose of this study is to investigate the efficacy, safety of Hetrombopag, another thrombopoietin-receptor agonist, in children with previously treated chronic immune thrombocytopenia between 6 and 17 years of age. In part A, patients will receive Hetrombopag for 8 weeks. In part B, all patients will receive Hetrombopag for 24 weeks.

China

https://www.clinicaltrials.gov/ct2/show/NCT04737850?recrs=a&cond=immune+thrombocytopenia&draw=3&rank=8

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

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

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

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

This multicenter prospective study conducted through the Institute of Hematology & Blood Diseases Hospital in China is looking to recruit 100 ITP patients at least 14 years of age or older to see how well the use of a thrombopoetin-receptor agonist (TPO-RA) is in treating ITP after failure to first-line therapies. The TPO-RAs used in this study include eltrombopag (Promacta/Revolade), Herombopag (Hengqu), and Avatrombopag (Doptelet). Recruitment criteria also includes having a diagnosis of primary ITP with two back-to-back platelet counts less than 30,000 µL or having two bleeding events less than seven days apart. Exclusion criteria includes previous use of TPO-RAs for a maximum of four weeks or two weeks with no response (defined as not achieving a platelet count higher than 30,000 after treatment or having bleeding with little change from baseline platelet count.

China

https://www.clinicaltrials.gov/ct2/show/NCT04890041?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=26

Status: Recruiting

Fujian Medical University Union Hospital is conducting a multicentered, phase 4, randomized controlled study looking at the effectiveness of two different dosing regimens of intravenous immune globulin (IVIG) in the treatment of children with newly diagnosed immune thrombocytopenia. Eligibility is restricted to those who are between the ages of 29 days to 14 years of age who have primary ITP and have not been treated for their ITP. Participants must also have a platelet count less than 20,000 µL. Participants will be excluded if they had any serious internal organ bleeding requiring emergency treatment in the last six months. ITP participants who are female and have started menses (menstruation) will also be excluded from participating.

China

https://www.clinicaltrials.gov/ct2/show/NCT05520892?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=36

Status: Recruiting

Fujian Medical University Union Hospital is conducting a multicenter phase 4 randomized control study to compare the efficacy of two different doses of intravenous immune globulin (IVIG) in the treatment of children with newly diagnosed immune thrombocytopenia, and to reduce related adverse reactions and economic burdens on the premise of ensuring the remission rate. Eligibility is restricted to those who have a platelet count below 20,000 µL and are between 28 days of age through to 14 years of age. You must also have a confirmed diagnosis of ITP and have never received treated for ITP in past.

China

https://www.clinicaltrials.gov/ct2/show/NCT05520892?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=39#contacts

Recruiting

This study conducted by Swedish Orphan Biovitrum (Sobi) is a phase 4 multicenter, observational, clinical trial for adults with ITP over the age of 18 years. The goal of the study is to describe the real-world effectiveness of Doptelet and assess the patterns of drug utilization to add to the knowledge base in routine medical practice. Both retrospective and prospective data will be collected.

The main part of the study will be prospective collecting data on usage, effectiveness, safety, and on patient- and clinician-reported outcomes using Doptelet. The retrospective part will consist of collecting information on previous treatments, reasons for treatment switches, healthcare resource use and Doptelet treatment prior to enrollment. The retrospective data collection will be based on the information available in the patient's medical records. Participants will be excluded from participating if they have secondary ITP or have taken an ’investigational medicinal product’ within three months of enrolled into the 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, Greece, Italy, Slovenia, Spain, and USA

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

Status: Recruiting

Novartis Pharmaceuticals is conducting a phase 3 multicenter study designed to assess the efficacy and safety of two different doses of ianalumab, a monoclonal antibody, versus placebo with the addition of eltrombopag (Promacta/Revolade), a thrombopoietin receptor agonist, in adults with primary ITP who are over the age of 18 years and have a platelet count less than 30,000, and who have failed previous first-line treatment with corticosteroids with/without IVIG.

After completion of the screening period to ensure eligibility, participants will enter the randomized treatment period where they will receive ianalumab plus eltrombopag, or they will receive the placebo drug with eltrombopag, followed by a period where the eltrombopag will be tapered. Afterwards, all participants will enter a follow-up period where they will be monitored for efficacy and/or safety.

Exclusions include participants who received second-line ITP treatments (other than steroid therapy± IVIG) including splenectomy, and participants with current or history of life-threatening bleeding. Other protocol-defined inclusion/exclusion criteria may apply.

Czechia, Germany, Hungary, Republic of Korea, Japan, Malaysia, Norway, Singapore, Spain, and United Kingdom

https://www.clinicaltrials.gov/ct2/show/NCT05653219?recrs=a&cond=immune+thrombocytopenia&draw=3&rank=9

Actively Recruiting

This study conducted by Swedish Orphan Biovitrum (Sobi) is a phase 4 multicenter, observational, clinical trial for adults with ITP over the age of 18 years. The goal of the study is to describe the real-world effectiveness of Doptelet and assess the patterns of drug utilization to add to the knowledge base in routine medical practice. Both retrospective and prospective data will be collected.

The main part of the study will be prospective collecting data on usage, effectiveness, safety, and on patient- and clinician-reported outcomes using Doptelet. The retrospective part will consist of collecting information on previous treatments, reasons for treatment switches, healthcare resource use and Doptelet treatment prior to enrollment. The retrospective data collection will be based on the information available in the patient's medical records. Participants will be excluded from participating if they have secondary ITP or have taken an ’investigational medicinal product’ within three months of enrolled into the 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

Novartis Pharmaceuticals is conducting a Phase 3 study involving multiple centers designed to assess the efficacy and safety of using two different doses of ianalumab, a monoclonal antibody. Eligibility is restricted to adults with primary ITP who are over the age of 18 years, and who had a platelet count less than 30,000 µL before starting first line therapy options and meet criteria for treatment.

After completion of the screening period to ensure eligibility, participants will be given (randomly) either ianalumab or placebo as a healthy control to compare responses. All participants will be monitored for efficacy and/or safety.

Participants with current serious bleeding will be excluded, in addition to those who have had a previous splenectomy or ITP treatment within 28 days prior to the start of the study post-screening. Participants will also be excluded if they have used rituximab in past and have an absolute neutrophil count below 1.0 G/L at randomization. Anyone with concurrent coagulation disorders and/or receiving anti-platelet or anticoagulant medication (with an exemption of low dose of acetylsalicylic acid) will also be excluded from participating in this study.

 Australia, Belgium, Czechia, France, Germany, Hong Kong, Hungary, Japan, Malaysia, Norway, Singapore, Spain, Romania, United Kingdom, and United States.

https://www.clinicaltrials.gov/ct2/show/NCT05653349?recrs=a&cond=immune+thrombocytopenia&draw=2&rank=15

Actively 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

This observational study led by researchers at Sohag University in Egypt is for adults with primary ITP between the ages of 18-90 years. The purpose of this research is to assess the role of mircoRNAs (miRNAs) and cytokines in the plasma of ITP patients; miRNAs are small RNAs (genetic material) that regulate gene expression. Participants would be required to provide a plasma sample for analysis. Participants will be excluded if they have secondary ITP, or have had a recent infection, or a recent vaccine.

Egypt

https://www.clinicaltrials.gov/ct2/show/NCT05371743?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=22

Status: Recruiting

This study compares the effectiveness and adverse event patterns of various ITP treatments. Eligible participants are those 18 years or older with a confirmed diagnosis of primary ITP with an initial platelet count less than 30,000/µl or with bleeding events. Participants with secondary ITP (such as low platelet counts caused by another autoimmune disease or infection); heart, kidney, or liver disease; or received NSAIDs within 1 month of trial initiation are ineligible for this study.

Upon diagnosis of primary ITP, all participants will be given high-dose dexamethasone. Afterwards, the participant will be randomly assigned to one of these groups: (1) continuation of high-dose dexamethasone, (2) prednisolone + azathioprine, (3) rituximab, (4) eltrombopag, or (5) romiplostim). Participants will be assessed weekly to adjust treatment doses. They will also be assessed at baseline, at end of treatment (6 months), and 6 months after treatment discontinuation for the following: increase in platelet count, bleeding, and adverse events.

Egypt

https://www.clinicaltrials.gov/study/NCT05861297?cond=immune%20thrombocytopenia&aggFilters=status:rec&viewType=Table&limit=100&page=1&rank=21

Actively Recruiting

University Hospital in Toulouse France is conducting a study to assess how platelet function is impacted in adults with ITP over the age of 18 years who have heart disease using 75 mg of aspirin. The goal is also to assess how the use of aspirin among adults with ITP impacts the incidence of arterial thrombosis. Adults with ITP who are using other antiplatelet or anticoagulant drugs will be excluding from participating.

France

https://www.clinicaltrials.gov/ct2/show/NCT04912505?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=45

Actively Recruiting

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

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

The Assistance Publique - Hôpitaux de Paris in conducting a study looking at the benefits of using IgIV in combination with prednisone compared to using dexamethasone as a stand-alone treatment for adults with ITP, in any disease phase (newly diagnosed, persistent, or chronic). Adults between the ages of 18-80 years with a confirmed diagnosis of ITP, a platelet count less than 20,000 µL, and a history of any type of bleeding (skin or mucosal bleeding) are eligible to participant.

France

https://www.clinicaltrials.gov/ct2/show/NCT04968899?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=31#contacts

Recruiting

Novartis Pharmaceuticals is conducting a Phase 3 study involving multiple centers designed to assess the efficacy and safety of using two different doses of ianalumab, a monoclonal antibody. Eligibility is restricted to adults with primary ITP who are over the age of 18 years, and who had a platelet count less than 30,000 µL before starting first line therapy options and meet criteria for treatment.

After completion of the screening period to ensure eligibility, participants will be given (randomly) either ianalumab or placebo as a healthy control to compare responses. All participants will be monitored for efficacy and/or safety.

Participants with current serious bleeding will be excluded, in addition to those who have had a previous splenectomy or ITP treatment within 28 days prior to the start of the study post-screening. Participants will also be excluded if they have used rituximab in past and have an absolute neutrophil count below 1.0 G/L at randomization. Anyone with concurrent coagulation disorders and/or receiving anti-platelet or anticoagulant medication (with an exemption of low dose of acetylsalicylic acid) will also be excluded from participating in this study.

 Australia, Belgium, Czechia, France, Germany, Hong Kong, Hungary, Japan, Malaysia, Norway, Singapore, Spain, Romania, United Kingdom, and United States.

https://www.clinicaltrials.gov/ct2/show/NCT05653349?recrs=a&cond=immune+thrombocytopenia&draw=2&rank=15

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

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

Novartis Pharmaceuticals is conducting a phase 3 multicenter study designed to assess the efficacy and safety of two different doses of ianalumab, a monoclonal antibody, versus placebo with the addition of eltrombopag (Promacta/Revolade), a thrombopoietin receptor agonist, in adults with primary ITP who are over the age of 18 years and have a platelet count less than 30,000, and who have failed previous first-line treatment with corticosteroids with/without IVIG.

After completion of the screening period to ensure eligibility, participants will enter the randomized treatment period where they will receive ianalumab plus eltrombopag, or they will receive the placebo drug with eltrombopag, followed by a period where the eltrombopag will be tapered. Afterwards, all participants will enter a follow-up period where they will be monitored for efficacy and/or safety.

Exclusions include participants who received second-line ITP treatments (other than steroid therapy± IVIG) including splenectomy, and participants with current or history of life-threatening bleeding. Other protocol-defined inclusion/exclusion criteria may apply.

Czechia, Germany, Hungary, Republic of Korea, Japan, Malaysia, Norway, Singapore, Spain, and United Kingdom

https://www.clinicaltrials.gov/ct2/show/NCT05653219?recrs=a&cond=immune+thrombocytopenia&draw=3&rank=9

Actively 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

This study conducted by Swedish Orphan Biovitrum (Sobi) is a phase 4 multicenter, observational, clinical trial for adults with ITP over the age of 18 years. The goal of the study is to describe the real-world effectiveness of Doptelet and assess the patterns of drug utilization to add to the knowledge base in routine medical practice. Both retrospective and prospective data will be collected.

The main part of the study will be prospective collecting data on usage, effectiveness, safety, and on patient- and clinician-reported outcomes using Doptelet. The retrospective part will consist of collecting information on previous treatments, reasons for treatment switches, healthcare resource use and Doptelet treatment prior to enrollment. The retrospective data collection will be based on the information available in the patient's medical records. Participants will be excluded from participating if they have secondary ITP or have taken an ’investigational medicinal product’ within three months of enrolled into the 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

Novartis Pharmaceuticals is conducting this open-label, multicenter, phase 2 basket study to assess the effectiveness and safety of Iptacopan (LNP023) in adults with persistent or chronic ITP, who are at least 18 years of age. Iptacopan is an orally administered therapy that targets the alternative complement pathway of the immune system. The drug will also be given to participants who have cold agglutinin disease, a different condition than ITP, to compare how well the drug does in both patient groups. Participants must be vaccinated against Neisseria meningitidis (meningitis) and Streptococcus pneumoniae (pneumonia) infections in addition to Haemophilus influenzae (the flu) infection. Participants will be excluded if they have secondary ITP or have used any therapies for their ITP apart from low-dose corticosteroids or a thrombopoetin receptor agonist (TPO-RA) at least four weeks before enrolling into the study.

United States, Germany, Italy, Republic of Korea, Spain, United Kingdom

https://www.clinicaltrials.gov/ct2/show/NCT05086744?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=50

Status: 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

Novartis Pharmaceuticals is conducting a Phase 3 study involving multiple centers designed to assess the efficacy and safety of using two different doses of ianalumab, a monoclonal antibody. Eligibility is restricted to adults with primary ITP who are over the age of 18 years, and who had a platelet count less than 30,000 µL before starting first line therapy options and meet criteria for treatment.

After completion of the screening period to ensure eligibility, participants will be given (randomly) either ianalumab or placebo as a healthy control to compare responses. All participants will be monitored for efficacy and/or safety.

Participants with current serious bleeding will be excluded, in addition to those who have had a previous splenectomy or ITP treatment within 28 days prior to the start of the study post-screening. Participants will also be excluded if they have used rituximab in past and have an absolute neutrophil count below 1.0 G/L at randomization. Anyone with concurrent coagulation disorders and/or receiving anti-platelet or anticoagulant medication (with an exemption of low dose of acetylsalicylic acid) will also be excluded from participating in this study.

 Australia, Belgium, Czechia, France, Germany, Hong Kong, Hungary, Japan, Malaysia, Norway, Singapore, Spain, Romania, United Kingdom, and United States.

https://www.clinicaltrials.gov/ct2/show/NCT05653349?recrs=a&cond=immune+thrombocytopenia&draw=2&rank=15

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

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, Greece, Italy, Slovenia, Spain, and USA

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

Status: 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, Greece, Italy, Slovenia, Spain, and USA

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

Status: 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

Novartis Pharmaceuticals is conducting a Phase 3 study involving multiple centers designed to assess the efficacy and safety of using two different doses of ianalumab, a monoclonal antibody. Eligibility is restricted to adults with primary ITP who are over the age of 18 years, and who had a platelet count less than 30,000 µL before starting first line therapy options and meet criteria for treatment.

After completion of the screening period to ensure eligibility, participants will be given (randomly) either ianalumab or placebo as a healthy control to compare responses. All participants will be monitored for efficacy and/or safety.

Participants with current serious bleeding will be excluded, in addition to those who have had a previous splenectomy or ITP treatment within 28 days prior to the start of the study post-screening. Participants will also be excluded if they have used rituximab in past and have an absolute neutrophil count below 1.0 G/L at randomization. Anyone with concurrent coagulation disorders and/or receiving anti-platelet or anticoagulant medication (with an exemption of low dose of acetylsalicylic acid) will also be excluded from participating in this study.

 Australia, Belgium, Czechia, France, Germany, Hong Kong, Hungary, Japan, Malaysia, Norway, Singapore, Spain, Romania, United Kingdom, and United States.

https://www.clinicaltrials.gov/ct2/show/NCT05653349?recrs=a&cond=immune+thrombocytopenia&draw=2&rank=15

Actively recruiting

Novartis Pharmaceuticals is conducting a phase 3 multicenter study designed to assess the efficacy and safety of two different doses of ianalumab, a monoclonal antibody, versus placebo with the addition of eltrombopag (Promacta/Revolade), a thrombopoietin receptor agonist, in adults with primary ITP who are over the age of 18 years and have a platelet count less than 30,000, and who have failed previous first-line treatment with corticosteroids with/without IVIG.

After completion of the screening period to ensure eligibility, participants will enter the randomized treatment period where they will receive ianalumab plus eltrombopag, or they will receive the placebo drug with eltrombopag, followed by a period where the eltrombopag will be tapered. Afterwards, all participants will enter a follow-up period where they will be monitored for efficacy and/or safety.

Exclusions include participants who received second-line ITP treatments (other than steroid therapy± IVIG) including splenectomy, and participants with current or history of life-threatening bleeding. Other protocol-defined inclusion/exclusion criteria may apply.

Czechia, Germany, Hungary, Republic of Korea, Japan, Malaysia, Norway, Singapore, Spain, and United Kingdom

https://www.clinicaltrials.gov/ct2/show/NCT05653219?recrs=a&cond=immune+thrombocytopenia&draw=3&rank=9

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

Novartis Pharmaceuticals is conducting a Phase 3 study involving multiple centers designed to assess the efficacy and safety of using two different doses of ianalumab, a monoclonal antibody. Eligibility is restricted to adults with primary ITP who are over the age of 18 years, and who had a platelet count less than 30,000 µL before starting first line therapy options and meet criteria for treatment.

After completion of the screening period to ensure eligibility, participants will be given (randomly) either ianalumab or placebo as a healthy control to compare responses. All participants will be monitored for efficacy and/or safety.

Participants with current serious bleeding will be excluded, in addition to those who have had a previous splenectomy or ITP treatment within 28 days prior to the start of the study post-screening. Participants will also be excluded if they have used rituximab in past and have an absolute neutrophil count below 1.0 G/L at randomization. Anyone with concurrent coagulation disorders and/or receiving anti-platelet or anticoagulant medication (with an exemption of low dose of acetylsalicylic acid) will also be excluded from participating in this study.

 Australia, Belgium, Czechia, France, Germany, Hong Kong, Hungary, Japan, Malaysia, Norway, Singapore, Spain, Romania, United Kingdom, and United States.

https://www.clinicaltrials.gov/ct2/show/NCT05653349?recrs=a&cond=immune+thrombocytopenia&draw=2&rank=15

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

This study conducted by Swedish Orphan Biovitrum (Sobi) is a phase 4 multicenter, observational, clinical trial for adults with ITP over the age of 18 years. The goal of the study is to describe the real-world effectiveness of Doptelet and assess the patterns of drug utilization to add to the knowledge base in routine medical practice. Both retrospective and prospective data will be collected.

The main part of the study will be prospective collecting data on usage, effectiveness, safety, and on patient- and clinician-reported outcomes using Doptelet. The retrospective part will consist of collecting information on previous treatments, reasons for treatment switches, healthcare resource use and Doptelet treatment prior to enrollment. The retrospective data collection will be based on the information available in the patient's medical records. Participants will be excluded from participating if they have secondary ITP or have taken an ’investigational medicinal product’ within three months of enrolled into the 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

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 study conducted by Swedish Orphan Biovitrum (Sobi) is a phase 4 multicenter, observational, clinical trial for adults with ITP over the age of 18 years. The goal of the study is to describe the real-world effectiveness of Doptelet and assess the patterns of drug utilization to add to the knowledge base in routine medical practice. Both retrospective and prospective data will be collected.

The main part of the study will be prospective collecting data on usage, effectiveness, safety, and on patient- and clinician-reported outcomes using Doptelet. The retrospective part will consist of collecting information on previous treatments, reasons for treatment switches, healthcare resource use and Doptelet treatment prior to enrollment. The retrospective data collection will be based on the information available in the patient's medical records. Participants will be excluded from participating if they have secondary ITP or have taken an ’investigational medicinal product’ within three months of enrolled into the 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

Novartis Pharmaceuticals is conducting this open-label, multicenter, phase 2 basket study to assess the effectiveness and safety of Iptacopan (LNP023) in adults with persistent or chronic ITP, who are at least 18 years of age. Iptacopan is an orally administered therapy that targets the alternative complement pathway of the immune system. The drug will also be given to participants who have cold agglutinin disease, a different condition than ITP, to compare how well the drug does in both patient groups. Participants must be vaccinated against Neisseria meningitidis (meningitis) and Streptococcus pneumoniae (pneumonia) infections in addition to Haemophilus influenzae (the flu) infection. Participants will be excluded if they have secondary ITP or have used any therapies for their ITP apart from low-dose corticosteroids or a thrombopoetin receptor agonist (TPO-RA) at least four weeks before enrolling into the study.

United States, Germany, Italy, Republic of Korea, Spain, United Kingdom

https://www.clinicaltrials.gov/ct2/show/NCT05086744?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=50

Status: Recruiting

The goal of this study is to better understand the diagnosis pattern and treatment options of various autoimmune conditions, including autoimmune hemolytic anemia, immune thrombocytopenia, and chronic idiopathic/autoimmune neutropenia.

This study will examine both traditional and new diagnostic tools (i.e., cytokine assays, bone marrow evaluation, and fecal microbiome). Additionally, the type of treatment administered, its effectiveness, and adverse event rate will be studied. Blood tests will be performed to evaluate possible predictors of outcome. Participants will also be evaluated of their autoimmune condition evolving into myelodysplastic syndromes (a class of disorders caused by poorly formed or dysfunctional blood cells).

Participants 18 years and older with any of the conditions mentioned above who are available for a 3-year follow-up are eligible to enroll in this study.

Italy

https://www.clinicaltrials.gov/study/NCT05931718?cond=immune%20thrombocytopenia&aggFilters=status:rec&viewType=Table&limit=100&page=1&rank=67

Actively 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

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, Greece, Italy, Slovenia, Spain, and USA

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

Status: Recruiting

Novartis Pharmaceuticals is conducting a phase 3 multicenter study designed to assess the efficacy and safety of two different doses of ianalumab, a monoclonal antibody, versus placebo with the addition of eltrombopag (Promacta/Revolade), a thrombopoietin receptor agonist, in adults with primary ITP who are over the age of 18 years and have a platelet count less than 30,000, and who have failed previous first-line treatment with corticosteroids with/without IVIG.

After completion of the screening period to ensure eligibility, participants will enter the randomized treatment period where they will receive ianalumab plus eltrombopag, or they will receive the placebo drug with eltrombopag, followed by a period where the eltrombopag will be tapered. Afterwards, all participants will enter a follow-up period where they will be monitored for efficacy and/or safety.

Exclusions include participants who received second-line ITP treatments (other than steroid therapy± IVIG) including splenectomy, and participants with current or history of life-threatening bleeding. Other protocol-defined inclusion/exclusion criteria may apply.

Czechia, Germany, Hungary, Republic of Korea, Japan, Malaysia, Norway, Singapore, Spain, and United Kingdom

https://www.clinicaltrials.gov/ct2/show/NCT05653219?recrs=a&cond=immune+thrombocytopenia&draw=3&rank=9

Actively 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

Novartis Pharmaceuticals is conducting a Phase 3 study involving multiple centers designed to assess the efficacy and safety of using two different doses of ianalumab, a monoclonal antibody. Eligibility is restricted to adults with primary ITP who are over the age of 18 years, and who had a platelet count less than 30,000 µL before starting first line therapy options and meet criteria for treatment.

After completion of the screening period to ensure eligibility, participants will be given (randomly) either ianalumab or placebo as a healthy control to compare responses. All participants will be monitored for efficacy and/or safety.

Participants with current serious bleeding will be excluded, in addition to those who have had a previous splenectomy or ITP treatment within 28 days prior to the start of the study post-screening. Participants will also be excluded if they have used rituximab in past and have an absolute neutrophil count below 1.0 G/L at randomization. Anyone with concurrent coagulation disorders and/or receiving anti-platelet or anticoagulant medication (with an exemption of low dose of acetylsalicylic acid) will also be excluded from participating in this study.

 Australia, Belgium, Czechia, France, Germany, Hong Kong, Hungary, Japan, Malaysia, Norway, Singapore, Spain, Romania, United Kingdom, and United States.

https://www.clinicaltrials.gov/ct2/show/NCT05653349?recrs=a&cond=immune+thrombocytopenia&draw=2&rank=15

Actively 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

Novartis Pharmaceuticals is conducting a phase 3 multicenter study designed to assess the efficacy and safety of two different doses of ianalumab, a monoclonal antibody, versus placebo with the addition of eltrombopag (Promacta/Revolade), a thrombopoietin receptor agonist, in adults with primary ITP who are over the age of 18 years and have a platelet count less than 30,000, and who have failed previous first-line treatment with corticosteroids with/without IVIG.

After completion of the screening period to ensure eligibility, participants will enter the randomized treatment period where they will receive ianalumab plus eltrombopag, or they will receive the placebo drug with eltrombopag, followed by a period where the eltrombopag will be tapered. Afterwards, all participants will enter a follow-up period where they will be monitored for efficacy and/or safety.

Exclusions include participants who received second-line ITP treatments (other than steroid therapy± IVIG) including splenectomy, and participants with current or history of life-threatening bleeding. Other protocol-defined inclusion/exclusion criteria may apply.

Czechia, Germany, Hungary, Republic of Korea, Japan, Malaysia, Norway, Singapore, Spain, and United Kingdom

https://www.clinicaltrials.gov/ct2/show/NCT05653219?recrs=a&cond=immune+thrombocytopenia&draw=3&rank=9

Actively Recruiting

Novartis Pharmaceuticals is conducting a Phase 3 study involving multiple centers designed to assess the efficacy and safety of using two different doses of ianalumab, a monoclonal antibody. Eligibility is restricted to adults with primary ITP who are over the age of 18 years, and who had a platelet count less than 30,000 µL before starting first line therapy options and meet criteria for treatment.

After completion of the screening period to ensure eligibility, participants will be given (randomly) either ianalumab or placebo as a healthy control to compare responses. All participants will be monitored for efficacy and/or safety.

Participants with current serious bleeding will be excluded, in addition to those who have had a previous splenectomy or ITP treatment within 28 days prior to the start of the study post-screening. Participants will also be excluded if they have used rituximab in past and have an absolute neutrophil count below 1.0 G/L at randomization. Anyone with concurrent coagulation disorders and/or receiving anti-platelet or anticoagulant medication (with an exemption of low dose of acetylsalicylic acid) will also be excluded from participating in this study.

 Australia, Belgium, Czechia, France, Germany, Hong Kong, Hungary, Japan, Malaysia, Norway, Singapore, Spain, Romania, United Kingdom, and United States.

https://www.clinicaltrials.gov/ct2/show/NCT05653349?recrs=a&cond=immune+thrombocytopenia&draw=2&rank=15

Actively 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 study conducted by Swedish Orphan Biovitrum (Sobi) is a phase 4 multicenter, observational, clinical trial for adults with ITP over the age of 18 years. The goal of the study is to describe the real-world effectiveness of Doptelet and assess the patterns of drug utilization to add to the knowledge base in routine medical practice. Both retrospective and prospective data will be collected.

The main part of the study will be prospective collecting data on usage, effectiveness, safety, and on patient- and clinician-reported outcomes using Doptelet. The retrospective part will consist of collecting information on previous treatments, reasons for treatment switches, healthcare resource use and Doptelet treatment prior to enrollment. The retrospective data collection will be based on the information available in the patient's medical records. Participants will be excluded from participating if they have secondary ITP or have taken an ’investigational medicinal product’ within three months of enrolled into the 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

Novartis Pharmaceuticals is conducting a phase 3 multicenter study designed to assess the efficacy and safety of two different doses of ianalumab, a monoclonal antibody, versus placebo with the addition of eltrombopag (Promacta/Revolade), a thrombopoietin receptor agonist, in adults with primary ITP who are over the age of 18 years and have a platelet count less than 30,000, and who have failed previous first-line treatment with corticosteroids with/without IVIG.

After completion of the screening period to ensure eligibility, participants will enter the randomized treatment period where they will receive ianalumab plus eltrombopag, or they will receive the placebo drug with eltrombopag, followed by a period where the eltrombopag will be tapered. Afterwards, all participants will enter a follow-up period where they will be monitored for efficacy and/or safety.

Exclusions include participants who received second-line ITP treatments (other than steroid therapy± IVIG) including splenectomy, and participants with current or history of life-threatening bleeding. Other protocol-defined inclusion/exclusion criteria may apply.

Czechia, Germany, Hungary, Republic of Korea, Japan, Malaysia, Norway, Singapore, Spain, and United Kingdom

https://www.clinicaltrials.gov/ct2/show/NCT05653219?recrs=a&cond=immune+thrombocytopenia&draw=3&rank=9

Actively Recruiting

This study conducted by Swedish Orphan Biovitrum (Sobi) is a phase 4 multicenter, observational, clinical trial for adults with ITP over the age of 18 years. The goal of the study is to describe the real-world effectiveness of Doptelet and assess the patterns of drug utilization to add to the knowledge base in routine medical practice. Both retrospective and prospective data will be collected.

The main part of the study will be prospective collecting data on usage, effectiveness, safety, and on patient- and clinician-reported outcomes using Doptelet. The retrospective part will consist of collecting information on previous treatments, reasons for treatment switches, healthcare resource use and Doptelet treatment prior to enrollment. The retrospective data collection will be based on the information available in the patient's medical records. Participants will be excluded from participating if they have secondary ITP or have taken an ’investigational medicinal product’ within three months of enrolled into the 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

Novartis Pharmaceuticals is conducting a Phase 3 study involving multiple centers designed to assess the efficacy and safety of using two different doses of ianalumab, a monoclonal antibody. Eligibility is restricted to adults with primary ITP who are over the age of 18 years, and who had a platelet count less than 30,000 µL before starting first line therapy options and meet criteria for treatment.

After completion of the screening period to ensure eligibility, participants will be given (randomly) either ianalumab or placebo as a healthy control to compare responses. All participants will be monitored for efficacy and/or safety.

Participants with current serious bleeding will be excluded, in addition to those who have had a previous splenectomy or ITP treatment within 28 days prior to the start of the study post-screening. Participants will also be excluded if they have used rituximab in past and have an absolute neutrophil count below 1.0 G/L at randomization. Anyone with concurrent coagulation disorders and/or receiving anti-platelet or anticoagulant medication (with an exemption of low dose of acetylsalicylic acid) will also be excluded from participating in this study.

 Australia, Belgium, Czechia, France, Germany, Hong Kong, Hungary, Japan, Malaysia, Norway, Singapore, Spain, Romania, United Kingdom, and United States.

https://www.clinicaltrials.gov/ct2/show/NCT05653349?recrs=a&cond=immune+thrombocytopenia&draw=2&rank=15

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

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

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

Novartis Pharmaceuticals is conducting a phase 3 multicenter study designed to assess the efficacy and safety of two different doses of ianalumab, a monoclonal antibody, versus placebo with the addition of eltrombopag (Promacta/Revolade), a thrombopoietin receptor agonist, in adults with primary ITP who are over the age of 18 years and have a platelet count less than 30,000, and who have failed previous first-line treatment with corticosteroids with/without IVIG.

After completion of the screening period to ensure eligibility, participants will enter the randomized treatment period where they will receive ianalumab plus eltrombopag, or they will receive the placebo drug with eltrombopag, followed by a period where the eltrombopag will be tapered. Afterwards, all participants will enter a follow-up period where they will be monitored for efficacy and/or safety.

Exclusions include participants who received second-line ITP treatments (other than steroid therapy± IVIG) including splenectomy, and participants with current or history of life-threatening bleeding. Other protocol-defined inclusion/exclusion criteria may apply.

Czechia, Germany, Hungary, Republic of Korea, Japan, Malaysia, Norway, Singapore, Spain, and United Kingdom

https://www.clinicaltrials.gov/ct2/show/NCT05653219?recrs=a&cond=immune+thrombocytopenia&draw=3&rank=9

Actively Recruiting

Novartis Pharmaceuticals is conducting a Phase 3 study involving multiple centers designed to assess the efficacy and safety of using two different doses of ianalumab, a monoclonal antibody. Eligibility is restricted to adults with primary ITP who are over the age of 18 years, and who had a platelet count less than 30,000 µL before starting first line therapy options and meet criteria for treatment.

After completion of the screening period to ensure eligibility, participants will be given (randomly) either ianalumab or placebo as a healthy control to compare responses. All participants will be monitored for efficacy and/or safety.

Participants with current serious bleeding will be excluded, in addition to those who have had a previous splenectomy or ITP treatment within 28 days prior to the start of the study post-screening. Participants will also be excluded if they have used rituximab in past and have an absolute neutrophil count below 1.0 G/L at randomization. Anyone with concurrent coagulation disorders and/or receiving anti-platelet or anticoagulant medication (with an exemption of low dose of acetylsalicylic acid) will also be excluded from participating in this study.

 Australia, Belgium, Czechia, France, Germany, Hong Kong, Hungary, Japan, Malaysia, Norway, Singapore, Spain, Romania, United Kingdom, and United States.

https://www.clinicaltrials.gov/ct2/show/NCT05653349?recrs=a&cond=immune+thrombocytopenia&draw=2&rank=15

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 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

Novartis Pharmaceuticals is conducting a phase 3 multicenter study designed to assess the efficacy and safety of two different doses of ianalumab, a monoclonal antibody, versus placebo with the addition of eltrombopag (Promacta/Revolade), a thrombopoietin receptor agonist, in adults with primary ITP who are over the age of 18 years and have a platelet count less than 30,000, and who have failed previous first-line treatment with corticosteroids with/without IVIG.

After completion of the screening period to ensure eligibility, participants will enter the randomized treatment period where they will receive ianalumab plus eltrombopag, or they will receive the placebo drug with eltrombopag, followed by a period where the eltrombopag will be tapered. Afterwards, all participants will enter a follow-up period where they will be monitored for efficacy and/or safety.

Exclusions include participants who received second-line ITP treatments (other than steroid therapy± IVIG) including splenectomy, and participants with current or history of life-threatening bleeding. Other protocol-defined inclusion/exclusion criteria may apply.

Czechia, Germany, Hungary, Republic of Korea, Japan, Malaysia, Norway, Singapore, Spain, and United Kingdom

https://www.clinicaltrials.gov/ct2/show/NCT05653219?recrs=a&cond=immune+thrombocytopenia&draw=3&rank=9

Actively Recruiting

Novartis Pharmaceuticals is conducting a Phase 3 study involving multiple centers designed to assess the efficacy and safety of using two different doses of ianalumab, a monoclonal antibody. Eligibility is restricted to adults with primary ITP who are over the age of 18 years, and who had a platelet count less than 30,000 µL before starting first line therapy options and meet criteria for treatment.

After completion of the screening period to ensure eligibility, participants will be given (randomly) either ianalumab or placebo as a healthy control to compare responses. All participants will be monitored for efficacy and/or safety.

Participants with current serious bleeding will be excluded, in addition to those who have had a previous splenectomy or ITP treatment within 28 days prior to the start of the study post-screening. Participants will also be excluded if they have used rituximab in past and have an absolute neutrophil count below 1.0 G/L at randomization. Anyone with concurrent coagulation disorders and/or receiving anti-platelet or anticoagulant medication (with an exemption of low dose of acetylsalicylic acid) will also be excluded from participating in this study.

 Australia, Belgium, Czechia, France, Germany, Hong Kong, Hungary, Japan, Malaysia, Norway, Singapore, Spain, Romania, United Kingdom, and United States.

https://www.clinicaltrials.gov/ct2/show/NCT05653349?recrs=a&cond=immune+thrombocytopenia&draw=2&rank=15

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

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, Greece, Italy, Slovenia, Spain, and USA

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

Status: Recruiting

Novartis Pharmaceuticals is conducting this open-label, multicenter, phase 2 basket study to assess the effectiveness and safety of Iptacopan (LNP023) in adults with persistent or chronic ITP, who are at least 18 years of age. Iptacopan is an orally administered therapy that targets the alternative complement pathway of the immune system. The drug will also be given to participants who have cold agglutinin disease, a different condition than ITP, to compare how well the drug does in both patient groups. Participants must be vaccinated against Neisseria meningitidis (meningitis) and Streptococcus pneumoniae (pneumonia) infections in addition to Haemophilus influenzae (the flu) infection. Participants will be excluded if they have secondary ITP or have used any therapies for their ITP apart from low-dose corticosteroids or a thrombopoetin receptor agonist (TPO-RA) at least four weeks before enrolling into the study.

United States, Germany, Italy, Republic of Korea, Spain, United Kingdom

https://www.clinicaltrials.gov/ct2/show/NCT05086744?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=50

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

Novartis Pharmaceuticals is conducting a phase 3 multicenter study designed to assess the efficacy and safety of two different doses of ianalumab, a monoclonal antibody, versus placebo with the addition of eltrombopag (Promacta/Revolade), a thrombopoietin receptor agonist, in adults with primary ITP who are over the age of 18 years and have a platelet count less than 30,000, and who have failed previous first-line treatment with corticosteroids with/without IVIG.

After completion of the screening period to ensure eligibility, participants will enter the randomized treatment period where they will receive ianalumab plus eltrombopag, or they will receive the placebo drug with eltrombopag, followed by a period where the eltrombopag will be tapered. Afterwards, all participants will enter a follow-up period where they will be monitored for efficacy and/or safety.

Exclusions include participants who received second-line ITP treatments (other than steroid therapy± IVIG) including splenectomy, and participants with current or history of life-threatening bleeding. Other protocol-defined inclusion/exclusion criteria may apply.

Czechia, Germany, Hungary, Republic of Korea, Japan, Malaysia, Norway, Singapore, Spain, and United Kingdom

https://www.clinicaltrials.gov/ct2/show/NCT05653219?recrs=a&cond=immune+thrombocytopenia&draw=3&rank=9

Actively Recruiting

This study conducted by Swedish Orphan Biovitrum (Sobi) is a phase 4 multicenter, observational, clinical trial for adults with ITP over the age of 18 years. The goal of the study is to describe the real-world effectiveness of Doptelet and assess the patterns of drug utilization to add to the knowledge base in routine medical practice. Both retrospective and prospective data will be collected.

The main part of the study will be prospective collecting data on usage, effectiveness, safety, and on patient- and clinician-reported outcomes using Doptelet. The retrospective part will consist of collecting information on previous treatments, reasons for treatment switches, healthcare resource use and Doptelet treatment prior to enrollment. The retrospective data collection will be based on the information available in the patient's medical records. Participants will be excluded from participating if they have secondary ITP or have taken an ’investigational medicinal product’ within three months of enrolled into the 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

Novartis Pharmaceuticals is conducting this open-label, multicenter, phase 2 basket study to assess the effectiveness and safety of Iptacopan (LNP023) in adults with persistent or chronic ITP, who are at least 18 years of age. Iptacopan is an orally administered therapy that targets the alternative complement pathway of the immune system. The drug will also be given to participants who have cold agglutinin disease, a different condition than ITP, to compare how well the drug does in both patient groups. Participants must be vaccinated against Neisseria meningitidis (meningitis) and Streptococcus pneumoniae (pneumonia) infections in addition to Haemophilus influenzae (the flu) infection. Participants will be excluded if they have secondary ITP or have used any therapies for their ITP apart from low-dose corticosteroids or a thrombopoetin receptor agonist (TPO-RA) at least four weeks before enrolling into the study.

United States, Germany, Italy, Republic of Korea, Spain, United Kingdom

https://www.clinicaltrials.gov/ct2/show/NCT05086744?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=50

Status: 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

The Fundación Pública Andaluza para la gestión de la Investigación en Sevilla is conducting a Phase 3 clinical trial to evaluate the benefit of using romiplostim plus dexamethasone versus dexamethasone alone in the treatment of primary immune thrombocytopenia. Study inclusion is open to those with sustained response to any ITP treatment and without World Health Organization grade 2 or higher bleeding, after six months from stopping treatment. The maximum time on treatment with romiplostim will be one year. Then, patients will be followed up for six additional months after stopping romiplostim. Inclusion is limited to adults over the age of 18 years, who have newly diagnosed ITP and have platelet counts less than 30,000 µL with bleeding symptoms.

Spain

https://www.clinicaltrials.gov/ct2/show/NCT05325593?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=14#contacts

Recruiting

Novartis Pharmaceuticals is conducting a Phase 3 study involving multiple centers designed to assess the efficacy and safety of using two different doses of ianalumab, a monoclonal antibody. Eligibility is restricted to adults with primary ITP who are over the age of 18 years, and who had a platelet count less than 30,000 µL before starting first line therapy options and meet criteria for treatment.

After completion of the screening period to ensure eligibility, participants will be given (randomly) either ianalumab or placebo as a healthy control to compare responses. All participants will be monitored for efficacy and/or safety.

Participants with current serious bleeding will be excluded, in addition to those who have had a previous splenectomy or ITP treatment within 28 days prior to the start of the study post-screening. Participants will also be excluded if they have used rituximab in past and have an absolute neutrophil count below 1.0 G/L at randomization. Anyone with concurrent coagulation disorders and/or receiving anti-platelet or anticoagulant medication (with an exemption of low dose of acetylsalicylic acid) will also be excluded from participating in this study.

 Australia, Belgium, Czechia, France, Germany, Hong Kong, Hungary, Japan, Malaysia, Norway, Singapore, Spain, Romania, United Kingdom, and United States.

https://www.clinicaltrials.gov/ct2/show/NCT05653349?recrs=a&cond=immune+thrombocytopenia&draw=2&rank=15

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

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, Greece, Italy, Slovenia, Spain, and USA

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

Status: Recruiting

This study conducted by Swedish Orphan Biovitrum (Sobi) is a phase 4 multicenter, observational, clinical trial for adults with ITP over the age of 18 years. The goal of the study is to describe the real-world effectiveness of Doptelet and assess the patterns of drug utilization to add to the knowledge base in routine medical practice. Both retrospective and prospective data will be collected.

The main part of the study will be prospective collecting data on usage, effectiveness, safety, and on patient- and clinician-reported outcomes using Doptelet. The retrospective part will consist of collecting information on previous treatments, reasons for treatment switches, healthcare resource use and Doptelet treatment prior to enrollment. The retrospective data collection will be based on the information available in the patient's medical records. Participants will be excluded from participating if they have secondary ITP or have taken an ’investigational medicinal product’ within three months of enrolled into the 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

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 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

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

Novartis Pharmaceuticals is conducting a phase 3 multicenter study designed to assess the efficacy and safety of two different doses of ianalumab, a monoclonal antibody, versus placebo with the addition of eltrombopag (Promacta/Revolade), a thrombopoietin receptor agonist, in adults with primary ITP who are over the age of 18 years and have a platelet count less than 30,000, and who have failed previous first-line treatment with corticosteroids with/without IVIG.

After completion of the screening period to ensure eligibility, participants will enter the randomized treatment period where they will receive ianalumab plus eltrombopag, or they will receive the placebo drug with eltrombopag, followed by a period where the eltrombopag will be tapered. Afterwards, all participants will enter a follow-up period where they will be monitored for efficacy and/or safety.

Exclusions include participants who received second-line ITP treatments (other than steroid therapy± IVIG) including splenectomy, and participants with current or history of life-threatening bleeding. Other protocol-defined inclusion/exclusion criteria may apply.

Czechia, Germany, Hungary, Republic of Korea, Japan, Malaysia, Norway, Singapore, Spain, and United Kingdom

https://www.clinicaltrials.gov/ct2/show/NCT05653219?recrs=a&cond=immune+thrombocytopenia&draw=3&rank=9

Actively Recruiting

This study conducted by Swedish Orphan Biovitrum (Sobi) is a phase 4 multicenter, observational, clinical trial for adults with ITP over the age of 18 years. The goal of the study is to describe the real-world effectiveness of Doptelet and assess the patterns of drug utilization to add to the knowledge base in routine medical practice. Both retrospective and prospective data will be collected.

The main part of the study will be prospective collecting data on usage, effectiveness, safety, and on patient- and clinician-reported outcomes using Doptelet. The retrospective part will consist of collecting information on previous treatments, reasons for treatment switches, healthcare resource use and Doptelet treatment prior to enrollment. The retrospective data collection will be based on the information available in the patient's medical records. Participants will be excluded from participating if they have secondary ITP or have taken an ’investigational medicinal product’ within three months of enrolled into the 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

Novartis Pharmaceuticals is conducting this open-label, multicenter, phase 2 basket study to assess the effectiveness and safety of Iptacopan (LNP023) in adults with persistent or chronic ITP, who are at least 18 years of age. Iptacopan is an orally administered therapy that targets the alternative complement pathway of the immune system. The drug will also be given to participants who have cold agglutinin disease, a different condition than ITP, to compare how well the drug does in both patient groups. Participants must be vaccinated against Neisseria meningitidis (meningitis) and Streptococcus pneumoniae (pneumonia) infections in addition to Haemophilus influenzae (the flu) infection. Participants will be excluded if they have secondary ITP or have used any therapies for their ITP apart from low-dose corticosteroids or a thrombopoetin receptor agonist (TPO-RA) at least four weeks before enrolling into the study.

United States, Germany, Italy, Republic of Korea, Spain, United Kingdom

https://www.clinicaltrials.gov/ct2/show/NCT05086744?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=50

Status: Recruiting

Novartis Pharmaceuticals is conducting a Phase 3 study involving multiple centers designed to assess the efficacy and safety of using two different doses of ianalumab, a monoclonal antibody. Eligibility is restricted to adults with primary ITP who are over the age of 18 years, and who had a platelet count less than 30,000 µL before starting first line therapy options and meet criteria for treatment.

After completion of the screening period to ensure eligibility, participants will be given (randomly) either ianalumab or placebo as a healthy control to compare responses. All participants will be monitored for efficacy and/or safety.

Participants with current serious bleeding will be excluded, in addition to those who have had a previous splenectomy or ITP treatment within 28 days prior to the start of the study post-screening. Participants will also be excluded if they have used rituximab in past and have an absolute neutrophil count below 1.0 G/L at randomization. Anyone with concurrent coagulation disorders and/or receiving anti-platelet or anticoagulant medication (with an exemption of low dose of acetylsalicylic acid) will also be excluded from participating in this study.

 Australia, Belgium, Czechia, France, Germany, Hong Kong, Hungary, Japan, Malaysia, Norway, Singapore, Spain, Romania, United Kingdom, and United States.

https://www.clinicaltrials.gov/ct2/show/NCT05653349?recrs=a&cond=immune+thrombocytopenia&draw=2&rank=15

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

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

Alpine Immune Sciences is conducting a Phase 1b Open-Label Study to Assess the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of a new treatment for immune thrombocytopenia (and other autoimmune cytopenias) called Povetacicept (also known as APLN-303). This study is open to adults with persistent or chronic ITP. Povetacicept is a is a dual B cell cytokine antagonist engineered to inhibit BAFF (BLyS) and APRIL which are clinically validated cytokine pathways that play a key role in B cell development, differentiation, and survival. The drug will be provided to participants every 4 weeks for 6 months, with the possibility of participating in a 6-month study treatment extension period. Adult participants must have shown failure to at least two previous treatments for ITP, including exposure to a TPO-RA, unless otherwise contraindicated or unavailable to them. Participants must also have a documented platelet count of less than 30,000 µL.

Australia, Canada, USA

https://www.clinicaltrials.gov/study/NCT05757570?term=%20Povetacicept%20&rank=2

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

Novartis Pharmaceuticals is conducting this open-label, multicenter, phase 2 basket study to assess the effectiveness and safety of Iptacopan (LNP023) in adults with persistent or chronic ITP, who are at least 18 years of age. Iptacopan is an orally administered therapy that targets the alternative complement pathway of the immune system. The drug will also be given to participants who have cold agglutinin disease, a different condition than ITP, to compare how well the drug does in both patient groups. Participants must be vaccinated against Neisseria meningitidis (meningitis) and Streptococcus pneumoniae (pneumonia) infections in addition to Haemophilus influenzae (the flu) infection. Participants will be excluded if they have secondary ITP or have used any therapies for their ITP apart from low-dose corticosteroids or a thrombopoetin receptor agonist (TPO-RA) at least four weeks before enrolling into the study.

United States, Germany, Italy, Republic of Korea, Spain, United Kingdom

https://www.clinicaltrials.gov/ct2/show/NCT05086744?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=50

Status: 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

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

Novartis Pharmaceuticals is conducting a Phase 3 study involving multiple centers designed to assess the efficacy and safety of using two different doses of ianalumab, a monoclonal antibody. Eligibility is restricted to adults with primary ITP who are over the age of 18 years, and who had a platelet count less than 30,000 µL before starting first line therapy options and meet criteria for treatment.

After completion of the screening period to ensure eligibility, participants will be given (randomly) either ianalumab or placebo as a healthy control to compare responses. All participants will be monitored for efficacy and/or safety.

Participants with current serious bleeding will be excluded, in addition to those who have had a previous splenectomy or ITP treatment within 28 days prior to the start of the study post-screening. Participants will also be excluded if they have used rituximab in past and have an absolute neutrophil count below 1.0 G/L at randomization. Anyone with concurrent coagulation disorders and/or receiving anti-platelet or anticoagulant medication (with an exemption of low dose of acetylsalicylic acid) will also be excluded from participating in this study.

 Australia, Belgium, Czechia, France, Germany, Hong Kong, Hungary, Japan, Malaysia, Norway, Singapore, Spain, Romania, United Kingdom, and United States.

https://www.clinicaltrials.gov/ct2/show/NCT05653349?recrs=a&cond=immune+thrombocytopenia&draw=2&rank=15

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

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, Greece, Italy, Slovenia, Spain, and USA

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

Status: 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