Clinical Trials
IMPORTANT!
Clinical trials listed for ITP are a starting point for discussion with a health care professional. Be sure you understand all the implications of the proposed treatment and read the informed consent very carefully before enrolling in a clinical trial.
Breakdown for each trial:
• Name of trial
• Short summary
• Countries recruiting
• Link to trial page on clinicaltrials.gov
• Trial Status
List of Clinical Trials
In alphabetical order by Country
Argentina - Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP) (LUNA3)
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, Australia, France, Poland, South Korea, Spain, USA
https://clinicaltrials.gov/ct2/show/NCT04562766
Recruiting
Australia - A Study of PRN1008 in Adult Patients With Immune Thrombocytopenic Purpura (ITP)
Principia Biopharma is conducting a study of their oral drug PRN1008 in patients with ITP who are refractory or relapsed with no available and approved therapeutic options and have a platelet count <30,000/μL on two counts no sooner than 7 days apart in the 15 days before treatment begins. Preclinical findings show that PRN1008 decreases the activity of B cells and other inflammatory-causing immune cells, and in a mouse model of ITP show that PRN1008 can reduce platelet loss.
https://clinicaltrials.gov/ct2/show/NCT03395210?term=PRN1008&rank=1
Recruiting
Australia - Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP) (LUNA3)
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, Australia, France, Poland, South Korea, Spain, USA
https://clinicaltrials.gov/ct2/show/NCT04562766
Recruiting
Australia - The International ITP Registry
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.
Recruiting
Austria - A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ADVANCE)
Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
USA, Austria, Belgium, Bulgaria, Czech Republic, France, Germany, Georgia, Hungary, Italy, Japan, Netherlands, Poland, Russian Federation, Spain, Turkey, Ukraine, and the United Kingdom.
Recruiting
Austria - A study to evaluate the efficacy, safety, and tolerability of Rozanolixizumab in adult study participants with persistent or chronic primary immune thrombocytopenia (ITP) (myOpportunITy2)
UCB Biopharma SRL is conducting a phase three, multicenter, double-blind, randomized, placebo-controlled study to assess the efficacy, safety, and tolerability of Rozanolixizumab, a humanized monoclonal antibody that binds to the human FcRn receptor, in adults (18 years of age or older) with persistent or chronic primary ITP. The purpose is also to demonstrate the clinical efficacy of Rozanolixizumab as a maintenance therapy. Rozanolixizumab is administered through a subcutaneous injection. Participants must have tried, and responded to, at least one previous ITP-related medication. Participants must also be able to receive immunosuppressive treatments.
Austria, Belgium, Bulgaria, Canada, China, Czechia, France, Germany, Italy, Poland, Russian Federation, Spain, Taiwan, Ukraine, UK, and USA.
https://clinicaltrials.gov/ct2/show/NCT04224688?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=5
Recruiting
Austria - Long-term Study to Assess the Safety and Efficacy of Efgartimod in Adult Patients with Primary Immune Thrombocytopenia (ITP) (ADVANCE+)
Argenx is conducting this open-label (no placebo) multicenter phase three trial for efgartimod (ARGX-113) in adult ITP patients with primary ITP. Patients must be over the age of 18 years. Administration of efgartimod through IV injection, a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
Belgium, Hungary, Italy, Japan, The Netherlands, Russian Federation, Spain, Ukraine, USA
Status: Recruiting
Belgium - A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ADVANCE)
Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
USA, Austria, Belgium, Bulgaria, Czech Republic, France, Germany, Georgia, Hungary, Italy, Japan, Netherlands, Poland, Russian Federation, Spain, Turkey, Ukraine, and the United Kingdom.
Recruiting
Belgium - A study to evaluate the efficacy, safety, and tolerability of Rozanolixizumab in adult study participants with persistent or chronic primary immune thrombocytopenia (ITP) (myOpportunITy1)
UCB Biopharma SRL is conducting a phase three, multicenter, double-blind, randomized, placebo-controlled study to assess the efficacy, safety, and tolerability of Rozanolixizumab, a humanized monoclonal antibody that binds to the human FcRn receptor, in adults (18 years of age or older) with persistent or chronic primary ITP. The purpose is also to demonstrate the clinical efficacy of Rozanolixizumab as a maintenance therapy. Rozanolixizumab is administered through a subcutaneous injection. Participants must have tried, and responded to, at least one previous ITP-related medication.
Belgium, Bulgaria, France, Georgia, Japan, Moldova, Poland, Russia, Spain, Ukraine, and USA
Currently recruiting
Belgium - A study to evaluate the efficacy, safety, and tolerability of Rozanolixizumab in adult study participants with persistent or chronic primary immune thrombocytopenia (ITP) (myOpportunITy2)
UCB Biopharma SRL is conducting a phase three, multicenter, double-blind, randomized, placebo-controlled study to assess the efficacy, safety, and tolerability of Rozanolixizumab, a humanized monoclonal antibody that binds to the human FcRn receptor, in adults (18 years of age or older) with persistent or chronic primary ITP. The purpose is also to demonstrate the clinical efficacy of Rozanolixizumab as a maintenance therapy. Rozanolixizumab is administered through a subcutaneous injection. Participants must have tried, and responded to, at least one previous ITP-related medication. Participants must also be able to receive immunosuppressive treatments.
Austria, Belgium, Bulgaria, Canada, China, Czechia, France, Germany, Italy, Poland, Russian Federation, Spain, Taiwan, Ukraine, UK, and USA.
https://clinicaltrials.gov/ct2/show/NCT04224688?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=5
Recruiting
Belgium - Long-term Study to Assess the Safety and Efficacy of Efgartimod in Adult Patients with Primary Immune Thrombocytopenia (ITP) (ADVANCE+)
Argenx is conducting this open-label (no placebo) multicenter phase three trial for efgartimod (ARGX-113) in adult ITP patients with primary ITP. Patients must be over the age of 18 years. Administration of efgartimod through IV injection, a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
Belgium, Hungary, Italy, Japan, The Netherlands, Russian Federation, Spain, Ukraine, USA
Status: Recruiting
Belgium - Safety and tolerability of M254 in healthy volunteers and immune thrombocytopenia patients
This is an open-label two phase study through Momenta Pharmaceuticals, Inc., to look at the safety, tolerability, pharmacodynamics/pharmacokinetics of M254 in healthy patients compared to patients with persistent or chronic immune thrombocytopenia (ITP). M254 is a hypersialylated Immunoglobulin G (hsIgG), a compound believed to be much stronger than IVIg alone. This study is looking to recruit participants between the ages of 18-55 years. Participants can expect to receive either M254 or a placebo if they are healthy, and M254 either alone or in conjunction with IVIg if they have ITP. Administration of IVIg may be at the same time or weeks apart.
USA, Belgium, France, Germany, Hungary, Italy, Netherlands, Poland, Spain.
Recruiting
Bulgaria - A Study of PRN1008 in Adult Patients With Immune Thrombocytopenic Purpura (ITP)
Principia Biopharma is conducting a study of their oral drug PRN1008 in patients with ITP who are refractory or relapsed with no available and approved therapeutic options and have a platelet count <30,000/μL on two counts no sooner than 7 days apart in the 15 days before treatment begins. Preclinical findings show that PRN1008 decreases the activity of B cells and other inflammatory-causing immune cells, and in a mouse model of ITP show that PRN1008 can reduce platelet loss.
https://clinicaltrials.gov/ct2/show/NCT03395210?term=PRN1008&rank=1
Recruiting
Bulgaria - A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ADVANCE)
Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
USA, Austria, Belgium, Bulgaria, Czech Republic, France, Germany, Georgia, Hungary, Italy, Japan, Netherlands, Poland, Russian Federation, Spain, Turkey, Ukraine, and the United Kingdom.
Recruiting
Bulgaria - A study to evaluate the efficacy, safety, and tolerability of Rozanolixizumab in adult study participants with persistent or chronic primary immune thrombocytopenia (ITP) (myOpportunITy1)
UCB Biopharma SRL is conducting a phase three, multicenter, double-blind, randomized, placebo-controlled study to assess the efficacy, safety, and tolerability of Rozanolixizumab, a humanized monoclonal antibody that binds to the human FcRn receptor, in adults (18 years of age or older) with persistent or chronic primary ITP. The purpose is also to demonstrate the clinical efficacy of Rozanolixizumab as a maintenance therapy. Rozanolixizumab is administered through a subcutaneous injection. Participants must have tried, and responded to, at least one previous ITP-related medication.
Belgium, Bulgaria, France, Georgia, Japan, Moldova, Poland, Russia, Spain, Ukraine, and USA
Currently recruiting
Bulgaria - A study to evaluate the efficacy, safety, and tolerability of Rozanolixizumab in adult study participants with persistent or chronic primary immune thrombocytopenia (ITP) (myOpportunITy2)
UCB Biopharma SRL is conducting a phase three, multicenter, double-blind, randomized, placebo-controlled study to assess the efficacy, safety, and tolerability of Rozanolixizumab, a humanized monoclonal antibody that binds to the human FcRn receptor, in adults (18 years of age or older) with persistent or chronic primary ITP. The purpose is also to demonstrate the clinical efficacy of Rozanolixizumab as a maintenance therapy. Rozanolixizumab is administered through a subcutaneous injection. Participants must have tried, and responded to, at least one previous ITP-related medication. Participants must also be able to receive immunosuppressive treatments.
Austria, Belgium, Bulgaria, Canada, China, Czechia, France, Germany, Italy, Poland, Russian Federation, Spain, Taiwan, Ukraine, UK, and USA.
https://clinicaltrials.gov/ct2/show/NCT04224688?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=5
Recruiting
Bulgaria - Long-term Study to Assess the Safety and Efficacy of Efgartimod in Adult Patients with Primary Immune Thrombocytopenia (ITP) (ADVANCE+)
Argenx is conducting this open-label (no placebo) multicenter phase three trial for efgartimod (ARGX-113) in adult ITP patients with primary ITP. Patients must be over the age of 18 years. Administration of efgartimod through IV injection, a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
Belgium, Hungary, Italy, Japan, The Netherlands, Russian Federation, Spain, Ukraine, USA
Status: Recruiting
Bulgaria - Study to Evaluate Safety, Tolerability, and Efficacy of TAK-079 in Participants with Persistent/Chronic Primary Immune Thrombocytopenia
Takeda pharmaceuticals is conducting a multi-centered phase two placebo-controlled double-blinded study to look at the safety, tolerability, and efficacy of a drug called TAK-079, which is a monoclonal antibody (anti-CD38 antibody). The trial is for adult ITP patients whose ITP is persistent or chronic, with a platelet count less than 30,000/µL for four consecutive weeks prior to participating in the trial. TAK-079 would be administered as a subcutaneous injection.
Bulgaria, Croatia, Germany, Italy, Slovenia, Ukraine, and USA.
Recruiting
Canada - A Study of PRN1008 in Adult Patients With Immune Thrombocytopenic Purpura (ITP)
Principia Biopharma is conducting a study of their oral drug PRN1008 in patients with ITP who are refractory or relapsed with no available and approved therapeutic options and have a platelet count <30,000/μL on two counts no sooner than 7 days apart in the 15 days before treatment begins. Preclinical findings show that PRN1008 decreases the activity of B cells and other inflammatory-causing immune cells, and in a mouse model of ITP show that PRN1008 can reduce platelet loss.
https://clinicaltrials.gov/ct2/show/NCT03395210?term=PRN1008&rank=1
Recruiting
Canada - A study to evaluate the efficacy, safety, and tolerability of Rozanolixizumab in adult study participants with persistent or chronic primary immune thrombocytopenia (ITP) (myOpportunITy2)
UCB Biopharma SRL is conducting a phase three, multicenter, double-blind, randomized, placebo-controlled study to assess the efficacy, safety, and tolerability of Rozanolixizumab, a humanized monoclonal antibody that binds to the human FcRn receptor, in adults (18 years of age or older) with persistent or chronic primary ITP. The purpose is also to demonstrate the clinical efficacy of Rozanolixizumab as a maintenance therapy. Rozanolixizumab is administered through a subcutaneous injection. Participants must have tried, and responded to, at least one previous ITP-related medication. Participants must also be able to receive immunosuppressive treatments.
Austria, Belgium, Bulgaria, Canada, China, Czechia, France, Germany, Italy, Poland, Russian Federation, Spain, Taiwan, Ukraine, UK, and USA.
https://clinicaltrials.gov/ct2/show/NCT04224688?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=5
Recruiting
Canada - Proof-of-Concept: A Pilot, Randomized, Double-Blind Study of Oseltamivir Versus Placebo for Immune Thrombocytopenia
Oseltamivir is an attractive drug for ITP since it specifically targets a pathophysiologic mechanism that appears to be important for the development of ITP and has a benign side effect profile compared to standard ITP therapy. This pilot, proof-of-concept, randomized controlled clinical trial has the potential to dramatically change the treatment of ITP.
https://clinicaltrials.gov/ct2/show/NCT03520049?recrs=a&cond=ITP&draw=4&rank=24
Status: Recruiting
China - A Clinical Study on the Efficacy and Safety of HBM9161 (Batoclimab) in Patients With Immune Thrombocytopenia (ITP)
Harbour BioMed (Guangzhou) Co. Ltd. Is conducting a phase 2/3 randomized, double-blind, placebo-controlled, clinical study to evaluate the efficacy and safety of HBM9161, a human anti-FcRN monoclonal antibody that removes IgG to reduce platelet destruction and increase the overall platelet count. This drug is administered through a subcutaneous injection and in this study would be provided to participants with ITP weekly. The study will be conducted in a Phase 2/3 operational seamless design. Eligible participants include adults with ITP who are 18 years of age or older. Participants must have chronic ITP and an average platelet count under 30,000 µL with no prior bleeding at least four weeks before enrollment. There are a number of exclusions including having a known autoimmune disease in addition to ITP, or a known secondary ITP.
China
https://www.clinicaltrials.gov/ct2/show/NCT04428255?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=39
Recruiting
China - A Study of Eltrombopag and Recombinant Human Thrombopoietin in Primary Immune Thrombocytopenia
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
Active, but no longer recruiting
China - A study to evaluate the efficacy, safety, and tolerability of Rozanolixizumab in adult study participants with persistent or chronic primary immune thrombocytopenia (ITP) (myOpportunITy2)
UCB Biopharma SRL is conducting a phase three, multicenter, double-blind, randomized, placebo-controlled study to assess the efficacy, safety, and tolerability of Rozanolixizumab, a humanized monoclonal antibody that binds to the human FcRn receptor, in adults (18 years of age or older) with persistent or chronic primary ITP. The purpose is also to demonstrate the clinical efficacy of Rozanolixizumab as a maintenance therapy. Rozanolixizumab is administered through a subcutaneous injection. Participants must have tried, and responded to, at least one previous ITP-related medication. Participants must also be able to receive immunosuppressive treatments.
Austria, Belgium, Bulgaria, Canada, China, Czechia, France, Germany, Italy, Poland, Russian Federation, Spain, Taiwan, Ukraine, UK, and USA.
https://clinicaltrials.gov/ct2/show/NCT04224688?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=5
Recruiting
China - Atorvastatin in Management of Newly Diagnosed ITP
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
Recruiting
China - Avatrombopag in Primary Immune Thrombocytopenia (ITP) in Adults
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
Status: Recruiting
China - Efficacy and Safety of rhTPO and Eltrombopag in Patients With Primary Immune Thrombocytopenia (ITP) ((ITP;rhTPO))
This study is a multicenter randomized, double-blind trial conducted through Wuhan Union Hospital, China, where participants are divided into two groups to analyze the efficacy and safety of using recombinant TPO agents (rhTPO) and eltrombopag to treat primary ITP in adult patients. In one group, participants can expect to receive a subcutaneous rhTPO agonist injection or an oral placebo tablet. In the second group, participants can expect to receive oral eltrombopag or a placebo injection. This study will only include adult ITP patients between the ages of 18 years – 75 years in good health.
China
https://www.clinicaltrials.gov/ct2/show/NCT03771378?cond=Immune+Thrombocytopenia&draw=2&rank=34
Recruiting
China - Eltrombopag Combining Rituximab vs Eltrombopag in the Management of Primary Immune Thrombocytopenia (ITP) in Adults
This phase 4 multicenter open-label trial is designed to compare the efficacy of using eltrombopag alone vs in combination with Rituximab in adult patients aged 18-60 years who have not responded to first-line therapies or splenectomy, including those who have relapsed ITP to determine if there is a benefit of combining therapies, and to determine the efficacy in previously treated ITP patients.
Recruiting
China - Eltrombopag Plus Diacerein vs Eltrombopag in Adult ITP
Qilu Hospital of Shandong University in China is conducting a phase two multicentre randomized controlled study to determine if adding diacerin, a slow-acting anthraquinone medicine also known as diacetylrhein used to treat joint diseases such as osteoarthritis, can elicit a good response in combination with eltrombopag. Study leads speculate that the addition of diacerein to eltrombopag may offer a sensitizer effect to maximize efficacy. Eligibility is restricted to adult participants with primary ITP who are 18 years of age or older and do not respond well (or at all) to eltrombopag, or who have relapsed following discontinuation of eltrombobag.
China
https://www.clinicaltrials.gov/ct2/show/NCT04917679
Recruiting
China - Eltrombopag Plus rhTPO vs Eltrombopag for ITP During the COVID-19 Pandemic (ELABORATE-19)
The Peking University People’s Hospital is conducting this phase two prospective multicenter clinical trial to investigate the efficacy and safety of using eltrombopag plus rhTPO vs eltrombopag alone for adult ITP patients (18-80 years) who are corticosteroid resistant or have relapsed ITP during the COVID-19 pandemic. Since they increase the number of platelets through different mechanisms, and previous studies demonstrated that they might exert synergic effect. The investigators hypothesized that the combination of these two agents could be a promising option for ITP treatment.
China
Recruiting
China - Evaluation of Immune Status Before and After Splenectomy in Immune Thrombocytopenia Patients
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
Status: Recruiting
China - Fixed Low-Dose Eltrombopag and rhTPO for ITP (FLOWER)
The Peking University People’s Hospital is conducting this single-arm phase one clinical trial to investigate the efficacy and safety of using a combination therapy of a fixed low-dose of eltrombopag coupled with recombinant human TPO for adults with ITP who are between the ages of 18-80 years, and who are resistant to steroids or have relapsed ITP.
China
Recruiting
China - Glycyrrhetinic Acid Combined With Dexamethasone in Management of Newly Diagnosed ITP
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
Recruiting
China - Multicentre, Randomised Phase III Study of the Efficacy and Safety of Hetrombopag Olamine in Idiopathic Thrombocytopenic Purpura (ITP) Patient
A multicentre, randomised, double-blind,4-stages phase III study enrolled 414 patients with chronic, previously treated ITP. Dosage of Hetrombopag olamine could be adjusted to maintain platelet counts 50~250×109/L. Hetrombopag olamine is a non-peptide thrombopoietin (TPO) receptor agonist that attempts to increase platelet production.
https://clinicaltrials.gov/ct2/show/NCT03222843?recrs=a&cond=ITP&draw=2&rank=3
Status: Unknown
China - Single-dose Anti-CD20 Antibody With Bortezomib for Relapsed Refractory Autoimmune Hemolytic Anemia (RRAIHA01)
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
China - Study of Human Umbilical Cord-derived Mesenchymal Stem Cells for Treatment of Refractory Immune Thrombocytopenia
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
Recruiting
China - The Combination of High-dose Dexamethasone and Acetylcysteine as the Treatment of Newly Diagnosed ITP
This is a phase two, single-arm, open-label study through Peking University People’s Hospital in China to study the efficacy and safety of acetylcysteine (also called N-acetylcysteine or NAC) and high dose dexamethasone for therapy in adults with newly diagnosed ITP. NAC is a therapy that has traditionally been used to treat cystic fibrosis and other respiratory conditions by reducing mucous secretions. Recently, it’s been studied in patients with other platelet disorders such as thrombotic thrombocytopenic purpura (TTP) and is now being investigated for its potential treatment of ITP. In this study, NAC would be given orally, three times a day, in combination with a four-day high-dose pulse treatment of dexamethasone. Eligibility is restricted to adult patients with newly diagnosed ITP who have never had cancer or a splenectomy, are between the ages of 18-80 years, and have not been treated previously with Rituxan®. Participants must also have a diagnosis of primary ITP and have a platelet count under 30,000μL.
China
https://clinicaltrials.gov/ct2/show/NCT04368598?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=13
Recruiting
China - The Combination of Low-dose Rituximab and ATRA as the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia
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
China - The Combination of Low-dose Rituximab and ATRA as the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia
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
China - The Combination of Terbutaline and Danazol as the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia
The Peking University People’s Hospital is conducting a phase two single-arm single center study to assess the efficacy and safety of using a combination of terbutaline and danazol in adult patients (over the age of 18 years) who are resistant to steroids or who have relapsed with ITP and have a platelet count less than 30,000. Terbutaline is a beta2-AR agonist that modulates T cell differentiation and effector cell function. It is branded as Bricanyl which is commonly used for asthma in inhalers.
China
Recruiting
China - The Efficacy and Safety of Huaiqihuang Granule in Children With Chronic Primary Immune Thrombocytopenia (ITP)
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.
https://clinicaltrials.gov/ct2/show/NCT03201848?recrs=a&cond=ITP&draw=1&rank=25
Recruiting
China - The Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of HMPL-523 in Immune Thrombocytopenia Patients
Hutchison Medipharma Limited is conducting a study to assess the Safety, tolerability, pharmacokinetics and preliminary efficacy of HMPL-523, a syk inhibitor, in adult patients with immune thrombocytopenia. This is a randomized, double blinded, placebo controlled phase Ib study where participants can expect to receive the sky inhibitor for up to eight weeks. Participants must be refractory to other therapies and be between ages 18 years – 75 years of age, and have either persistent or chronic ITP.
China
https://www.clinicaltrials.gov/ct2/show/NCT03951623?cond=Immune+Thrombocytopenia&draw=2&rank=35
Recruiting
China - To Evaluate the Efficacy and Safety of Eltrombopag for Thrombocytopenia With Chronic HBV Infection
Novartis Pharmaceuticals and a number of participating medical centres/universities in China are conducting this multi-center single-arm study alongside the Institute of Hematology & Blood Diseases Hospital to evaluate the efficacy and safety of eltrombopag to treat thrombocytopenia associated with chronic hepatitis B virus infection. This study includes two parts. In the first part short-term efficacy, safety and tolerability of eltrombopag is evaluated. Any subjects who benefit from eltrombopag treatment (platelet count ≥ 50×109/L) will then be offered the option to enter part two designed to evaluate the long-term efficacy and safety of the drug. This study is open to patients with primary ITP who are over the age of 18 years and were diagnosed with an HBV-infection for at least 6 months prior to the study. They also have to have a platelet count of less than 30K microlitres <30 ×109/L.
China
https://www.clinicaltrials.gov/ct2/show/NCT03664518?titles=To+evaluate+the+efficacy+and+safety+of+eltrombopag+for+thrombocytopenia+with+chronic+HBV+infection&draw=2&rank=1
Recruiting
Croatia - Study to Evaluate Safety, Tolerability, and Efficacy of TAK-079 in Participants with Persistent/Chronic Primary Immune Thrombocytopenia
Takeda pharmaceuticals is conducting a multi-centered phase two placebo-controlled double-blinded study to look at the safety, tolerability, and efficacy of a drug called TAK-079, which is a monoclonal antibody (anti-CD38 antibody). The trial is for adult ITP patients whose ITP is persistent or chronic, with a platelet count less than 30,000/µL for four consecutive weeks prior to participating in the trial. TAK-079 would be administered as a subcutaneous injection.
Bulgaria, Croatia, Germany, Italy, Slovenia, Ukraine, and USA.
Recruiting
Czech Republic - A Study of PRN1008 in Adult Patients With Immune Thrombocytopenic Purpura (ITP)
Principia Biopharma is conducting a study of their oral drug PRN1008 in patients with ITP who are refractory or relapsed with no available and approved therapeutic options and have a platelet count <30,000/μL on two counts no sooner than 7 days apart in the 15 days before treatment begins. Preclinical findings show that PRN1008 decreases the activity of B cells and other inflammatory-causing immune cells, and in a mouse model of ITP show that PRN1008 can reduce platelet loss.
https://clinicaltrials.gov/ct2/show/NCT03395210?term=PRN1008&rank=1
Recruiting
Czech Republic - A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ADVANCE)
Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
USA, Austria, Belgium, Bulgaria, Czech Republic, France, Germany, Georgia, Hungary, Italy, Japan, Netherlands, Poland, Russian Federation, Spain, Turkey, Ukraine, and the United Kingdom.
Recruiting
Czech Republic - A study to evaluate the efficacy, safety, and tolerability of Rozanolixizumab in adult study participants with persistent or chronic primary immune thrombocytopenia (ITP) (myOpportunITy2)
UCB Biopharma SRL is conducting a phase three, multicenter, double-blind, randomized, placebo-controlled study to assess the efficacy, safety, and tolerability of Rozanolixizumab, a humanized monoclonal antibody that binds to the human FcRn receptor, in adults (18 years of age or older) with persistent or chronic primary ITP. The purpose is also to demonstrate the clinical efficacy of Rozanolixizumab as a maintenance therapy. Rozanolixizumab is administered through a subcutaneous injection. Participants must have tried, and responded to, at least one previous ITP-related medication. Participants must also be able to receive immunosuppressive treatments.
Austria, Belgium, Bulgaria, Canada, China, Czechia, France, Germany, Italy, Poland, Russian Federation, Spain, Taiwan, Ukraine, UK, and USA.
https://clinicaltrials.gov/ct2/show/NCT04224688?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=5
Recruiting
Czech Republic - Long-term Study to Assess the Safety and Efficacy of Efgartimod in Adult Patients with Primary Immune Thrombocytopenia (ITP) (ADVANCE+)
Argenx is conducting this open-label (no placebo) multicenter phase three trial for efgartimod (ARGX-113) in adult ITP patients with primary ITP. Patients must be over the age of 18 years. Administration of efgartimod through IV injection, a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
Belgium, Hungary, Italy, Japan, The Netherlands, Russian Federation, Spain, Ukraine, USA
Status: Recruiting
Denmark - The DART Study- Daratumumab (Darzalex) Treatment in ITP
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
Egypt - Effects of Helicobacter Pylori Eradication in Children With Chronic Immune Thrombocytopenic Purpura
Assiut University is looking at whether treatment for H.pylori increases the overall platelet count in individuals who are infected with H. Pylori, a bacteria in the digestive tract that has been associated in some cases with ITP. Both adults and children over the age of one year are eligible to participate. Participants must have chronic ITP and be resistant to steroids and IVIg.
Egypt
https://clinicaltrials.gov/ct2/show/NCT03172676?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=14
Recruiting
Egypt - The Effect of Eltrombopag on the Expression of Platelet Collagen Receptor GPVI in Pediatric ITP
This study investigates Eltrombopag as an alternative treatment pathway, which depend on stimulation of thrombocyte synthesis, in chronic and persistent immune thrombocytopenia that may be a more promising treatment than the classic type, not only by increasing platelets count but also through enhancing of the platelets activation and up regulation of GPVI expression on platelet surface.
This non-randomized single center study conducted through Ain Shams University in Egypt seeks to explore the efficacy, safety, and tolerability of eltrombopag in pediatric patients between the ages of 1-18 years who have persistent or chronic ITP. The study also seeks to better understand how eltrombopag effects platelet activation by up-regulating the glycoprotein VI (GPVI) receptor and altering it’s natural expression, in comparison to other available ITP treatments. Participants can expect to be followed every two weeks for the duration of the study and will have a baseline bone marrow examination (with reticulin stain) performed at the start as well as at the 24 week mark. Baseline liver and kidney function tests will be performed and repeated at 24 weeks into the study. Participants will also be asked to report on their quality of life using the Kid’s ITP Tools (KIT) questionnaires and this will be repeated at the end of the study.
Egypt
https://www.clinicaltrials.gov/ct2/show/NCT03412188?recrs=ab&cond=Immune+Thrombocytopenia&draw=2&rank=36
Recruiting
France - A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ADVANCE)
Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
USA, Austria, Belgium, Bulgaria, Czech Republic, France, Germany, Georgia, Hungary, Italy, Japan, Netherlands, Poland, Russian Federation, Spain, Turkey, Ukraine, and the United Kingdom.
Recruiting
France - A study to evaluate the efficacy, safety, and tolerability of Rozanolixizumab in adult study participants with persistent or chronic primary immune thrombocytopenia (ITP) (myOpportunITy1)
UCB Biopharma SRL is conducting a phase three, multicenter, double-blind, randomized, placebo-controlled study to assess the efficacy, safety, and tolerability of Rozanolixizumab, a humanized monoclonal antibody that binds to the human FcRn receptor, in adults (18 years of age or older) with persistent or chronic primary ITP. The purpose is also to demonstrate the clinical efficacy of Rozanolixizumab as a maintenance therapy. Rozanolixizumab is administered through a subcutaneous injection. Participants must have tried, and responded to, at least one previous ITP-related medication.
Belgium, Bulgaria, France, Georgia, Japan, Moldova, Poland, Russia, Spain, Ukraine, and USA
Currently recruiting
France - A study to evaluate the efficacy, safety, and tolerability of Rozanolixizumab in adult study participants with persistent or chronic primary immune thrombocytopenia (ITP) (myOpportunITy2)
UCB Biopharma SRL is conducting a phase three, multicenter, double-blind, randomized, placebo-controlled study to assess the efficacy, safety, and tolerability of Rozanolixizumab, a humanized monoclonal antibody that binds to the human FcRn receptor, in adults (18 years of age or older) with persistent or chronic primary ITP. The purpose is also to demonstrate the clinical efficacy of Rozanolixizumab as a maintenance therapy. Rozanolixizumab is administered through a subcutaneous injection. Participants must have tried, and responded to, at least one previous ITP-related medication. Participants must also be able to receive immunosuppressive treatments.
Austria, Belgium, Bulgaria, Canada, China, Czechia, France, Germany, Italy, Poland, Russian Federation, Spain, Taiwan, Ukraine, UK, and USA.
https://clinicaltrials.gov/ct2/show/NCT04224688?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=5
Recruiting
France - Assessment of Quality of Life of Children With Chronic Idiopathic Thrombocytopenic Purpura (Kid's ITP Tools) (KIT)
This study plans to examine the quality of life of patient with chronic idiopathic thrombocytopenic purpura according to the type of treatment received.
France
https://clinicaltrials.gov/ct2/show/NCT03123497?recrs=a&cond=ITP&draw=2&rank=11
Recruiting
France - Autoimmune Cytopenias : Midi-Pyrenees Registry (CARMEN)
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.
https://clinicaltrials.gov/ct2/show/NCT02877706?recrs=a&cond=ITP&draw=1&rank=38
Recruiting
France - Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia
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
France - Biological Bank for the Patients Followed in a Constitutive Reference Center for Autoimmune Cytopenia (BIOCAI)
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
Recruiting
France - Dietary Phytoestrogens as Risk Factors for Systemic Lupus Erythematosus (ISOLED)
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
France - French Adult Primary Immune Thrombocytopenia (FAITH)
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
Recruiting
France - Long-term Study to Assess the Safety and Efficacy of Efgartimod in Adult Patients with Primary Immune Thrombocytopenia (ITP) (ADVANCE+)
Argenx is conducting this open-label (no placebo) multicenter phase three trial for efgartimod (ARGX-113) in adult ITP patients with primary ITP. Patients must be over the age of 18 years. Administration of efgartimod through IV injection, a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
Belgium, Hungary, Italy, Japan, The Netherlands, Russian Federation, Spain, Ukraine, USA
Status: Recruiting
France - Rate of Prolonged Response After Stopping Thrombopoietin-receptor Agonists Treatment in ITP (STOP-AGO)
TPO-RAs have been thought to play only a supporting role in ITP management. The purpose of this study is to demonstrate that a substantial proportion of ITP patients may achieve a prolonged response after TPO-RA discontinuation.
https://clinicaltrials.gov/ct2/show/NCT03119974?recrs=a&cond=ITP&rank=1
Recruiting
France - Risk Factors for Thrombosis in Immune Thrombocytopenia (RiFT-ITP)
This study through Toulouse University Hospital is a retrospective case-control observational population-based study specifically focusing on adult patients with ITP between July 2009 and June 2015 who were identified through the French national insurance database. The goal of the study is to identify risk factors for thrombosis in this patient population.
France
Recruiting
France - Role of Fcgamma Receptors in Immune Thrombocytopenia (ITP) (PTI Fc)
Monocytes/macrophages play a double role by phagocyting platelets recognized by autoantibodies and by maintaining the autoimmune response via their antigen-presenting cell functions. Fcgamma receptors (FcγR), that are represented by activating receptors (FcγRI, FcγRIIa, FcγRIII) and an inhibiting one (FcγRIIb), are involved in the regulation of macrophages and have been reported to be dysregulated in autoimmune diseases such as rheumatoid arthritis and systemic lupus erythematous.
The aim of this study is to compare the expression of FcγR in patients with ITP on circulating monocytes and on splenic macrophages.
France
https://clinicaltrials.gov/ct2/show/NCT02821572?recrs=a&cond=ITP&rank=4
Unknown
France - Safety and tolerability of M254 in healthy volunteers and immune thrombocytopenia patients
This is an open-label two phase study through Momenta Pharmaceuticals, Inc., to look at the safety, tolerability, pharmacodynamics/pharmacokinetics of M254 in healthy patients compared to patients with persistent or chronic immune thrombocytopenia (ITP). M254 is a hypersialylated Immunoglobulin G (hsIgG), a compound believed to be much stronger than IVIg alone. This study is looking to recruit participants between the ages of 18-55 years. Participants can expect to receive either M254 or a placebo if they are healthy, and M254 either alone or in conjunction with IVIg if they have ITP. Administration of IVIg may be at the same time or weeks apart.
USA, Belgium, France, Germany, Hungary, Italy, Netherlands, Poland, Spain.
Recruiting
France - Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP) (LUNA3)
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, Australia, France, Poland, South Korea, Spain, USA
https://clinicaltrials.gov/ct2/show/NCT04562766
Recruiting
France - The DART Study- Daratumumab (Darzalex) Treatment in ITP
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
France - Thrombocytopenia Drug Immune Validation of New Biological Test Diagnostic (TITIM)
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
Recruiting
Georgia - A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ADVANCE)
Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
USA, Austria, Belgium, Bulgaria, Czech Republic, France, Germany, Georgia, Hungary, Italy, Japan, Netherlands, Poland, Russian Federation, Spain, Turkey, Ukraine, and the United Kingdom.
Recruiting
Georgia - A study to evaluate the efficacy, safety, and tolerability of Rozanolixizumab in adult study participants with persistent or chronic primary immune thrombocytopenia (ITP) (myOpportunITy1)
UCB Biopharma SRL is conducting a phase three, multicenter, double-blind, randomized, placebo-controlled study to assess the efficacy, safety, and tolerability of Rozanolixizumab, a humanized monoclonal antibody that binds to the human FcRn receptor, in adults (18 years of age or older) with persistent or chronic primary ITP. The purpose is also to demonstrate the clinical efficacy of Rozanolixizumab as a maintenance therapy. Rozanolixizumab is administered through a subcutaneous injection. Participants must have tried, and responded to, at least one previous ITP-related medication.
Belgium, Bulgaria, France, Georgia, Japan, Moldova, Poland, Russia, Spain, Ukraine, and USA
Currently recruiting
Germany - A Study to Assess Efficacy and Safety of Eltrombopag in Combination with a Short Course of Dexamethasone in Patients with Newly Diagnosed ITP (XPAG-ITP)
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
Germany - A Study to Assess Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple- Dose BIVV009 in Participants With Chronic Immune Thrombocytopenia (ITP)
The purpose of this study is to explore the safety, preliminary clinical benefit, and activity of BIVV009 in patients with chronic immune thrombocytopenia. BIVV009 has been designed to block the pathway that triggers the immune system to remove damaged cells. It is believed that this approach may limit the number of cells immune system removes and allow the extra cells to help promote platelet production.
https://clinicaltrials.gov/ct2/show/NCT03275454?recrs=a&cond=ITP&draw=4&rank=30
Active, but no longer recruiting
Germany - A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ADVANCE)
Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
USA, Austria, Belgium, Bulgaria, Czech Republic, France, Germany, Georgia, Hungary, Italy, Japan, Netherlands, Poland, Russian Federation, Spain, Turkey, Ukraine, and the United Kingdom.
Recruiting
Germany - A study to evaluate the efficacy, safety, and tolerability of Rozanolixizumab in adult study participants with persistent or chronic primary immune thrombocytopenia (ITP) (myOpportunITy2)
UCB Biopharma SRL is conducting a phase three, multicenter, double-blind, randomized, placebo-controlled study to assess the efficacy, safety, and tolerability of Rozanolixizumab, a humanized monoclonal antibody that binds to the human FcRn receptor, in adults (18 years of age or older) with persistent or chronic primary ITP. The purpose is also to demonstrate the clinical efficacy of Rozanolixizumab as a maintenance therapy. Rozanolixizumab is administered through a subcutaneous injection. Participants must have tried, and responded to, at least one previous ITP-related medication. Participants must also be able to receive immunosuppressive treatments.
Austria, Belgium, Bulgaria, Canada, China, Czechia, France, Germany, Italy, Poland, Russian Federation, Spain, Taiwan, Ukraine, UK, and USA.
https://clinicaltrials.gov/ct2/show/NCT04224688?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=5
Recruiting
Germany - Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia
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
Germany - Long-term Study to Assess the Safety and Efficacy of Efgartimod in Adult Patients with Primary Immune Thrombocytopenia (ITP) (ADVANCE+)
Argenx is conducting this open-label (no placebo) multicenter phase three trial for efgartimod (ARGX-113) in adult ITP patients with primary ITP. Patients must be over the age of 18 years. Administration of efgartimod through IV injection, a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
Belgium, Hungary, Italy, Japan, The Netherlands, Russian Federation, Spain, Ukraine, USA
Status: Recruiting
Germany - Safety and tolerability of M254 in healthy volunteers and immune thrombocytopenia patients
This is an open-label two phase study through Momenta Pharmaceuticals, Inc., to look at the safety, tolerability, pharmacodynamics/pharmacokinetics of M254 in healthy patients compared to patients with persistent or chronic immune thrombocytopenia (ITP). M254 is a hypersialylated Immunoglobulin G (hsIgG), a compound believed to be much stronger than IVIg alone. This study is looking to recruit participants between the ages of 18-55 years. Participants can expect to receive either M254 or a placebo if they are healthy, and M254 either alone or in conjunction with IVIg if they have ITP. Administration of IVIg may be at the same time or weeks apart.
USA, Belgium, France, Germany, Hungary, Italy, Netherlands, Poland, Spain.
Recruiting
Germany - Study to Evaluate Safety, Tolerability, and Efficacy of TAK-079 in Participants with Persistent/Chronic Primary Immune Thrombocytopenia
Takeda pharmaceuticals is conducting a multi-centered phase two placebo-controlled double-blinded study to look at the safety, tolerability, and efficacy of a drug called TAK-079, which is a monoclonal antibody (anti-CD38 antibody). The trial is for adult ITP patients whose ITP is persistent or chronic, with a platelet count less than 30,000/µL for four consecutive weeks prior to participating in the trial. TAK-079 would be administered as a subcutaneous injection.
Bulgaria, Croatia, Germany, Italy, Slovenia, Ukraine, and USA.
Recruiting
Greece - The Oscotec Clinical Trial called Study of Oral SKI-O-703, SYK Inhibitor, in Patients with Persistent and Chronic ITP
This is a phase two double-blind, randomized, multicenter study through Oscotec Inc. designed to evaluate the efficacy and safety of SKI-0-703, an oral SYK inhibitor. This study is targeting adult ITP patients who have persistent or chronic ITP and have failed to respond adequately to other approved therapies, or who have relapsed following exposure to other approved ITP treatments. SYK inhibitors play important roles in signalling within the immune system, particularly in the adaptive immune system. Inhibition of SYK leads to reduced antibody mediated destruction of platelets. Eligibility includes individuals diagnosed with ITP for at least three months who have a platelet count less than 30,000 µL and are at least 18 years of age.
USA, Greece, Republic of Korea, Poland, Spain
Recruiting
Hong Kong - Safety and Efficacy of Eltrombopag Plus Pulsed Dexamethasone for Subjects With Idiopathic Thrombocytopenic Purpura
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
Hong Kong - Safety and Efficacy of Fecal Microbiota Transplantation
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
Hungary - A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ADVANCE)
Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
USA, Austria, Belgium, Bulgaria, Czech Republic, France, Germany, Georgia, Hungary, Italy, Japan, Netherlands, Poland, Russian Federation, Spain, Turkey, Ukraine, and the United Kingdom.
Recruiting
Hungary - Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia
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
Hungary - Long-term Study to Assess the Safety and Efficacy of Efgartimod in Adult Patients with Primary Immune Thrombocytopenia (ITP) (ADVANCE+)
Argenx is conducting this open-label (no placebo) multicenter phase three trial for efgartimod (ARGX-113) in adult ITP patients with primary ITP. Patients must be over the age of 18 years. Administration of efgartimod through IV injection, a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
Belgium, Hungary, Italy, Japan, The Netherlands, Russian Federation, Spain, Ukraine, USA
Status: Recruiting
Hungary - Safety and tolerability of M254 in healthy volunteers and immune thrombocytopenia patients
This is an open-label two phase study through Momenta Pharmaceuticals, Inc., to look at the safety, tolerability, pharmacodynamics/pharmacokinetics of M254 in healthy patients compared to patients with persistent or chronic immune thrombocytopenia (ITP). M254 is a hypersialylated Immunoglobulin G (hsIgG), a compound believed to be much stronger than IVIg alone. This study is looking to recruit participants between the ages of 18-55 years. Participants can expect to receive either M254 or a placebo if they are healthy, and M254 either alone or in conjunction with IVIg if they have ITP. Administration of IVIg may be at the same time or weeks apart.
USA, Belgium, France, Germany, Hungary, Italy, Netherlands, Poland, Spain.
Recruiting
Italy - A Narrative Approach to Describe QoL in Children With Chronic ITP
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
Italy - A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ADVANCE)
Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
USA, Austria, Belgium, Bulgaria, Czech Republic, France, Germany, Georgia, Hungary, Italy, Japan, Netherlands, Poland, Russian Federation, Spain, Turkey, Ukraine, and the United Kingdom.
Recruiting
Italy - A study to evaluate the efficacy, safety, and tolerability of Rozanolixizumab in adult study participants with persistent or chronic primary immune thrombocytopenia (ITP) (myOpportunITy2)
UCB Biopharma SRL is conducting a phase three, multicenter, double-blind, randomized, placebo-controlled study to assess the efficacy, safety, and tolerability of Rozanolixizumab, a humanized monoclonal antibody that binds to the human FcRn receptor, in adults (18 years of age or older) with persistent or chronic primary ITP. The purpose is also to demonstrate the clinical efficacy of Rozanolixizumab as a maintenance therapy. Rozanolixizumab is administered through a subcutaneous injection. Participants must have tried, and responded to, at least one previous ITP-related medication. Participants must also be able to receive immunosuppressive treatments.
Austria, Belgium, Bulgaria, Canada, China, Czechia, France, Germany, Italy, Poland, Russian Federation, Spain, Taiwan, Ukraine, UK, and USA.
https://clinicaltrials.gov/ct2/show/NCT04224688?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=5
Recruiting
Italy - Investigation on a Dynamic Cohort of Italian Patients with Active ITP (ITP0918)
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
Recruiting
Italy - Long-term Study to Assess the Safety and Efficacy of Efgartimod in Adult Patients with Primary Immune Thrombocytopenia (ITP) (ADVANCE+)
Argenx is conducting this open-label (no placebo) multicenter phase three trial for efgartimod (ARGX-113) in adult ITP patients with primary ITP. Patients must be over the age of 18 years. Administration of efgartimod through IV injection, a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
Belgium, Hungary, Italy, Japan, The Netherlands, Russian Federation, Spain, Ukraine, USA
Status: Recruiting
Italy - Response Rate and Duration After Discontinuation Thrombopoietin Receptor Agonists Primary Immune Thrombocytopenia
Gruppo Italiano Malattie EMatologiche dell'Adulto is conducting this study to determine if the use of TPO agonists (specifically eltrombopag and romiplostim) can cause a sustained or persistent remission in adults patients with either persistent or chronic ITP who have failed to respond to at least one other therapy.
Italy
Unknown
Italy - Safety and tolerability of M254 in healthy volunteers and immune thrombocytopenia patients
This is an open-label two phase study through Momenta Pharmaceuticals, Inc., to look at the safety, tolerability, pharmacodynamics/pharmacokinetics of M254 in healthy patients compared to patients with persistent or chronic immune thrombocytopenia (ITP). M254 is a hypersialylated Immunoglobulin G (hsIgG), a compound believed to be much stronger than IVIg alone. This study is looking to recruit participants between the ages of 18-55 years. Participants can expect to receive either M254 or a placebo if they are healthy, and M254 either alone or in conjunction with IVIg if they have ITP. Administration of IVIg may be at the same time or weeks apart.
USA, Belgium, France, Germany, Hungary, Italy, Netherlands, Poland, Spain.
Recruiting
Italy - Study to Evaluate Safety, Tolerability, and Efficacy of TAK-079 in Participants with Persistent/Chronic Primary Immune Thrombocytopenia
Takeda pharmaceuticals is conducting a multi-centered phase two placebo-controlled double-blinded study to look at the safety, tolerability, and efficacy of a drug called TAK-079, which is a monoclonal antibody (anti-CD38 antibody). The trial is for adult ITP patients whose ITP is persistent or chronic, with a platelet count less than 30,000/µL for four consecutive weeks prior to participating in the trial. TAK-079 would be administered as a subcutaneous injection.
Bulgaria, Croatia, Germany, Italy, Slovenia, Ukraine, and USA.
Recruiting
Japan - A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ADVANCE)
Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
USA, Austria, Belgium, Bulgaria, Czech Republic, France, Germany, Georgia, Hungary, Italy, Japan, Netherlands, Poland, Russian Federation, Spain, Turkey, Ukraine, and the United Kingdom.
Recruiting
Japan - A study to evaluate the efficacy, safety, and tolerability of Rozanolixizumab in adult study participants with persistent or chronic primary immune thrombocytopenia (ITP) (myOpportunITy1)
UCB Biopharma SRL is conducting a phase three, multicenter, double-blind, randomized, placebo-controlled study to assess the efficacy, safety, and tolerability of Rozanolixizumab, a humanized monoclonal antibody that binds to the human FcRn receptor, in adults (18 years of age or older) with persistent or chronic primary ITP. The purpose is also to demonstrate the clinical efficacy of Rozanolixizumab as a maintenance therapy. Rozanolixizumab is administered through a subcutaneous injection. Participants must have tried, and responded to, at least one previous ITP-related medication.
Belgium, Bulgaria, France, Georgia, Japan, Moldova, Poland, Russia, Spain, Ukraine, and USA
Currently recruiting
Japan - Long-term Study to Assess the Safety and Efficacy of Efgartimod in Adult Patients with Primary Immune Thrombocytopenia (ITP) (ADVANCE+)
Argenx is conducting this open-label (no placebo) multicenter phase three trial for efgartimod (ARGX-113) in adult ITP patients with primary ITP. Patients must be over the age of 18 years. Administration of efgartimod through IV injection, a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
Belgium, Hungary, Italy, Japan, The Netherlands, Russian Federation, Spain, Ukraine, USA
Status: Recruiting
Japan - The International ITP Registry
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.
Recruiting
Malaysia - The International ITP Registry
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.
Recruiting
Mexico - Romiplostim, Rituximab, and Dexamethasone as Frontline Treatment for Immune Thrombocytopenia
The Servicio de Hematología, Hospital Universitario in Mexico is conducting a n open-label study looking at the benefit of using corticosteroids together with second line therapies as front-line treatment in adults with primary ITP. Specifically, they are looking to determine if the response rate and duration of therapy outweighs using corticosteroids alone. Participants will receive a 1-4 day pulse dose of steroid, and then four weeks of Rituximab and romiplostim. Participants will be evaluated at 4 weeks and 6 months. In this study, patients must be between 16-90 years and have a platelet count less than 30,000 µL. Exclusion: Previous splenectomy.
Mexico
Recruiting
Moldova - A study to evaluate the efficacy, safety, and tolerability of Rozanolixizumab in adult study participants with persistent or chronic primary immune thrombocytopenia (ITP) (myOpportunITy1)
UCB Biopharma SRL is conducting a phase three, multicenter, double-blind, randomized, placebo-controlled study to assess the efficacy, safety, and tolerability of Rozanolixizumab, a humanized monoclonal antibody that binds to the human FcRn receptor, in adults (18 years of age or older) with persistent or chronic primary ITP. The purpose is also to demonstrate the clinical efficacy of Rozanolixizumab as a maintenance therapy. Rozanolixizumab is administered through a subcutaneous injection. Participants must have tried, and responded to, at least one previous ITP-related medication.
Belgium, Bulgaria, France, Georgia, Japan, Moldova, Poland, Russia, Spain, Ukraine, and USA
Currently recruiting
Netherlands - A Study of PRN1008 in Adult Patients With Immune Thrombocytopenic Purpura (ITP)
Principia Biopharma is conducting a study of their oral drug PRN1008 in patients with ITP who are refractory or relapsed with no available and approved therapeutic options and have a platelet count <30,000/μL on two counts no sooner than 7 days apart in the 15 days before treatment begins. Preclinical findings show that PRN1008 decreases the activity of B cells and other inflammatory-causing immune cells, and in a mouse model of ITP show that PRN1008 can reduce platelet loss.
https://clinicaltrials.gov/ct2/show/NCT03395210?term=PRN1008&rank=1
Recruiting
Netherlands - A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ADVANCE)
Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
USA, Austria, Belgium, Bulgaria, Czech Republic, France, Germany, Georgia, Hungary, Italy, Japan, Netherlands, Poland, Russian Federation, Spain, Turkey, Ukraine, and the United Kingdom.
Recruiting
Netherlands - Long-term Study to Assess the Safety and Efficacy of Efgartimod in Adult Patients with Primary Immune Thrombocytopenia (ITP) (ADVANCE+)
Argenx is conducting this open-label (no placebo) multicenter phase three trial for efgartimod (ARGX-113) in adult ITP patients with primary ITP. Patients must be over the age of 18 years. Administration of efgartimod through IV injection, a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
Belgium, Hungary, Italy, Japan, The Netherlands, Russian Federation, Spain, Ukraine, USA
Status: Recruiting
Netherlands - Safety and tolerability of M254 in healthy volunteers and immune thrombocytopenia patients
This is an open-label two phase study through Momenta Pharmaceuticals, Inc., to look at the safety, tolerability, pharmacodynamics/pharmacokinetics of M254 in healthy patients compared to patients with persistent or chronic immune thrombocytopenia (ITP). M254 is a hypersialylated Immunoglobulin G (hsIgG), a compound believed to be much stronger than IVIg alone. This study is looking to recruit participants between the ages of 18-55 years. Participants can expect to receive either M254 or a placebo if they are healthy, and M254 either alone or in conjunction with IVIg if they have ITP. Administration of IVIg may be at the same time or weeks apart.
USA, Belgium, France, Germany, Hungary, Italy, Netherlands, Poland, Spain.
Recruiting
New Zealand - A first-in-human trial to evaluate the safety, tolerability, and pharmacokinetics of PF-06755347
Pfizer is conducting a phase one, randomized, double-blind, placebo controlled human trial to evaluate the safety, tolerability, and pharmacokinetics of PF-06755347, a recombinant fragment crystallisable (rFc), in healthy adult male patients between 18-40 years of age. Male participants must have no evidence or history of clinically significant hematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, psychiatric, neurologic, or allergic disease (including drug allergies, but excluding untreated, asymptomatic, seasonal allergies at the time of dosing). Participants with a history of autoimmune disorders and other conditions that compromise or impair the immune system are also excluded from participating. It is hoped that this therapy could be used to treat ITP if tolerable and safe in healthy controls.
New Zealand
https://clinicaltrials.gov/ct2/show/NCT03275740?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=37
Recruiting
Norway - A Study of PRN1008 in Adult Patients With Immune Thrombocytopenic Purpura (ITP)
Principia Biopharma is conducting a study of their oral drug PRN1008 in patients with ITP who are refractory or relapsed with no available and approved therapeutic options and have a platelet count <30,000/μL on two counts no sooner than 7 days apart in the 15 days before treatment begins. Preclinical findings show that PRN1008 decreases the activity of B cells and other inflammatory-causing immune cells, and in a mouse model of ITP show that PRN1008 can reduce platelet loss.
https://clinicaltrials.gov/ct2/show/NCT03395210?term=PRN1008&rank=1
Recruiting
Norway - The DART Study- Daratumumab (Darzalex) Treatment in ITP
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
Norway - The PROLONG Trial - Rituximab Maintenance Therapy in ITP
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
Recruiting
Poland - A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ADVANCE)
Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
USA, Austria, Belgium, Bulgaria, Czech Republic, France, Germany, Georgia, Hungary, Italy, Japan, Netherlands, Poland, Russian Federation, Spain, Turkey, Ukraine, and the United Kingdom.
Recruiting
Poland - A study to evaluate the efficacy, safety, and tolerability of Rozanolixizumab in adult study participants with persistent or chronic primary immune thrombocytopenia (ITP) (myOpportunITy1)
UCB Biopharma SRL is conducting a phase three, multicenter, double-blind, randomized, placebo-controlled study to assess the efficacy, safety, and tolerability of Rozanolixizumab, a humanized monoclonal antibody that binds to the human FcRn receptor, in adults (18 years of age or older) with persistent or chronic primary ITP. The purpose is also to demonstrate the clinical efficacy of Rozanolixizumab as a maintenance therapy. Rozanolixizumab is administered through a subcutaneous injection. Participants must have tried, and responded to, at least one previous ITP-related medication.
Belgium, Bulgaria, France, Georgia, Japan, Moldova, Poland, Russia, Spain, Ukraine, and USA
Currently recruiting
Poland - A study to evaluate the efficacy, safety, and tolerability of Rozanolixizumab in adult study participants with persistent or chronic primary immune thrombocytopenia (ITP) (myOpportunITy2)
UCB Biopharma SRL is conducting a phase three, multicenter, double-blind, randomized, placebo-controlled study to assess the efficacy, safety, and tolerability of Rozanolixizumab, a humanized monoclonal antibody that binds to the human FcRn receptor, in adults (18 years of age or older) with persistent or chronic primary ITP. The purpose is also to demonstrate the clinical efficacy of Rozanolixizumab as a maintenance therapy. Rozanolixizumab is administered through a subcutaneous injection. Participants must have tried, and responded to, at least one previous ITP-related medication. Participants must also be able to receive immunosuppressive treatments.
Austria, Belgium, Bulgaria, Canada, China, Czechia, France, Germany, Italy, Poland, Russian Federation, Spain, Taiwan, Ukraine, UK, and USA.
https://clinicaltrials.gov/ct2/show/NCT04224688?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=5
Recruiting
Poland - Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia
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
Poland - Long-term Study to Assess the Safety and Efficacy of Efgartimod in Adult Patients with Primary Immune Thrombocytopenia (ITP) (ADVANCE+)
Argenx is conducting this open-label (no placebo) multicenter phase three trial for efgartimod (ARGX-113) in adult ITP patients with primary ITP. Patients must be over the age of 18 years. Administration of efgartimod through IV injection, a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
Belgium, Hungary, Italy, Japan, The Netherlands, Russian Federation, Spain, Ukraine, USA
Status: Recruiting
Poland - Safety and tolerability of M254 in healthy volunteers and immune thrombocytopenia patients
This is an open-label two phase study through Momenta Pharmaceuticals, Inc., to look at the safety, tolerability, pharmacodynamics/pharmacokinetics of M254 in healthy patients compared to patients with persistent or chronic immune thrombocytopenia (ITP). M254 is a hypersialylated Immunoglobulin G (hsIgG), a compound believed to be much stronger than IVIg alone. This study is looking to recruit participants between the ages of 18-55 years. Participants can expect to receive either M254 or a placebo if they are healthy, and M254 either alone or in conjunction with IVIg if they have ITP. Administration of IVIg may be at the same time or weeks apart.
USA, Belgium, France, Germany, Hungary, Italy, Netherlands, Poland, Spain.
Recruiting
Poland - Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP) (LUNA3)
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, Australia, France, Poland, South Korea, Spain, USA
https://clinicaltrials.gov/ct2/show/NCT04562766
Recruiting
Poland - The Oscotec Clinical Trial called Study of Oral SKI-O-703, SYK Inhibitor, in Patients with Persistent and Chronic ITP
This is a phase two double-blind, randomized, multicenter study through Oscotec Inc. designed to evaluate the efficacy and safety of SKI-0-703, an oral SYK inhibitor. This study is targeting adult ITP patients who have persistent or chronic ITP and have failed to respond adequately to other approved therapies, or who have relapsed following exposure to other approved ITP treatments. SYK inhibitors play important roles in signalling within the immune system, particularly in the adaptive immune system. Inhibition of SYK leads to reduced antibody mediated destruction of platelets. Eligibility includes individuals diagnosed with ITP for at least three months who have a platelet count less than 30,000 µL and are at least 18 years of age.
USA, Greece, Republic of Korea, Poland, Spain
Recruiting
Russia - Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia
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
Russian Federation - A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ADVANCE)
Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
USA, Austria, Belgium, Bulgaria, Czech Republic, France, Germany, Georgia, Hungary, Italy, Japan, Netherlands, Poland, Russian Federation, Spain, Turkey, Ukraine, and the United Kingdom.
Recruiting
Russian Federation - A study to evaluate the efficacy, safety, and tolerability of Rozanolixizumab in adult study participants with persistent or chronic primary immune thrombocytopenia (ITP) (myOpportunITy1)
UCB Biopharma SRL is conducting a phase three, multicenter, double-blind, randomized, placebo-controlled study to assess the efficacy, safety, and tolerability of Rozanolixizumab, a humanized monoclonal antibody that binds to the human FcRn receptor, in adults (18 years of age or older) with persistent or chronic primary ITP. The purpose is also to demonstrate the clinical efficacy of Rozanolixizumab as a maintenance therapy. Rozanolixizumab is administered through a subcutaneous injection. Participants must have tried, and responded to, at least one previous ITP-related medication.
Belgium, Bulgaria, France, Georgia, Japan, Moldova, Poland, Russia, Spain, Ukraine, and USA
Currently recruiting
Russian Federation - A study to evaluate the efficacy, safety, and tolerability of Rozanolixizumab in adult study participants with persistent or chronic primary immune thrombocytopenia (ITP) (myOpportunITy2)
UCB Biopharma SRL is conducting a phase three, multicenter, double-blind, randomized, placebo-controlled study to assess the efficacy, safety, and tolerability of Rozanolixizumab, a humanized monoclonal antibody that binds to the human FcRn receptor, in adults (18 years of age or older) with persistent or chronic primary ITP. The purpose is also to demonstrate the clinical efficacy of Rozanolixizumab as a maintenance therapy. Rozanolixizumab is administered through a subcutaneous injection. Participants must have tried, and responded to, at least one previous ITP-related medication. Participants must also be able to receive immunosuppressive treatments.
Austria, Belgium, Bulgaria, Canada, China, Czechia, France, Germany, Italy, Poland, Russian Federation, Spain, Taiwan, Ukraine, UK, and USA.
https://clinicaltrials.gov/ct2/show/NCT04224688?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=5
Recruiting
Russian Federation - Long-term Study to Assess the Safety and Efficacy of Efgartimod in Adult Patients with Primary Immune Thrombocytopenia (ITP) (ADVANCE+)
Argenx is conducting this open-label (no placebo) multicenter phase three trial for efgartimod (ARGX-113) in adult ITP patients with primary ITP. Patients must be over the age of 18 years. Administration of efgartimod through IV injection, a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
Belgium, Hungary, Italy, Japan, The Netherlands, Russian Federation, Spain, Ukraine, USA
Status: Recruiting
Singapore - The International ITP Registry
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.
Recruiting
Slovenia - Study to Evaluate Safety, Tolerability, and Efficacy of TAK-079 in Participants with Persistent/Chronic Primary Immune Thrombocytopenia
Takeda pharmaceuticals is conducting a multi-centered phase two placebo-controlled double-blinded study to look at the safety, tolerability, and efficacy of a drug called TAK-079, which is a monoclonal antibody (anti-CD38 antibody). The trial is for adult ITP patients whose ITP is persistent or chronic, with a platelet count less than 30,000/µL for four consecutive weeks prior to participating in the trial. TAK-079 would be administered as a subcutaneous injection.
Bulgaria, Croatia, Germany, Italy, Slovenia, Ukraine, and USA.
Recruiting
South Korea - H. Pylori Eradication for Moderate ITP
The purpose of this study is to determine the efficacy of H. pylori eradication for the treatment of chronic or persistent immune thrombocytopenic purpura (ITP) patients with moderate thrombocytopenia. H. pylori can lead to increased inflammation that can exacerbate ITP symptoms.
This is a multi-center, open label, prospective randomized phase IIl study.
Republic of Korea
https://clinicaltrials.gov/ct2/show/NCT03177629?recrs=a&cond=ITP&draw=1&rank=20
Status: Unknown
South Korea - Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP) (LUNA3)
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, Australia, France, Poland, South Korea, Spain, USA
https://clinicaltrials.gov/ct2/show/NCT04562766
Recruiting
South Korea - The International ITP Registry
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.
Recruiting
South Korea - The Oscotec Clinical Trial called Study of Oral SKI-O-703, SYK Inhibitor, in Patients with Persistent and Chronic ITP
This is a phase two double-blind, randomized, multicenter study through Oscotec Inc. designed to evaluate the efficacy and safety of SKI-0-703, an oral SYK inhibitor. This study is targeting adult ITP patients who have persistent or chronic ITP and have failed to respond adequately to other approved therapies, or who have relapsed following exposure to other approved ITP treatments. SYK inhibitors play important roles in signalling within the immune system, particularly in the adaptive immune system. Inhibition of SYK leads to reduced antibody mediated destruction of platelets. Eligibility includes individuals diagnosed with ITP for at least three months who have a platelet count less than 30,000 µL and are at least 18 years of age.
USA, Greece, Republic of Korea, Poland, Spain
Recruiting
Spain - A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ADVANCE)
Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
USA, Austria, Belgium, Bulgaria, Czech Republic, France, Germany, Georgia, Hungary, Italy, Japan, Netherlands, Poland, Russian Federation, Spain, Turkey, Ukraine, and the United Kingdom.
Recruiting
Spain - A study to evaluate the efficacy, safety, and tolerability of Rozanolixizumab in adult study participants with persistent or chronic primary immune thrombocytopenia (ITP) (myOpportunITy1)
UCB Biopharma SRL is conducting a phase three, multicenter, double-blind, randomized, placebo-controlled study to assess the efficacy, safety, and tolerability of Rozanolixizumab, a humanized monoclonal antibody that binds to the human FcRn receptor, in adults (18 years of age or older) with persistent or chronic primary ITP. The purpose is also to demonstrate the clinical efficacy of Rozanolixizumab as a maintenance therapy. Rozanolixizumab is administered through a subcutaneous injection. Participants must have tried, and responded to, at least one previous ITP-related medication.
Belgium, Bulgaria, France, Georgia, Japan, Moldova, Poland, Russia, Spain, Ukraine, and USA
Currently recruiting
Spain - A study to evaluate the efficacy, safety, and tolerability of Rozanolixizumab in adult study participants with persistent or chronic primary immune thrombocytopenia (ITP) (myOpportunITy2)
UCB Biopharma SRL is conducting a phase three, multicenter, double-blind, randomized, placebo-controlled study to assess the efficacy, safety, and tolerability of Rozanolixizumab, a humanized monoclonal antibody that binds to the human FcRn receptor, in adults (18 years of age or older) with persistent or chronic primary ITP. The purpose is also to demonstrate the clinical efficacy of Rozanolixizumab as a maintenance therapy. Rozanolixizumab is administered through a subcutaneous injection. Participants must have tried, and responded to, at least one previous ITP-related medication. Participants must also be able to receive immunosuppressive treatments.
Austria, Belgium, Bulgaria, Canada, China, Czechia, France, Germany, Italy, Poland, Russian Federation, Spain, Taiwan, Ukraine, UK, and USA.
https://clinicaltrials.gov/ct2/show/NCT04224688?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=5
Recruiting
Spain - Differential Diagnostic of Immune ThrombocytoPenia (ITP) and Myelodysplastic Syndrome (MDS)
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.
https://clinicaltrials.gov/ct2/show/NCT03469661?recrs=a&cond=ITP&draw=1&rank=15
Active, but no longer recruiting
Spain - Long-term Study to Assess the Safety and Efficacy of Efgartimod in Adult Patients with Primary Immune Thrombocytopenia (ITP) (ADVANCE+)
Argenx is conducting this open-label (no placebo) multicenter phase three trial for efgartimod (ARGX-113) in adult ITP patients with primary ITP. Patients must be over the age of 18 years. Administration of efgartimod through IV injection, a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
Belgium, Hungary, Italy, Japan, The Netherlands, Russian Federation, Spain, Ukraine, USA
Status: Recruiting
Spain - Safety and tolerability of M254 in healthy volunteers and immune thrombocytopenia patients
This is an open-label two phase study through Momenta Pharmaceuticals, Inc., to look at the safety, tolerability, pharmacodynamics/pharmacokinetics of M254 in healthy patients compared to patients with persistent or chronic immune thrombocytopenia (ITP). M254 is a hypersialylated Immunoglobulin G (hsIgG), a compound believed to be much stronger than IVIg alone. This study is looking to recruit participants between the ages of 18-55 years. Participants can expect to receive either M254 or a placebo if they are healthy, and M254 either alone or in conjunction with IVIg if they have ITP. Administration of IVIg may be at the same time or weeks apart.
USA, Belgium, France, Germany, Hungary, Italy, Netherlands, Poland, Spain.
Recruiting
Spain - Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP) (LUNA3)
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, Australia, France, Poland, South Korea, Spain, USA
https://clinicaltrials.gov/ct2/show/NCT04562766
Recruiting
Spain - The Oscotec Clinical Trial called Study of Oral SKI-O-703, SYK Inhibitor, in Patients with Persistent and Chronic ITP
This is a phase two double-blind, randomized, multicenter study through Oscotec Inc. designed to evaluate the efficacy and safety of SKI-0-703, an oral SYK inhibitor. This study is targeting adult ITP patients who have persistent or chronic ITP and have failed to respond adequately to other approved therapies, or who have relapsed following exposure to other approved ITP treatments. SYK inhibitors play important roles in signalling within the immune system, particularly in the adaptive immune system. Inhibition of SYK leads to reduced antibody mediated destruction of platelets. Eligibility includes individuals diagnosed with ITP for at least three months who have a platelet count less than 30,000 µL and are at least 18 years of age.
USA, Greece, Republic of Korea, Poland, Spain
Recruiting
Taiwan - A study to evaluate the efficacy, safety, and tolerability of Rozanolixizumab in adult study participants with persistent or chronic primary immune thrombocytopenia (ITP) (myOpportunITy2)
UCB Biopharma SRL is conducting a phase three, multicenter, double-blind, randomized, placebo-controlled study to assess the efficacy, safety, and tolerability of Rozanolixizumab, a humanized monoclonal antibody that binds to the human FcRn receptor, in adults (18 years of age or older) with persistent or chronic primary ITP. The purpose is also to demonstrate the clinical efficacy of Rozanolixizumab as a maintenance therapy. Rozanolixizumab is administered through a subcutaneous injection. Participants must have tried, and responded to, at least one previous ITP-related medication. Participants must also be able to receive immunosuppressive treatments.
Austria, Belgium, Bulgaria, Canada, China, Czechia, France, Germany, Italy, Poland, Russian Federation, Spain, Taiwan, Ukraine, UK, and USA.
https://clinicaltrials.gov/ct2/show/NCT04224688?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=5
Recruiting
Taiwan - The International ITP Registry
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
Thailand - The International ITP Registry
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
The Combination of Tacrolimus and High-dose Dexamethasone as First-line Treatment in Adult Immune Thrombocytopenia
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
Turkey - A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ADVANCE)
Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
USA, Austria, Belgium, Bulgaria, Czech Republic, France, Germany, Georgia, Hungary, Italy, Japan, Netherlands, Poland, Russian Federation, Spain, Turkey, Ukraine, and the United Kingdom.
Recruiting
Turkey - Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia
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
Turkey - Long-term Study to Assess the Safety and Efficacy of Efgartimod in Adult Patients with Primary Immune Thrombocytopenia (ITP) (ADVANCE+)
Argenx is conducting this open-label (no placebo) multicenter phase three trial for efgartimod (ARGX-113) in adult ITP patients with primary ITP. Patients must be over the age of 18 years. Administration of efgartimod through IV injection, a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
Belgium, Hungary, Italy, Japan, The Netherlands, Russian Federation, Spain, Ukraine, USA
Status: Recruiting
Turkey - The International ITP Registry
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
UK - A Study of PRN1008 in Adult Patients With Immune Thrombocytopenic Purpura (ITP)
Principia Biopharma is conducting a study of their oral drug PRN1008 in patients with ITP who are refractory or relapsed with no available and approved therapeutic options and have a platelet count <30,000/μL on two counts no sooner than 7 days apart in the 15 days before treatment begins. Preclinical findings show that PRN1008 decreases the activity of B cells and other inflammatory-causing immune cells, and in a mouse model of ITP show that PRN1008 can reduce platelet loss.
https://clinicaltrials.gov/ct2/show/NCT03395210?term=PRN1008&rank=1
Recruiting
UK - A Study to Assess Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple- Dose BIVV009 in Participants With Chronic Immune Thrombocytopenia (ITP)
The purpose of this study is to explore the safety, preliminary clinical benefit, and activity of BIVV009 in patients with chronic immune thrombocytopenia. BIVV009 has been designed to block the pathway that triggers the immune system to remove damaged cells. It is believed that this approach may limit the number of cells immune system removes and allow the extra cells to help promote platelet production.
https://clinicaltrials.gov/ct2/show/NCT03275454?recrs=a&cond=ITP&draw=4&rank=30
Active, but no longer recruiting
UK - A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ADVANCE)
Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
USA, Austria, Belgium, Bulgaria, Czech Republic, France, Germany, Georgia, Hungary, Italy, Japan, Netherlands, Poland, Russian Federation, Spain, Turkey, Ukraine, and the United Kingdom.
Recruiting
UK - A study to evaluate the efficacy, safety, and tolerability of Rozanolixizumab in adult study participants with persistent or chronic primary immune thrombocytopenia (ITP) (myOpportunITy1)
UCB Biopharma SRL is conducting a phase three, multicenter, double-blind, randomized, placebo-controlled study to assess the efficacy, safety, and tolerability of Rozanolixizumab, a humanized monoclonal antibody that binds to the human FcRn receptor, in adults (18 years of age or older) with persistent or chronic primary ITP. The purpose is also to demonstrate the clinical efficacy of Rozanolixizumab as a maintenance therapy. Rozanolixizumab is administered through a subcutaneous injection. Participants must have tried, and responded to, at least one previous ITP-related medication.
Belgium, Bulgaria, France, Georgia, Japan, Moldova, Poland, Russia, Spain, Ukraine, and USA
Currently recruiting
UK - A study to evaluate the efficacy, safety, and tolerability of Rozanolixizumab in adult study participants with persistent or chronic primary immune thrombocytopenia (ITP) (myOpportunITy2)
UCB Biopharma SRL is conducting a phase three, multicenter, double-blind, randomized, placebo-controlled study to assess the efficacy, safety, and tolerability of Rozanolixizumab, a humanized monoclonal antibody that binds to the human FcRn receptor, in adults (18 years of age or older) with persistent or chronic primary ITP. The purpose is also to demonstrate the clinical efficacy of Rozanolixizumab as a maintenance therapy. Rozanolixizumab is administered through a subcutaneous injection. Participants must have tried, and responded to, at least one previous ITP-related medication. Participants must also be able to receive immunosuppressive treatments.
Austria, Belgium, Bulgaria, Canada, China, Czechia, France, Germany, Italy, Poland, Russian Federation, Spain, Taiwan, Ukraine, UK, and USA.
https://clinicaltrials.gov/ct2/show/NCT04224688?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=5
Recruiting
UK - Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia
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
Ukraine - A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ADVANCE)
Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
USA, Austria, Belgium, Bulgaria, Czech Republic, France, Germany, Georgia, Hungary, Italy, Japan, Netherlands, Poland, Russian Federation, Spain, Turkey, Ukraine, and the United Kingdom.
Recruiting
Ukraine - A study to evaluate the efficacy, safety, and tolerability of Rozanolixizumab in adult study participants with persistent or chronic primary immune thrombocytopenia (ITP) (myOpportunITy1)
UCB Biopharma SRL is conducting a phase three, multicenter, double-blind, randomized, placebo-controlled study to assess the efficacy, safety, and tolerability of Rozanolixizumab, a humanized monoclonal antibody that binds to the human FcRn receptor, in adults (18 years of age or older) with persistent or chronic primary ITP. The purpose is also to demonstrate the clinical efficacy of Rozanolixizumab as a maintenance therapy. Rozanolixizumab is administered through a subcutaneous injection. Participants must have tried, and responded to, at least one previous ITP-related medication.
Belgium, Bulgaria, France, Georgia, Japan, Moldova, Poland, Russia, Spain, Ukraine, and USA
Currently recruiting
Ukraine - A study to evaluate the efficacy, safety, and tolerability of Rozanolixizumab in adult study participants with persistent or chronic primary immune thrombocytopenia (ITP) (myOpportunITy2)
UCB Biopharma SRL is conducting a phase three, multicenter, double-blind, randomized, placebo-controlled study to assess the efficacy, safety, and tolerability of Rozanolixizumab, a humanized monoclonal antibody that binds to the human FcRn receptor, in adults (18 years of age or older) with persistent or chronic primary ITP. The purpose is also to demonstrate the clinical efficacy of Rozanolixizumab as a maintenance therapy. Rozanolixizumab is administered through a subcutaneous injection. Participants must have tried, and responded to, at least one previous ITP-related medication. Participants must also be able to receive immunosuppressive treatments.
Austria, Belgium, Bulgaria, Canada, China, Czechia, France, Germany, Italy, Poland, Russian Federation, Spain, Taiwan, Ukraine, UK, and USA.
https://clinicaltrials.gov/ct2/show/NCT04224688?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=5
Recruiting
Ukraine - Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia
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
Ukraine - Long-term Study to Assess the Safety and Efficacy of Efgartimod in Adult Patients with Primary Immune Thrombocytopenia (ITP) (ADVANCE+)
Argenx is conducting this open-label (no placebo) multicenter phase three trial for efgartimod (ARGX-113) in adult ITP patients with primary ITP. Patients must be over the age of 18 years. Administration of efgartimod through IV injection, a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
Belgium, Hungary, Italy, Japan, The Netherlands, Russian Federation, Spain, Ukraine, USA
Status: Recruiting
Ukraine - Study to Evaluate Safety, Tolerability, and Efficacy of TAK-079 in Participants with Persistent/Chronic Primary Immune Thrombocytopenia
Takeda pharmaceuticals is conducting a multi-centered phase two placebo-controlled double-blinded study to look at the safety, tolerability, and efficacy of a drug called TAK-079, which is a monoclonal antibody (anti-CD38 antibody). The trial is for adult ITP patients whose ITP is persistent or chronic, with a platelet count less than 30,000/µL for four consecutive weeks prior to participating in the trial. TAK-079 would be administered as a subcutaneous injection.
Bulgaria, Croatia, Germany, Italy, Slovenia, Ukraine, and USA.
Recruiting
USA - A Phase 2a Study Evaluating BIVV020 in Adults With Persistent/Chronic Immune Thrombocytopenia (ITP)
Bioverativ (Sanofi) is conducting a multicentre, Phase 2a, open-label non-randomized study to evaluate the safety, efficacy, and tolerability of BIVV020, also known as Sutimlimab, on the durability of platelet response in individuals who have had ITP for longer than three months. Participants must be at least 18 years of age and have a confirmed diagnosis of primary ITP. For participants who were included in the first trial for this drug, they must have had a response to BIVV020 (platelet count must have increased over 30,000 µL) in order for them to continue on into this next phase. For participants who have never received this drug, there platelet count must be less than 30,000 µL and have demonstrated a lack of response to at least two other ITP treatments. If rituximab was last used by the participant, it must have been received at least 12 weeks before the first dose of BIV020.
USA
Recruiting
USA - A Study of PRN1008 in Adult Patients With Immune Thrombocytopenic Purpura (ITP)
Principia Biopharma is conducting a study of their oral drug PRN1008 in patients with ITP who are refractory or relapsed with no available and approved therapeutic options and have a platelet count <30,000/μL on two counts no sooner than 7 days apart in the 15 days before treatment begins. Preclinical findings show that PRN1008 decreases the activity of B cells and other inflammatory-causing immune cells, and in a mouse model of ITP show that PRN1008 can reduce platelet loss.
https://clinicaltrials.gov/ct2/show/NCT03395210?term=PRN1008&rank=1
Recruiting
USA - A Study to Assess Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple- Dose BIVV009 in Participants With Chronic Immune Thrombocytopenia (ITP)
The purpose of this study is to explore the safety, preliminary clinical benefit, and activity of BIVV009 in patients with chronic immune thrombocytopenia. BIVV009 has been designed to block the pathway that triggers the immune system to remove damaged cells. It is believed that this approach may limit the number of cells immune system removes and allow the extra cells to help promote platelet production.
https://clinicaltrials.gov/ct2/show/NCT03275454?recrs=a&cond=ITP&draw=4&rank=30
Active, but no longer recruiting
USA - A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ADVANCE)
Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
USA, Austria, Belgium, Bulgaria, Czech Republic, France, Germany, Georgia, Hungary, Italy, Japan, Netherlands, Poland, Russian Federation, Spain, Turkey, Ukraine, and the United Kingdom.
Recruiting
USA - A study to evaluate the efficacy, safety, and tolerability of Rozanolixizumab in adult study participants with persistent or chronic primary immune thrombocytopenia (ITP) (myOpportunITy1)
UCB Biopharma SRL is conducting a phase three, multicenter, double-blind, randomized, placebo-controlled study to assess the efficacy, safety, and tolerability of Rozanolixizumab, a humanized monoclonal antibody that binds to the human FcRn receptor, in adults (18 years of age or older) with persistent or chronic primary ITP. The purpose is also to demonstrate the clinical efficacy of Rozanolixizumab as a maintenance therapy. Rozanolixizumab is administered through a subcutaneous injection. Participants must have tried, and responded to, at least one previous ITP-related medication.
Belgium, Bulgaria, France, Georgia, Japan, Moldova, Poland, Russia, Spain, Ukraine, and USA
Currently recruiting
USA - A study to evaluate the efficacy, safety, and tolerability of Rozanolixizumab in adult study participants with persistent or chronic primary immune thrombocytopenia (ITP) (myOpportunITy2)
UCB Biopharma SRL is conducting a phase three, multicenter, double-blind, randomized, placebo-controlled study to assess the efficacy, safety, and tolerability of Rozanolixizumab, a humanized monoclonal antibody that binds to the human FcRn receptor, in adults (18 years of age or older) with persistent or chronic primary ITP. The purpose is also to demonstrate the clinical efficacy of Rozanolixizumab as a maintenance therapy. Rozanolixizumab is administered through a subcutaneous injection. Participants must have tried, and responded to, at least one previous ITP-related medication. Participants must also be able to receive immunosuppressive treatments.
Austria, Belgium, Bulgaria, Canada, China, Czechia, France, Germany, Italy, Poland, Russian Federation, Spain, Taiwan, Ukraine, UK, and USA.
https://clinicaltrials.gov/ct2/show/NCT04224688?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=5
Recruiting
USA - A Study to Evalute the Efficacy and Safety of Efgartigimod PH20 Subcutaneous in Adult Patients with Primary Immune Thrombocytopenia (ADVANCE SC)
Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), PH20 administered subcutaneously. This is a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
USA
Recruiting
USA - Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia
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
USA - Eltrombopag vs standard front-line management for newly diagnosed immune thrombocytopenia (ITP) in children
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
USA - Long-term Study to Assess the Safety and Efficacy of Efgartimod in Adult Patients with Primary Immune Thrombocytopenia (ITP) (ADVANCE+)
Argenx is conducting this open-label (no placebo) multicenter phase three trial for efgartimod (ARGX-113) in adult ITP patients with primary ITP. Patients must be over the age of 18 years. Administration of efgartimod through IV injection, a specially formulated antibody depletion therapy which is a human IgG Fc fragment that targets and binds to the neonatal Fc receptor (FcRn) to increase IgG clearance and block IgG recycling in the body. IgG antibodies accelerate platelet clearance and destruction, and also interfere with proper platelet production. Eligibility is restricted to adult patients 18 years of age or older who have persistent or chronic ITP and a platelet count lower than 30 x 109/L.
Belgium, Hungary, Italy, Japan, The Netherlands, Russian Federation, Spain, Ukraine, USA
Status: Recruiting
USA - Open-label Study Measuring Safety and Treatment Satisfaction in Adult Subjects with Chronic ITP After Switching to Avatrombopag From Eltrombopag or Romiplostim
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
USA - Safety and tolerability of M254 in healthy volunteers and immune thrombocytopenia patients
This is an open-label two phase study through Momenta Pharmaceuticals, Inc., to look at the safety, tolerability, pharmacodynamics/pharmacokinetics of M254 in healthy patients compared to patients with persistent or chronic immune thrombocytopenia (ITP). M254 is a hypersialylated Immunoglobulin G (hsIgG), a compound believed to be much stronger than IVIg alone. This study is looking to recruit participants between the ages of 18-55 years. Participants can expect to receive either M254 or a placebo if they are healthy, and M254 either alone or in conjunction with IVIg if they have ITP. Administration of IVIg may be at the same time or weeks apart.
USA, Belgium, France, Germany, Hungary, Italy, Netherlands, Poland, Spain.
Recruiting
USA - Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP) (LUNA3)
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, Australia, France, Poland, South Korea, Spain, USA
https://clinicaltrials.gov/ct2/show/NCT04562766
Recruiting
USA - Study to Evaluate Safety, Tolerability, and Efficacy of TAK-079 in Participants with Persistent/Chronic Primary Immune Thrombocytopenia
Takeda pharmaceuticals is conducting a multi-centered phase two placebo-controlled double-blinded study to look at the safety, tolerability, and efficacy of a drug called TAK-079, which is a monoclonal antibody (anti-CD38 antibody). The trial is for adult ITP patients whose ITP is persistent or chronic, with a platelet count less than 30,000/µL for four consecutive weeks prior to participating in the trial. TAK-079 would be administered as a subcutaneous injection.
Bulgaria, Croatia, Germany, Italy, Slovenia, Ukraine, and USA.
Recruiting
USA - Study to Evaluate the Efficacy and Safety of PANZYGA in Pediatric Patients With Chronic Immune Thrombocytopenia (ITP)
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
USA - The Oscotec Clinical Trial called Study of Oral SKI-O-703, SYK Inhibitor, in Patients with Persistent and Chronic ITP
This is a phase two double-blind, randomized, multicenter study through Oscotec Inc. designed to evaluate the efficacy and safety of SKI-0-703, an oral SYK inhibitor. This study is targeting adult ITP patients who have persistent or chronic ITP and have failed to respond adequately to other approved therapies, or who have relapsed following exposure to other approved ITP treatments. SYK inhibitors play important roles in signalling within the immune system, particularly in the adaptive immune system. Inhibition of SYK leads to reduced antibody mediated destruction of platelets. Eligibility includes individuals diagnosed with ITP for at least three months who have a platelet count less than 30,000 µL and are at least 18 years of age.
USA, Greece, Republic of Korea, Poland, Spain
Recruiting