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.

Argentina

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

Recruiting

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

AUSTRALIA

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

Recruiting

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

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

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

Recruiting

This is an open-label, phase 2, prospective single-armed study through Novartis Pharmaceuticals. The goal of this study is to assess the ability of eltrombopag to induce a sustained remission (defined as a platelet count at a minimum of 100K microliters) in patients with ITP who have failed previous steroid treatment, or relapsed after previous steroid treatment. Participants can expect to receive eltrombopag for two months and then be tapered off. To be eligible, participants with ITP must be adults, with a platelet count under 30K microliters.

USA, Austria, Brazil, France, Chile, Greece, Italy, Japan, Oman, Russian Federation, Spain, Switzerland, Turkey, United Kingdom.

https://www.clinicaltrials.gov/ct2/show/NCT03524612?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=56

Recruiting

This Phase 2 trial is underway to determine the safety, efficacy, tolerability and Pharmacokinetics of ARGX-113 in patients with primary immune thrombocytopenia. The primary focus of this study is to determine if this treatment lowers incidence and severity of serious adverse events. ARGX-113, Efgartigimod, works by degrading circulating disease-causing autoimmune antibodies.

United Kingdom, Austria, Belgium, France, Germany, Hungary, Poland, Spain, Ukraine, Czechia

https://clinicaltrials.gov/ct2/show/NCT03102593?term=ARGX-113&recrs=a&rank=2

Recruiting

Pfizer is conducting a phase one, randomized, double-blind, placebo controlled human trial to evaluate the safety, tolerability, and pharmacokinetics of PF-06755347, a recombinant fragment crystallisable (rFc), in healthy adult male patients between 18-40 years of age. Male participants much 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.

Belgium

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

Recruiting

This Phase 2 trial is underway to determine the safety, efficacy, tolerability and Pharmacokinetics of ARGX-113 in patients with primary immune thrombocytopenia. The primary focus of this study is to determine if this treatment lowers incidence and severity of serious adverse events. ARGX-113, Efgartigimod, works by degrading circulating disease-causing autoimmune antibodies.

United Kingdom, Austria, Belgium, France, Germany, Hungary, Poland, Spain, Ukraine, Czechia

https://clinicaltrials.gov/ct2/show/NCT03102593?term=ARGX-113&recrs=a&rank=2

Recruiting

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.

https://www.clinicaltrials.gov/ct2/show/NCT03866577?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=42

Recruiting

This is an open-label, phase 2, prospective single-armed study through Novartis Pharmaceuticals. The goal of this study is to assess the ability of eltrombopag to induce a sustained remission (defined as a platelet count at a minimum of 100K microliters) in patients with ITP who have failed previous steroid treatment, or relapsed after previous steroid treatment. Participants can expect to receive eltrombopag for two months and then be tapered off. To be eligible, participants with ITP must be adults, with a platelet count under 30K microliters.

USA, Austria, Brazil, France, Chile, Greece, Italy, Japan, Oman, Russian Federation, Spain, Switzerland, Turkey, United Kingdom.

https://www.clinicaltrials.gov/ct2/show/NCT03524612?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=56

Recruiting

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

BULGARIA

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

Recruiting

Idiopathic thrombocytopenia purpura (ITP) is the destruction of platelets in the body. Autoimmune hemolytic anemia (AIHA) is a rare disease characterized by the body’s premature destruction of red blood cells (hemolysis). Both of these conditions are autoimmune disorders, which means that the body’s immune system malfunctions and attacks healthy blood cells. These two disorders can occur at the same time (destruction of both red blood cells and platelets) or one disorder develops after the other. Evans syndrome is the combination of AIHA with ITP. The cause of Evans syndrome is currently unknown.
A Phase 2 clinical study of an investigational drug is seeking AIHA patients who meet certain criteria.

USA, Canada

https://clinicaltrials.gov/ct2/show/study/NCT02612558

Recruiting

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

CANADA

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

Recruiting

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.

Canada

https://clinicaltrials.gov/ct2/show/NCT03520049?recrs=a&cond=ITP&draw=4&rank=24

Recruiting

This is an open-label, phase 2, prospective single-armed study through Novartis Pharmaceuticals. The goal of this study is to assess the ability of eltrombopag to induce a sustained remission (defined as a platelet count at a minimum of 100K microliters) in patients with ITP who have failed previous steroid treatment, or relapsed after previous steroid treatment. Participants can expect to receive eltrombopag for two months and then be tapered off. To be eligible, participants with ITP must be adults, with a platelet count under 30K microliters.

USA, Austria, Brazil, France, Chile, Greece, Italy, Japan, Oman, Russian Federation, Spain, Switzerland, Turkey, United Kingdom.

https://www.clinicaltrials.gov/ct2/show/NCT03524612?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=56

Recruiting

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

China

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

Recruiting

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

China

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

Recruiting

This prospective case-controlled study conducted through the Institute of Hematology & Blood Diseases Hospital in China is looking to enrol 30 eligible adult patients with ITP who wish to pursue a splenectomy as their next treatment option. The goal of this study is to determine how the immune system in ITP patients benefit from a splenectomy by comparing between those who have had a splenectomy as a result of their ITP with controls (participants who are having a splenectomy for non-ITP reasons). Eligibility includes adults with chronic ITP between the ages of 18-60 years who have failed to maintain a satisfactory platelet response using first and second line therapies. Participants will be offered a splenectomy (provided a safe enough platelet count level can be achieved pre-surgery). Peripheral blood (PB) platelet counts will be collected at various time points during and after the surgery and compared with the control group. Tissue from the spleen will also be analyzed from all enrolled.

China

https://www.clinicaltrials.gov/ct2/show/NCT03998059?recrs=ab&cond=Immune+Thrombocytopenia&draw=2&rank=38

Recruiting

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

China

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

Recruiting

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

China

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

Recruiting

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

China

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

Recruiting

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

China

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

Recruiting

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

China

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

Recruiting

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

China

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

Recruiting

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

China

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

Recruiting

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

China

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

Recruiting

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

China

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

Recruiting

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

CZECH REPUBLIC

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

Recruiting

This Phase 2 trial is underway to determine the safety, efficacy, tolerability and Pharmacokinetics of ARGX-113 in patients with primary immune thrombocytopenia. The primary focus of this study is to determine if this treatment lowers incidence and severity of serious adverse events. ARGX-113, Efgartigimod, works by degrading circulating disease-causing autoimmune antibodies.

United Kingdom, Austria, Belgium, France, Germany, Hungary, Poland, Spain, Ukraine, Czechia

https://clinicaltrials.gov/ct2/show/NCT03102593?term=ARGX-113&recrs=a&rank=2

Recruiting

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

Egypt

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

Recruiting

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

This is an open-label, phase 2, prospective single-armed study through Novartis Pharmaceuticals. The goal of this study is to assess the ability of eltrombopag to induce a sustained remission (defined as a platelet count at a minimum of 100K microliters) in patients with ITP who have failed previous steroid treatment, or relapsed after previous steroid treatment. Participants can expect to receive eltrombopag for two months and then be tapered off. To be eligible, participants with ITP must be adults, with a platelet count under 30K microliters.

USA, Austria, Brazil, France, Chile, Greece, Italy, Japan, Oman, Russian Federation, Spain, Switzerland, Turkey, United Kingdom.

https://www.clinicaltrials.gov/ct2/show/NCT03524612?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=56

Recruiting

This Phase 2 trial is underway to determine the safety, efficacy, tolerability and Pharmacokinetics of ARGX-113 in patients with primary immune thrombocytopenia. The primary focus of this study is to determine if this treatment lowers incidence and severity of serious adverse events. ARGX-113, Efgartigimod, works by degrading circulating disease-causing autoimmune antibodies.

United Kingdom, Austria, Belgium, France, Germany, Hungary, Poland, Spain, Ukraine, Czechia

https://clinicaltrials.gov/ct2/show/NCT03102593?term=ARGX-113&recrs=a&rank=2

Recruiting

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

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

France

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

Recruiting

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

France

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

Recruiting

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

France

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

Recruiting

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

France

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

Recruiting

TRANSREG will assess the safety and biological efficacy of low-dose IL2 as a Treg inducer in a set of 14 autoimmune and auto-inflammatory diseases, with the aim to select diseases in which further therapeutic development will be performed. IL2 is a cytokine signaling molecule that can limit immune suppression.

France

https://clinicaltrials.gov/ct2/show/NCT01988506?recrs=a&cond=ITP&draw=2&rank=41

Recruiting

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

France

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

Recruiting

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

https://www.clinicaltrials.gov/ct2/show/NCT03820960?recrs=ab&cond=Immune+Thrombocytopenia&draw=2&rank=5

Recruiting

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

Recruiting

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.

https://www.clinicaltrials.gov/ct2/show/NCT03866577?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=42

Recruiting

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.

Republic of Moldova, Georgia

Recruiting on hold

https://www.clinicaltrials.gov/ct2/show/NCT04200456?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=5

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.

USA, UK, Germany

https://clinicaltrials.gov/ct2/show/NCT03275454?recrs=a&cond=ITP&draw=4&rank=30

Recruiting

This Phase 2 trial is underway to determine the safety, efficacy, tolerability and Pharmacokinetics of ARGX-113 in patients with primary immune thrombocytopenia. The primary focus of this study is to determine if this treatment lowers incidence and severity of serious adverse events. ARGX-113, Efgartigimod, works by degrading circulating disease-causing autoimmune antibodies.

United Kingdom, Austria, Belgium, France, Germany, Hungary, Poland, Spain, Ukraine, Czechia

https://clinicaltrials.gov/ct2/show/NCT03102593?term=ARGX-113&recrs=a&rank=2

Recruiting

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.

https://www.clinicaltrials.gov/ct2/show/NCT03866577?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=42

Recruiting

This is an open-label, phase 2, prospective single-armed study through Novartis Pharmaceuticals. The goal of this study is to assess the ability of eltrombopag to induce a sustained remission (defined as a platelet count at a minimum of 100K microliters) in patients with ITP who have failed previous steroid treatment, or relapsed after previous steroid treatment. Participants can expect to receive eltrombopag for two months and then be tapered off. To be eligible, participants with ITP must be adults, with a platelet count under 30K microliters.

USA, Austria, Brazil, France, Chile, Greece, Italy, Japan, Oman, Russian Federation, Spain, Switzerland, Turkey, United Kingdom.

https://www.clinicaltrials.gov/ct2/show/NCT03524612?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=56

Recruiting

Sponsored by Oscotec Inc., this is a phase 2, multicenter, randomized, double-blind, placebo-controlled, parallel dose study to evaluate the efficacy and safety of SKI-0-703, and SYK inhibitor, in patient’s with persistent and chronic ITP who have failed to respond (or relapsed after trying) other treatments. Patients in this study must have a platelet count less than 30,000μL on two separate occasion as at least 7 days apart. Participants will take part in three treatment groups for twenty weeks.

USA, Greece, Poland, Spain, Republic of Korea.

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

Recruiting

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

Hong Kong

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

Recruiting

This Phase 2 trial is underway to determine the safety, efficacy, tolerability and Pharmacokinetics of ARGX-113 in patients with primary immune thrombocytopenia. The primary focus of this study is to determine if this treatment lowers incidence and severity of serious adverse events. ARGX-113, Efgartigimod, works by degrading circulating disease-causing autoimmune antibodies.

United Kingdom, Austria, Belgium, France, Germany, Hungary, Poland, Spain, Ukraine, Czechia

https://clinicaltrials.gov/ct2/show/NCT03102593?term=ARGX-113&recrs=a&rank=2

Recruiting

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.

https://www.clinicaltrials.gov/ct2/show/NCT03866577?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=42

Recruiting

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

Italy

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

Complete

This study led by Kezar Life Sciences is a phase two, randomized, dose-blinded, multicenter study to evaluate the safety and efficacy of KZR-616 (a selective immunoproteasome inhibitor) in the treatment of patients with autoimmune hemolytic anemia (AIHA) and immune thrombocytopenia (ITP). KZR-616 has been shown in earlier analysis to have a positive effect on taming the inflammatory response in other autoimmune conditions in animal models, without causing immunosuppression. Study participants must be over the age of 18 years. For ITP patients specifically, eligibility is restricted to those who have a platelet count under 30,000μL on two separate blood draws over a week apart, or for ITP patients who are receiving on-going treatment and have a platelet count less than 50,000μL.

This study has multiple phases. Other active trials looking at the safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of treatment with KZR-616 are now active for the following autoimmune diseases: lupus (MISSION study: https://clinicaltrials.gov/ct2/show/NCT03393013), dermatomyositis and polymyositis (PRESIDIO study: https://clinicaltrials.gov/ct2/show/NCT04033926).

USA, Italy, Poland, Russia

https://clinicaltrials.gov/ct2/show/NCT04039477

Recruiting

This is an open-label, phase 2, prospective single-armed study through Novartis Pharmaceuticals. The goal of this study is to assess the ability of eltrombopag to induce a sustained remission (defined as a platelet count at a minimum of 100K microliters) in patients with ITP who have failed previous steroid treatment, or relapsed after previous steroid treatment. Participants can expect to receive eltrombopag for two months and then be tapered off. To be eligible, participants with ITP must be adults, with a platelet count under 30K microliters.

USA, Austria, Brazil, France, Chile, Greece, Italy, Japan, Oman, Russian Federation, Spain, Switzerland, Turkey, United Kingdom.

https://www.clinicaltrials.gov/ct2/show/NCT03524612?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=56

Recruiting

The primary objective of the study is the evaluation of sustained response rate after discontinuation of treatment with TPO-RAs, Eltrombopag and Romiplostim, in persistent or chronic pITP patients who failed one or more therapy lines, splenectomy included. Increasing evidence exists in the literature on the persistent response after TPO-RAs discontinuation.

Italy

https://clinicaltrials.gov/ct2/show/NCT02298075?recrs=a&cond=ITP&draw=1&rank=23

Recruiting

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

Italy

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

Recruiting

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.

https://www.clinicaltrials.gov/ct2/show/NCT03866577?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=42

Recruiting

This is a retrospective case-based study conducted through the Gruppo Italiano Malattie EMatologiche dell'Adulto group looking at the clinical utility and benefit of using thrombopoietin receptor agents (TPO-RAs) as a ‘bridge’ before undergoing a splenectomy. The aim of the present study is also to verify on an Italian national scale the frequency of TPO-RA use and its impact and the safety of using these drugs in adults over the age of 18 years.

Italy

https://www.clinicaltrials.gov/ct2/show/NCT02063763?recrs=ab&cond=Immune+Thrombocytopenia&draw=2&rank=40

Recruiting

This study aims at analyzing the therapeutic activity of TPO-mimetics Eltrombopag and Romiplostim as bridge therapy for splenectomy in adult patients with primary immune thrombocytopenia.

Italy

https://clinicaltrials.gov/ct2/show/NCT02063763?recrs=a&cond=ITP&draw=1&rank=36

Recruiting

Through Kissei Pharmaceutical Co., Ltd, this randomized, phase III study investigate the efficacy, safety, and pharmacokinetics of R788 (also known as Fostamatinib) in adult patients with chronic immune thrombocytopenia. The goal of this study is also to investigate the safety and efficacy of long term dosing of R788 in patients with chronic idiopathic thrombocytopenic purpura. Participants are required to be over the age of 20 years, and have chronic ITP with platelet counts below 30K microliters. Participants must also have failed at least one ITP therapy previously.

Japan

https://www.clinicaltrials.gov/ct2/show/NCT04132050?recrs=ab&cond=Immune+Thrombocytopenia&draw=2&rank=24

Recruiting

This is an open-label, phase 2, prospective single-armed study through Novartis Pharmaceuticals. The goal of this study is to assess the ability of eltrombopag to induce a sustained remission (defined as a platelet count at a minimum of 100K microliters) in patients with ITP who have failed previous steroid treatment, or relapsed after previous steroid treatment. Participants can expect to receive eltrombopag for two months and then be tapered off. To be eligible, participants with ITP must be adults, with a platelet count under 30K microliters.

USA, Austria, Brazil, France, Chile, Greece, Italy, Japan, Oman, Russian Federation, Spain, Switzerland, Turkey, United Kingdom.

https://www.clinicaltrials.gov/ct2/show/NCT03524612?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=56

Recruiting

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

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

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

Recruiting

This 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

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.

Republic of Moldova, Georgia

Recruiting on hold

https://www.clinicaltrials.gov/ct2/show/NCT04200456?recrs=a&cond=Immune+Thrombocytopenia&draw=2&rank=5

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

NETHERLANDS

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

Recruiting

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, Netherlands

https://clinicaltrials.gov/ct2/show/NCT04188379?recrs=a&cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=2

Recruiting

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.

https://www.clinicaltrials.gov/ct2/show/NCT03866577?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=42

Recruiting

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

NORWAY

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

Recruiting

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

Norway

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

Recruiting

This is an open-label, phase 2, prospective single-armed study through Novartis Pharmaceuticals. The goal of this study is to assess the ability of eltrombopag to induce a sustained remission (defined as a platelet count at a minimum of 100K microliters) in patients with ITP who have failed previous steroid treatment, or relapsed after previous steroid treatment. Participants can expect to receive eltrombopag for two months and then be tapered off. To be eligible, participants with ITP must be adults, with a platelet count under 30K microliters.

USA, Austria, Brazil, France, Chile, Greece, Italy, Japan, Oman, Russian Federation, Spain, Switzerland, Turkey, United Kingdom.

https://www.clinicaltrials.gov/ct2/show/NCT03524612?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=56

Recruiting

This study led by Kezar Life Sciences is a phase two, randomized, dose-blinded, multicenter study to evaluate the safety and efficacy of KZR-616 (a selective immunoproteasome inhibitor) in the treatment of patients with autoimmune hemolytic anemia (AIHA) and immune thrombocytopenia (ITP). KZR-616 has been shown in earlier analysis to have a positive effect on taming the inflammatory response in other autoimmune conditions in animal models, without causing immunosuppression. Study participants must be over the age of 18 years. For ITP patients specifically, eligibility is restricted to those who have a platelet count under 30,000μL on two separate blood draws over a week apart, or for ITP patients who are receiving on-going treatment and have a platelet count less than 50,000μL.

This study has multiple phases. Other active trials looking at the safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of treatment with KZR-616 are now active for the following autoimmune diseases: lupus (MISSION study: https://clinicaltrials.gov/ct2/show/NCT03393013), dermatomyositis and polymyositis (PRESIDIO study: https://clinicaltrials.gov/ct2/show/NCT04033926).

USA, Italy, Poland, Russia

https://clinicaltrials.gov/ct2/show/NCT04039477

Recruiting

This Phase 2 trial is underway to determine the safety, efficacy, tolerability and Pharmacokinetics of ARGX-113 in patients with primary immune thrombocytopenia. The primary focus of this study is to determine if this treatment lowers incidence and severity of serious adverse events. ARGX-113, Efgartigimod, works by degrading circulating disease-causing autoimmune antibodies.

United Kingdom, Austria, Belgium, France, Germany, Hungary, Poland, Spain, Ukraine, Czechia

https://clinicaltrials.gov/ct2/show/NCT03102593?term=ARGX-113&recrs=a&rank=2

Recruiting

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.

https://www.clinicaltrials.gov/ct2/show/NCT03866577?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=42

Recruiting

Sponsored by Oscotec Inc., this is a phase 2, multicenter, randomized, double-blind, placebo-controlled, parallel dose study to evaluate the efficacy and safety of SKI-0-703, and SYK inhibitor, in patient’s with persistent and chronic ITP who have failed to respond (or relapsed after trying) other treatments. Patients in this study must have a platelet count less than 30,000μL on two separate occasion as at least 7 days apart. Participants will take part in three treatment groups for twenty weeks.

USA, Greece, Poland, Spain, Republic of Korea.

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

Recruiting

This study led by Kezar Life Sciences is a phase two, randomized, dose-blinded, multicenter study to evaluate the safety and efficacy of KZR-616 (a selective immunoproteasome inhibitor) in the treatment of patients with autoimmune hemolytic anemia (AIHA) and immune thrombocytopenia (ITP). KZR-616 has been shown in earlier analysis to have a positive effect on taming the inflammatory response in other autoimmune conditions in animal models, without causing immunosuppression. Study participants must be over the age of 18 years. For ITP patients specifically, eligibility is restricted to those who have a platelet count under 30,000μL on two separate blood draws over a week apart, or for ITP patients who are receiving on-going treatment and have a platelet count less than 50,000μL.

This study has multiple phases. Other active trials looking at the safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of treatment with KZR-616 are now active for the following autoimmune diseases: lupus (MISSION study: https://clinicaltrials.gov/ct2/show/NCT03393013), dermatomyositis and polymyositis (PRESIDIO study: https://clinicaltrials.gov/ct2/show/NCT04033926).

USA, Italy, Poland, Russia

https://clinicaltrials.gov/ct2/show/NCT04039477

Recruiting

This is an open-label, phase 2, prospective single-armed study through Novartis Pharmaceuticals. The goal of this study is to assess the ability of eltrombopag to induce a sustained remission (defined as a platelet count at a minimum of 100K microliters) in patients with ITP who have failed previous steroid treatment, or relapsed after previous steroid treatment. Participants can expect to receive eltrombopag for two months and then be tapered off. To be eligible, participants with ITP must be adults, with a platelet count under 30K microliters.

USA, Austria, Brazil, France, Chile, Greece, Italy, Japan, Oman, Russian Federation, Spain, Switzerland, Turkey, United Kingdom.

https://www.clinicaltrials.gov/ct2/show/NCT03524612?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=56

Recruiting

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

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

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

Recruiting

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

Recruiting

Sponsored by Oscotec Inc., this is a phase 2, multicenter, randomized, double-blind, placebo-controlled, parallel dose study to evaluate the efficacy and safety of SKI-0-703, and SYK inhibitor, in patient’s with persistent and chronic ITP who have failed to respond (or relapsed after trying) other treatments. Patients in this study must have a platelet count less than 30,000μL on two separate occasion as at least 7 days apart. Participants will take part in three treatment groups for twenty weeks.

USA, Greece, Poland, Spain, Republic of Korea.

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

Recruiting

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

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

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

Recruiting

This is an open-label, phase 2, prospective single-armed study through Novartis Pharmaceuticals. The goal of this study is to assess the ability of eltrombopag to induce a sustained remission (defined as a platelet count at a minimum of 100K microliters) in patients with ITP who have failed previous steroid treatment, or relapsed after previous steroid treatment. Participants can expect to receive eltrombopag for two months and then be tapered off. To be eligible, participants with ITP must be adults, with a platelet count under 30K microliters.

USA, Austria, Brazil, France, Chile, Greece, Italy, Japan, Oman, Russian Federation, Spain, Switzerland, Turkey, United Kingdom.

https://www.clinicaltrials.gov/ct2/show/NCT03524612?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=56

Recruiting

This Phase 2 trial is underway to determine the safety, efficacy, tolerability and Pharmacokinetics of ARGX-113 in patients with primary immune thrombocytopenia. The primary focus of this study is to determine if this treatment lowers incidence and severity of serious adverse events. ARGX-113, Efgartigimod, works by degrading circulating disease-causing autoimmune antibodies.

United Kingdom, Austria, Belgium, France, Germany, Hungary, Poland, Spain, Ukraine, Czechia

https://clinicaltrials.gov/ct2/show/NCT03102593?term=ARGX-113&recrs=a&rank=2

Recruiting

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

Spain

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

Recruiting

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.

https://www.clinicaltrials.gov/ct2/show/NCT03866577?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=42

Recruiting

Sponsored by Oscotec Inc., this is a phase 2, multicenter, randomized, double-blind, placebo-controlled, parallel dose study to evaluate the efficacy and safety of SKI-0-703, and SYK inhibitor, in patient’s with persistent and chronic ITP who have failed to respond (or relapsed after trying) other treatments. Patients in this study must have a platelet count less than 30,000μL on two separate occasion as at least 7 days apart. Participants will take part in three treatment groups for twenty weeks.

USA, Greece, Poland, Spain, Republic of Korea.

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

Recruiting

This is an open-label, phase 2, prospective single-armed study through Novartis Pharmaceuticals. The goal of this study is to assess the ability of eltrombopag to induce a sustained remission (defined as a platelet count at a minimum of 100K microliters) in patients with ITP who have failed previous steroid treatment, or relapsed after previous steroid treatment. Participants can expect to receive eltrombopag for two months and then be tapered off. To be eligible, participants with ITP must be adults, with a platelet count under 30K microliters.

USA, Austria, Brazil, France, Chile, Greece, Italy, Japan, Oman, Russian Federation, Spain, Switzerland, Turkey, United Kingdom.

https://www.clinicaltrials.gov/ct2/show/NCT03524612?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=56

Recruiting

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

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

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

Recruiting

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

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

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

Recruiting

This is an open-label, phase 2, prospective single-armed study through Novartis Pharmaceuticals. The goal of this study is to assess the ability of eltrombopag to induce a sustained remission (defined as a platelet count at a minimum of 100K microliters) in patients with ITP who have failed previous steroid treatment, or relapsed after previous steroid treatment. Participants can expect to receive eltrombopag for two months and then be tapered off. To be eligible, participants with ITP must be adults, with a platelet count under 30K microliters.

USA, Austria, Brazil, France, Chile, Greece, Italy, Japan, Oman, Russian Federation, Spain, Switzerland, Turkey, United Kingdom.

https://www.clinicaltrials.gov/ct2/show/NCT03524612?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=56

Recruiting

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

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

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

Recruiting

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

UNITED KINGDOM

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

Recruiting

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.

USA, UK, Germany

https://clinicaltrials.gov/ct2/show/NCT03275454?recrs=a&cond=ITP&draw=4&rank=30

Recruiting

This is an open-label, phase 2, prospective single-armed study through Novartis Pharmaceuticals. The goal of this study is to assess the ability of eltrombopag to induce a sustained remission (defined as a platelet count at a minimum of 100K microliters) in patients with ITP who have failed previous steroid treatment, or relapsed after previous steroid treatment. Participants can expect to receive eltrombopag for two months and then be tapered off. To be eligible, participants with ITP must be adults, with a platelet count under 30K microliters.

USA, Austria, Brazil, France, Chile, Greece, Italy, Japan, Oman, Russian Federation, Spain, Switzerland, Turkey, United Kingdom.

https://www.clinicaltrials.gov/ct2/show/NCT03524612?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=56

Recruiting

This Phase 2 trial is underway to determine the safety, efficacy, tolerability and Pharmacokinetics of ARGX-113 in patients with primary immune thrombocytopenia. The primary focus of this study is to determine if this treatment lowers incidence and severity of serious adverse events. ARGX-113, Efgartigimod, works by degrading circulating disease-causing autoimmune antibodies.

United Kingdom, Austria, Belgium, France, Germany, Hungary, Poland, Spain, Ukraine, Czechia

https://clinicaltrials.gov/ct2/show/NCT03102593?term=ARGX-113&recrs=a&rank=2

Recruiting

This Phase 2 trial is underway to determine the safety, efficacy, tolerability and Pharmacokinetics of ARGX-113 in patients with primary immune thrombocytopenia. The primary focus of this study is to determine if this treatment lowers incidence and severity of serious adverse events. ARGX-113, Efgartigimod, works by degrading circulating disease-causing autoimmune antibodies.

United Kingdom, Austria, Belgium, France, Germany, Hungary, Poland, Spain, Ukraine, Czechia

https://clinicaltrials.gov/ct2/show/NCT03102593?term=ARGX-113&recrs=a&rank=2

Recruiting

This study led by Kezar Life Sciences is a phase two, randomized, dose-blinded, multicenter study to evaluate the safety and efficacy of KZR-616 (a selective immunoproteasome inhibitor) in the treatment of patients with autoimmune hemolytic anemia (AIHA) and immune thrombocytopenia (ITP). KZR-616 has been shown in earlier analysis to have a positive effect on taming the inflammatory response in other autoimmune conditions in animal models, without causing immunosuppression. Study participants must be over the age of 18 years. For ITP patients specifically, eligibility is restricted to those who have a platelet count under 30,000μL on two separate blood draws over a week apart, or for ITP patients who are receiving on-going treatment and have a platelet count less than 50,000μL.

This study has multiple phases. Other active trials looking at the safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of treatment with KZR-616 are now active for the following autoimmune diseases: lupus (MISSION study: https://clinicaltrials.gov/ct2/show/NCT03393013), dermatomyositis and polymyositis (PRESIDIO study: https://clinicaltrials.gov/ct2/show/NCT04033926).

USA, Italy, Poland, Russia

https://clinicaltrials.gov/ct2/show/NCT04039477

Recruiting

Idiopathic thrombocytopenia purpura (ITP) is the destruction of platelets in the body. Autoimmune hemolytic anemia (AIHA) is a rare disease characterized by the body’s premature destruction of red blood cells (hemolysis). Both of these conditions are autoimmune disorders, which means that the body’s immune system malfunctions and attacks healthy blood cells. These two disorders can occur at the same time (destruction of both red blood cells and platelets) or one disorder develops after the other. Evans syndrome is the combination of AIHA with ITP. The cause of Evans syndrome is currently unknown.

A Phase 2 clinical study of an investigational drug is seeking AIHA patients who meet certain criteria.

USA, Canada

https://clinicaltrials.gov/ct2/show/study/NCT02612558

Recruiting

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

USA

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

Recruiting

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.

USA

https://clinicaltrials.gov/ct2/show/NCT03275454?recrs=a&cond=ITP&draw=4&rank=30

Recruiting

This is an open-label, phase 2, prospective single-armed study through Novartis Pharmaceuticals. The goal of this study is to assess the ability of eltrombopag to induce a sustained remission (defined as a platelet count at a minimum of 100K microliters) in patients with ITP who have failed previous steroid treatment, or relapsed after previous steroid treatment. Participants can expect to receive eltrombopag for two months and then be tapered off. To be eligible, participants with ITP must be adults, with a platelet count under 30K microliters.

USA, Austria, Brazil, France, Chile, Greece, Italy, Japan, Oman, Russian Federation, Spain, Switzerland, Turkey, United Kingdom.

https://www.clinicaltrials.gov/ct2/show/NCT03524612?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=56

Recruiting

Argenx BVBA is conducting a phase 3, multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of efgartigimod (ARGX 113), 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, Netherlands

https://clinicaltrials.gov/ct2/show/NCT04188379?recrs=a&cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=2

Recruiting

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

USA

Recruiting

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

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.

https://www.clinicaltrials.gov/ct2/show/NCT03866577?cond=Immune+Thrombocytopenia&cntry=US&draw=2&rank=42

Recruiting

Sponsored by Oscotec Inc., this is a phase 2, multicenter, randomized, double-blind, placebo-controlled, parallel dose study to evaluate the efficacy and safety of SKI-0-703, and SYK inhibitor, in patient’s with persistent and chronic ITP who have failed to respond (or relapsed after trying) other treatments. Patients in this study must have a platelet count less than 30,000μL on two separate occasion as at least 7 days apart. Participants will take part in three treatment groups for twenty weeks.

USA, Greece, Poland, Spain, Republic of Korea.

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

Recruiting

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

USA

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

Recruiting