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Ongoing Clinical Trials for Immune Thrombocytopenia

This article provides information about ongoing clinical trials for immune thrombocytopenia, a blood disorder where the immune system attacks platelets. Currently, there are 18 active trials testing various treatments across multiple European countries, offering patients opportunities to access new therapies and contribute to medical research. (Also known as: ITP, Primary Immune Thrombocytopenia, Idiopathic Thrombocytopenic Purpura)

Clinical trial locations

Long-term Study on the Safety and Efficacy of Efgartigimod in Adults with Primary Immune Thrombocytopenia

This trial is investigating the long-term safety and effectiveness of efgartigimod, a treatment given through intravenous infusion, in adults with primary immune thrombocytopenia. The medication, also known as ARGX-113, is administered at a dose of 10 mg per kilogram of body weight.

Who can participate: Eligible participants must have completed a previous 24-week trial period and be able to understand and provide written consent. Women of childbearing age need a negative pregnancy test before starting and must use reliable birth control during the trial and for 90 days after the last dose.

Who cannot participate: The trial excludes people with conditions other than primary immune thrombocytopenia, those under 18 years of age, and individuals who cannot provide informed consent.

Main focus: The study primarily evaluates the long-term safety of efgartigimod by tracking adverse effects, vital signs, and laboratory results over 52 weeks, with a possible additional 52-week extension if the medication is not yet commercially available. Secondary objectives include monitoring platelet counts and assessing the need for additional treatments to control the disease.

Treatment being tested: Efgartigimod works by targeting and reducing the activity of certain antibodies that contribute to the destruction of platelets in the blood. It belongs to a class of medications called immunomodulatory agents.

Study Comparing Avatrombopag and Rituximab for Adults with Immune Thrombocytopenia Not Responding to Corticosteroids

This Norwegian trial compares two treatment approaches for adults who have not responded well to corticosteroids, the initial treatment for immune thrombocytopenia. One group receives avatrombopag tablets taken orally, while the other receives rituximab given through intravenous infusion.

Who can participate: Adults aged 18 or older with a diagnosis of primary immune thrombocytopenia for less than one year are eligible. Participants must have a platelet count below 30,000 per microliter, measured within two weeks before joining the study. They should have previously tried corticosteroid treatment without adequate response or with relapse, and need further treatment as determined by their doctor.

Who cannot participate: The study excludes people who did not respond to a short course of corticosteroids, those under 18 years of age, and vulnerable populations.

Main focus: The study aims to determine which treatment better maintains healthy platelet counts without additional medications. Regular monitoring occurs bi-weekly between weeks 20 and 28, with assessments of platelet counts, quality of life, and any treatment failures or adverse events extending to 78 weeks.

Treatments being tested: Avatrombopag stimulates platelet production by activating the thrombopoietin receptor and is classified as a thrombopoietin receptor agonist. Rituximab targets and depletes B-cells, reducing the immune system’s attack on platelets, and is classified as a monoclonal antibody.

Study comparing dexamethasone versus immunoglobulin plus prednisone for adults with immune thrombocytopenia (ITP) who have moderate to severe bleeding

This French study compares two treatment combinations for adults experiencing moderate to severe bleeding symptoms. One group receives intravenous immunoglobulin combined with oral prednisone for three weeks, while the other takes high-dose dexamethasone tablets for four days.

Who can participate: Adults between 18 and 80 years old with newly diagnosed or recurring immune thrombocytopenia are eligible. Participants must have a platelet count at or below 20,000 per microliter with visible bleeding on the skin or mucous membranes, be registered with social security, and able to provide written informed consent.

Who cannot participate: Exclusions include people under 18, those with active infections, uncontrolled high blood pressure, active peptic ulcers, uncontrolled diabetes, known allergies to the study medications, pregnant or breastfeeding women, severe liver or kidney disease, active cancer requiring treatment, participation in other trials, inability to consent, history of blood clotting disorders, or severe cardiovascular disease.

Main focus: The study tracks how quickly patients respond during the first five days, monitoring platelet count improvements and bleeding reduction. Long-term effectiveness is evaluated over six months, with regular platelet counts and bleeding assessments.

Treatments being tested: Intravenous immunoglobulin contains pooled antibodies that block platelet destruction. Prednisone and dexamethasone are corticosteroids that suppress immune system activity and reduce inflammation, helping increase platelet counts. Dexamethasone is more potent and given for a shorter duration.

Study of NVG-2089 given by intravenous infusion in patients with immune thrombocytopenia to evaluate safety and effectiveness

This trial, conducted in Poland, Spain, and Greece, tests NVG-2089, a new medication given through intravenous infusion alongside standard immunoglobulin treatment. The study uses gradually increasing doses to understand patient response.

Who can participate: Adults aged 18 to 80 with primary immune thrombocytopenia lasting more than 3 months (but less than 12 months) or more than 12 months are eligible. Participants must have responded to at least one previous treatment by achieving a platelet count of at least 50,000 cells per cubic millimeter. Current platelet counts must be between 20,000 and 50,000 cells per cubic millimeter with either no symptoms or minor bleeding. Those previously treated with intravenous immunoglobulin must have shown a positive response. Women of childbearing age must have negative pregnancy tests and use two forms of birth control, while men with partners who can become pregnant must use barrier birth control.

Who cannot participate: Exclusions include pregnancy, breastfeeding, planning pregnancy, severe allergic reactions to medications or vaccines, active bleeding or clotting disorders besides ITP, major surgery within 3 months, participation in other trials within 30 days, serious heart/liver/kidney problems, active infections, use of blood thinners, blood cancer or other cancers in the past 5 years, uncontrolled high blood pressure, substance abuse within the past year, and mental health conditions affecting participation.

Main focus: The study evaluates safety and tolerability while monitoring platelet count changes throughout the treatment period until December 2026. Success is defined differently based on initial platelet counts: if starting below 30,000, success means doubling the count or reaching 50,000 or higher; if starting at 30,000 or higher, success means an increase of 20,000 or reaching 80,000 or higher.

Treatment being tested: NVG-2089 is an investigational medication administered intravenously, being tested at different dose levels to understand its safety and effectiveness. While its exact mechanism is still under investigation, it is being developed to address the autoimmune component by potentially modulating the immune response leading to platelet destruction.

Study of rituximab and belimumab combination compared to rituximab alone in adults with persistent or chronic immune thrombocytopenia (ITP)

This French trial evaluates whether combining rituximab with weekly belimumab injections works better than rituximab with placebo in treating persistent or chronic immune thrombocytopenia over 104 weeks.

Who can participate: Adults aged 18 or older who have temporarily responded to corticosteroids or intravenous immunoglobulin but experienced relapse are eligible. Participants must have current platelet counts of 30,000 per microliter or lower within the past month, or below 50,000 if bleeding is present, and have been diagnosed between 2 months and 10 years ago. Those over 60 need normal bone marrow tests. Women who can become pregnant need negative pregnancy tests and reliable birth control. Participants must have completed full COVID-19 vaccination and have gammaglobulin levels of 7 grams per liter or higher.

Who cannot participate: Exclusions include age below 18 or above 65, previous unsuccessful rituximab treatment, active or chronic infections including hepatitis B/C or HIV, pregnancy, breastfeeding, blood clotting disorders other than ITP, active or recent cancer, major surgery within 8 weeks, participation in other trials within 30 days, severe kidney or liver disease, uncontrolled high blood pressure, severe allergic reactions to medications, mental health conditions affecting participation, use of prohibited medications within 4 weeks, or any condition making participation unsafe.

Main focus: The study monitors health at specific timepoints (weeks 6, 12, 24, 36, 52, 78, and 104) with blood tests checking platelet counts and immune function. Quality of life questionnaires are completed at weeks 12, 24, and 52. The study tracks bleeding events, blood protein levels, and hospitalizations requiring treatment for infections.

Treatments being tested: Rituximab is a monoclonal antibody given intravenously at 1,000 mg doses on days 7 and 21, working by binding to CD20 proteins on B lymphocytes to reduce their numbers. Belimumab is injected subcutaneously weekly for 24 weeks at 200 mg, targeting and inhibiting B-lymphocyte stimulator protein to reduce abnormal immune activity.

Study on the Effectiveness and Safety of Efgartigimod IV for Adults with Primary Immune Thrombocytopenia

This large European trial, conducted across 14 countries including Romania, Italy, Bulgaria, Poland, Croatia, Spain, France, Czechia, Germany, Portugal, Latvia, Hungary, Austria, and Ireland, evaluates efgartigimod given through intravenous infusion. The study has both a double-blinded phase and an open-label extension phase.

Who can participate: Adults at least 18 years old with documented average platelet counts below 30,000 per microliter are eligible. Participants must have had primary immune thrombocytopenia for more than 12 months and previously received at least one treatment such as corticosteroids, intravenous immunoglobulin, anti-D immunoglobulin, thrombopoietin receptor agonists, or rituximab. They must have shown insufficient response to previous treatments but have demonstrated at least one platelet count of 50,000 or higher in response to certain treatments within the past three years.

Who cannot participate: The study excludes people with conditions other than primary immune thrombocytopenia, those not in the specified age range, and individuals not part of the specific trial group.

Main focus: During the double-blinded period, participants receive either efgartigimod or placebo through regular infusions, with neither participants nor study teams knowing treatment assignments. The study monitors platelet counts to assess disease control, followed by an open-label phase where all participants receive efgartigimod. Regular health assessments including blood tests track platelet counts, side effects, and overall response throughout the study.

Treatment being tested: Efgartigimod is an immunomodulatory agent that works by reducing the activity of certain antibodies contributing to platelet destruction, helping to regulate immune system function.

Study on the Effectiveness of Eltrombopag in Combination with Other Drugs for Patients with Immune Thrombocytopenia

This Czech trial evaluates a complex treatment combining multiple medications: Revolade (eltrombopag), Nplate (romiplostim/AMG 531), Doptelet (avatrombopag), mycophenolate mofetil, ciclosporin, and rituximab, compared to standard treatments.

Who can participate: Adults aged 18 to 70 who can understand the study and sign consent are eligible. Participants must have primary immune thrombocytopenia with platelet counts below 80,000 per microliter after stopping or while on corticosteroids, with a need for therapy due to bleeding, anticoagulation requirements, symptoms affecting daily life, or inability to stop corticosteroids. They must have been treated with corticosteroids for at least 14 days, after which continued treatment was deemed necessary. Women who can have children need negative pregnancy tests and must use effective birth control during treatment and for specified periods after final doses (12 months after rituximab, 90 days after mycophenolate for men).

Who cannot participate: Exclusions include conditions other than immune thrombocytopenia, age outside the specified range, and vulnerable populations.

Main focus: The study evaluates how well the medication combination works together to increase platelet counts and manage symptoms. Regular monitoring includes platelet count measurements, disease control assessments, quality of life questionnaires (SF-36), and tracking remission status (improvement without ongoing therapy). Follow-up visits occur at months 3, 7, 12, and 18 to measure treatment effectiveness and record relapses within 12 months post-treatment.

Treatment being tested: The trial uses a complex immunomodulatory therapy called T-MEM, involving four different medicinal products working together to regulate the immune system, increase platelet counts, and reduce bleeding risk.

Study on the Safety and Effectiveness of Belantamab Mafodotin for Adults with Primary Immune Thrombocytopenia Previously Treated with Standard Medications

This Greek trial investigates belantamab mafodotin, a medication given through intravenous injection, for adults who have previously been treated with standard medications like corticosteroids, thrombopoietin receptor agonists, and/or rituximab.

Who can participate: Adults aged 18 or older with primary immune thrombocytopenia and platelet counts below 30 billion per liter are eligible. Participants must have undergone first-line therapy with corticosteroids and second-line therapy with thrombopoietin receptor agonists and/or rituximab without achieving or maintaining response. They need adequate organ function including specific thresholds for absolute neutrophil count (at least 1.5 billion per liter), hemoglobin (at least 8.0 grams per deciliter), total bilirubin, liver enzymes, and kidney function. Women of childbearing potential must use contraception, have negative pregnancy tests, and not be pregnant or breastfeeding. Men must also use contraception and not donate sperm. Participants need an Eastern Cooperative Oncology Group Performance Status of 2 or less and must be able to provide written informed consent.

Who cannot participate: Exclusions include conditions other than primary immune thrombocytopenia, age outside the specified range, unwillingness to follow study procedures, other interfering medical conditions, pregnancy, breastfeeding, recent participation in other trials, and allergies to study medication.

Main focus: The study evaluates complete and partial response rates at 6 months, with additional assessments at 2, 6, and 12 months. Treatment continues for up to 12 months with regular monitoring of platelet levels and side effects. The study aims to determine if belantamab mafodotin can increase platelet counts and reduce bleeding symptoms.

Treatment being tested: Belantamab mafodotin is an antibody-drug conjugate administered intravenously that targets and binds to a specific protein on certain cell surfaces, leading to cell death. It is currently being studied for improving platelet counts in patients not responding well to other treatments.

Study of efgartigimod given by IV infusion in adolescents aged 12-17 years with chronic immune thrombocytopenia

This trial, conducted in Romania, Hungary, Poland, Italy, France, Spain, and Lithuania, tests efgartigimod in younger patients aged 12 to 17 years. The study consists of a double-blind phase followed by an open-label period.

Who can participate: Young people aged 12 to 17 with chronic immune thrombocytopenia lasting more than 12 months are eligible. Participants need platelet counts below 30,000 per microliter and must have received previous treatment with corticosteroids, intravenous immunoglobulin, anti-D immunoglobulin (for eligible patients), thrombopoietin receptor agonists, or rituximab. They must have shown response to at least one previous treatment by achieving a platelet count of at least 50,000 per microliter and demonstrated insufficient response to previous treatments. Female participants who can become pregnant need to use approved contraception and have negative pregnancy tests. Multiple documented platelet count measurements are required in the months before starting.

Who cannot participate: Exclusions include recent platelet transfusions within 2 weeks, recent rituximab treatment within 6 months, active bacterial infections requiring antibiotics, known active viral infections (hepatitis B/C, HIV), blood clots within the past 12 months, major surgery within 4 weeks, participation in other trials within 30 days, serious heart/liver/kidney problems, active infections, use of blood thinners, history of cancer within the past 5 years, uncontrolled high blood pressure, substance abuse within the past year, and mental health conditions affecting participation.

Main focus: During the double-blind phase, participants receive either efgartigimod or placebo without knowing which treatment they’re receiving. Platelet counts, bleeding symptoms, immune system responses, and medication levels in the body are monitored regularly. Quality of life and fatigue levels are assessed through questionnaires. The open-label phase lasts 24 weeks, during which all participants receive efgartigimod with continued platelet count monitoring. Success is defined as platelet counts reaching 50,000 per microliter or higher.

Treatment being tested: Efgartigimod works by reducing levels of immunoglobulin G antibodies responsible for destroying platelets, helping maintain normal platelet counts. It belongs to the class of immunomodulating agents.

Study of oral rilzabrutinib for adults with immune thrombocytopenia who did not respond to first-line treatment

This trial, conducted in Poland, Italy, Hungary, Spain, Austria, Czechia, France, and Germany, tests rilzabrutinib tablets taken orally in adults whose condition has not improved with initial treatments such as corticosteroids or immunoglobulins.

Who can participate: Adults aged 18 or older with confirmed primary immune thrombocytopenia and an ECOG performance status of 2 or lower are eligible. Participants must have received at least one first-line therapy (corticosteroids, intravenous immunoglobulin, or anti-D treatment) and shown improvement in platelet counts above 30,000 per microliter during previous treatment. They need up-to-date vaccinations, must use appropriate contraception if sexually active, and have adequate blood test results showing hemoglobin above 9 grams per deciliter, adequate white blood cell and platelet counts, normal liver and kidney function, and normal blood clotting tests. Participants must be able to understand and sign informed consent.

Who cannot participate: Exclusions include age below 18 or above 65, pregnancy, breastfeeding, history of blood clots, active bleeding or high bleeding risk, recent major surgery within 30 days, current participation in other trials, severe liver or kidney problems, cancer in the past 5 years, uncontrolled high blood pressure, heart problems including heart failure, active infections (hepatitis B/C, HIV), autoimmune disorders other than ITP, regular use of blood thinners, and alcohol or substance abuse within the past year.

Main focus: Participants take rilzabrutinib tablets daily for approximately 80 weeks with a maximum daily dose of 800 milligrams. The study determines if the medication can maintain higher platelet counts, reduce bleeding problems, and allow reduction or discontinuation of corticosteroid medications. Platelet counts are checked regularly, with positive response indicated by achieving at least 50,000 per microliter or between 30,000 and 50,000 with at least double the starting count. During the final 12 weeks, participants must maintain target platelet levels for at least half their scheduled check-ups.

Treatment being tested: Rilzabrutinib is a Bruton’s tyrosine kinase inhibitor taken orally that works by blocking specific enzymes involved in the immune system’s response, helping prevent platelet destruction. It represents a novel approach particularly for patients who haven’t responded to first-line treatments.

Summary

Across Europe, 18 clinical trials are actively recruiting patients with immune thrombocytopenia, offering diverse treatment options and research opportunities. The trials span 18 countries, with notable concentrations in Italy (8 trials), Spain (8 trials), Poland (7 trials), France (7 trials), Hungary (6 trials), and Germany (4 trials).

Several medications feature prominently across multiple trials. Efgartigimod appears in five different studies, being tested as both intravenous and subcutaneous formulations in adult and adolescent populations. Rilzabrutinib is being evaluated in three trials, exploring its effectiveness in different patient populations including those who haven’t responded to initial treatments. Other frequently studied medications include rituximab, corticosteroids, and various thrombopoietin receptor agonists.

The trials encompass different disease stages and patient populations, from newly diagnosed patients to those with persistent or chronic conditions lasting more than 12 months. Some studies focus on patients who have failed previous treatments, while others compare different medication combinations or dosing strategies. Several trials specifically evaluate long-term safety and effectiveness over periods ranging from 24 weeks to 104 weeks.

Treatment approaches vary considerably, including monoclonal antibodies, immunomodulatory agents, kinase inhibitors, and combination therapies. Many studies employ double-blind placebo-controlled designs followed by open-label extensions, ensuring rigorous evaluation while ultimately allowing all participants access to the investigational treatment.

The geographic distribution of trials provides patients across Europe with local access to cutting-edge research. Countries like France, Spain, Italy, and Poland offer particularly diverse options, with multiple trials testing different medications and approaches. This broad availability means patients in many European countries can find trials matching their specific situation, disease stage, and previous treatment history.

Ongoing Clinical Trials on Immune thrombocytopenia

  • Study on the Effectiveness of Eltrombopag in Combination with Other Drugs for Patients with Immune Thrombocytopenia

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Czechia

  • Study of ianalumab for adults with primary immune thrombocytopenia or warm-antibody autoimmune hemolytic anemia who previously responded to ianalumab

    Not yet recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Belgium Bulgaria Czechia France Germany Hungary +3

  • Study of rituximab and belimumab combination compared to rituximab alone in adults with persistent or chronic immune thrombocytopenia (ITP)

    Not recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    France

  • Study of Rilzabrutinib for Adults with Relapsed Immune Thrombocytopenia

    Not recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Bulgaria Czechia The Netherlands

  • Study on Aspirin (D,L-Lysine Acetylsalicylate) for Patients with Immune Thrombocytopenia and Cardiovascular Disease

    Not recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    France

  • Study Comparing Romiplostim and Dexamethasone with Dexamethasone Alone for Patients with Newly Diagnosed Primary Immune Thrombocytopenia

    Not recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    Italy Spain

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