Clinical Trials for Thrombocytopenia
This article provides detailed information about 3 ongoing clinical trials investigating new treatments for thrombocytopenia, a condition characterized by low platelet counts that can lead to bleeding and bruising. These studies are being conducted across multiple European countries and are testing different medications including selinexor, pirtobrutinib, and immunoglobulin therapy for various forms of the condition.
Clinical trial locations
- Belgium
- Bulgaria
- Czechia
- Denmark
- France
- Germany
- Greece
- Hungary
- Italy
- Study on the Safety and Effectiveness of Pirtobrutinib for Adults with Immune Thrombocytopenia
- Study on the Effectiveness and Safety of Human Normal Immunoglobulin (IV) for Adults with Chronic Immune Thrombocytopenia
- Study on the Effectiveness and Safety of Selinexor for Patients with Myelofibrosis and Moderate Thrombocytopenia Who Have Not Used JAK Inhibitors
- Netherlands
- Poland
- Romania
- Spain
- Study on the Safety and Effectiveness of Pirtobrutinib for Adults with Immune Thrombocytopenia
- Study on the Effectiveness and Safety of Human Normal Immunoglobulin (IV) for Adults with Chronic Immune Thrombocytopenia
- Study on the Effectiveness and Safety of Selinexor for Patients with Myelofibrosis and Moderate Thrombocytopenia Who Have Not Used JAK Inhibitors
Study on the Effectiveness and Safety of Selinexor for Patients with Myelofibrosis and Moderate Thrombocytopenia Who Have Not Used JAK Inhibitors
This clinical trial is investigating selinexor, an oral medication, for patients who have myelofibrosis combined with moderate thrombocytopenia and have not previously been treated with JAK inhibitor medications. The study aims to evaluate how well selinexor works on its own in treating this specific group of patients.
Main inclusion criteria: Participants must be 18 years or older with a confirmed diagnosis of myelofibrosis. They need to have an enlarged spleen visible on scans and active symptoms of the condition. Platelet counts must be within a specific range without recent transfusions, and participants must have adequate liver and kidney function. A bone marrow sample is required, and participants must have a life expectancy of more than 6 months. Those with active hepatitis B, C, or HIV may be included if they meet specific treatment and viral load requirements. Women of childbearing potential must have a negative pregnancy test and agree to use effective birth control during and after the study.
Main exclusion criteria: Patients who have previously been treated with JAK inhibitors for myelofibrosis cannot participate. Additionally, those who are candidates for stem cell transplantation are excluded from the study.
Trial focus: The primary goal is to assess whether selinexor can reduce spleen size by week 24 of treatment. Secondary objectives include evaluating symptom improvement, anemia response, and overall survival. Participants will take selinexor in tablet form for up to 105 days, with regular monitoring including blood tests and imaging scans such as MRI or CT to measure spleen volume. The study also compares selinexor’s effects with those of a placebo to better understand its effectiveness.
Investigational drug: Selinexor works by inhibiting the export of tumor suppressor proteins from the nucleus of cancer cells, leading to cell death. It is classified as a selective inhibitor of nuclear export and is being tested specifically for patients with myelofibrosis who have not received JAK inhibitor therapy.
Study on the Safety and Effectiveness of Pirtobrutinib for Adults with Immune Thrombocytopenia
This trial is studying pirtobrutinib, an oral tablet, for adults with immune thrombocytopenia, a disorder where the immune system mistakenly destroys platelets, leading to easy bruising and bleeding. The study is designed in two phases to assess both safety and effectiveness of this new treatment approach.
Main inclusion criteria: Participants must be 18 years of age or older with a confirmed diagnosis of primary immune thrombocytopenia. They must have a documented history of responding to at least one previous treatment but have not responded adequately to prior therapies. The study requires evidence that conventional treatments have not been sufficiently effective.
Main exclusion criteria: People with other serious health conditions that might interfere with the study cannot participate. Pregnant or breastfeeding women are excluded, as are those who have had recent major surgery or are planning surgery during the study period. Participants currently using other medications that might interfere with the study treatment, those with a history of allergic reactions to similar medications, or those who have participated in another clinical trial within the last 30 days are not eligible. Additionally, individuals with a history of drug or alcohol abuse within the past year or any condition that makes them unsuitable for the study according to the doctor’s opinion are excluded.
Trial focus: The first phase focuses on determining the safest and most effective dose of pirtobrutinib through careful monitoring with blood tests and heart monitoring. In the second phase, the medication’s effectiveness is compared to a placebo to evaluate how well it improves platelet counts. Participants will have visits every two weeks to check platelet levels and monitor overall health. The study, expected to continue until 2027 with recruitment starting in 2025, aims to determine if pirtobrutinib can help maintain stable platelet counts without the need for additional treatments.
Investigational drug: Pirtobrutinib is a Bruton’s tyrosine kinase inhibitor that targets specific proteins involved in the immune response. By inhibiting these pathways, it may help reduce the destruction of platelets and improve symptoms in people with immune thrombocytopenia.
Study on the Effectiveness and Safety of Human Normal Immunoglobulin (IV) for Adults with Chronic Immune Thrombocytopenia
This study is evaluating KIg10, a form of human normal immunoglobulin given through intravenous infusion, for adults with chronic immune thrombocytopenia. The condition causes persistently low platelet counts due to the immune system attacking platelets, leading to bleeding and bruising problems.
Main inclusion criteria: Participants must be male or female between 18 and 70 years old with a diagnosis of chronic immune thrombocytopenia lasting more than 12 months. They must have a low platelet count (less than 30 × 10⁹/L) confirmed by two tests taken at least one day apart, with one test possibly from the past 14 days before the first treatment. Women of childbearing potential must have a negative pregnancy test at the start and agree to use effective birth control during the study. Participants must be willing to follow all study requirements and provide authorization to access personal health information.
Main exclusion criteria: Patients with any other serious health condition that might interfere with the study are excluded. Pregnant or breastfeeding women cannot participate. Those who have had recent major surgery, are planning surgery during the study period, or are currently participating in another clinical trial are not eligible. People with a history of substance abuse or alcohol dependency, those who have received any investigational drug within the last 30 days, or those with known allergies to the study medication are excluded. Additionally, participants with uncontrolled high blood pressure, other uncontrolled medical conditions, a history of cancer within the last 5 years (except certain types of skin cancer), or active infections requiring treatment cannot join the study.
Trial focus: The study aims to determine how effectively and safely KIg10 can increase platelet counts in adults with chronic immune thrombocytopenia. Participants will have their platelet levels monitored regularly to track improvements. A response is defined as achieving a platelet count greater than 30 × 10⁹/L with at least a two-fold increase from baseline, confirmed on at least two separate occasions seven days apart, with no bleeding during this period. The study will assess how quickly the treatment works, how long the effects last, and monitor for any side effects or bleeding episodes. Regular follow-up visits will evaluate the maximum platelet count achieved and the time taken to reach this level.
Investigational drug: KIg10 is a 10% human normal immunoglobulin solution administered intravenously. It works by providing antibodies that help modulate the immune system, reducing the destruction of platelets. As an immunoglobulin therapy, it is a type of protein normally used by the immune system to fight infections, but in this context, it helps regulate the immune response that is mistakenly attacking platelets.
Summary
These three ongoing clinical trials represent different therapeutic approaches to managing various forms of thrombocytopenia across Europe. The studies collectively involve 13 countries, with the largest geographical reach belonging to the selinexor trial for myelofibrosis-related thrombocytopenia, spanning multiple countries including France, Germany, Italy, Spain, and Poland.
Each trial targets a distinct patient population: the selinexor study focuses on patients with myelofibrosis who have moderate thrombocytopenia and have not received JAK inhibitor therapy; the pirtobrutinib trial addresses immune thrombocytopenia in patients who have not adequately responded to previous treatments; and the immunoglobulin study targets adults with chronic immune thrombocytopenia lasting more than 12 months.
The therapeutic approaches vary significantly, with selinexor representing a novel oral selective inhibitor of nuclear export, pirtobrutinib offering a new Bruton’s tyrosine kinase inhibitor mechanism, and the immunoglobulin study evaluating a more established but important intravenous treatment approach. All three trials emphasize careful monitoring of platelet counts and safety assessments, reflecting the serious nature of thrombocytopenia and the need for effective treatments that minimize bleeding risks while improving patient outcomes.
