Ongoing Clinical Trials for Thrombocytosis
Currently, there are 3 ongoing clinical trials testing new treatments for patients with thrombocytosis, a condition involving abnormal blood cell production. These studies are investigating medications called pelabresib, gandotinib, and ropeginterferon alfa-2b, and are being conducted across multiple European countries including Italy, Belgium, Netherlands, Austria, Spain, Germany, Poland, Hungary, Romania, France, Czechia, and Greece.
Clinical trial locations
- Austria
- Belgium
- Czechia
- France
- Germany
- Greece
- Hungary
- Italy
- Netherlands
- Poland
- Romania
- Spain
Study on Long-Term Safety of Pelabresib for Patients with Myelofibrosis or Essential Thrombocythemia
This study is evaluating the long-term safety and benefits of pelabresib, a medication that works by targeting specific proteins involved in abnormal blood cell growth. It is designed for patients with myeloproliferative neoplasms, specifically myelofibrosis and essential thrombocythemia, who have been receiving pelabresib in previous clinical studies and are continuing to benefit from it.
Who can participate: Patients must be at least 18 years old and currently enrolled in a previous pelabresib study where they are still receiving treatment and experiencing clinical benefits. Participants need to be willing to attend scheduled visits and follow treatment plans. Both men and women must agree to use effective birth control during the study and for a specified period afterward. Women of childbearing potential must have a negative pregnancy test before joining and agree to regular testing throughout the study.
Who cannot participate: Patients with medical conditions other than myeloproliferative neoplasms like myelofibrosis or essential thrombocythemia are excluded. Those outside the specified age range or not part of the designated clinical trial group cannot join. Vulnerable populations requiring special protection are also excluded.
What the study involves: Participants will continue taking pelabresib as an oral tablet. The study will monitor long-term safety, assess survival rates, and evaluate clinical benefits. Regular check-ups will track any side effects, overall survival, leukemia-free survival, and quality of life. The medication is a type of BET inhibitor that helps control abnormal cell growth by affecting gene expression.
Study of Gandotinib for Patients with Myeloproliferative Neoplasms, Myelofibrosis, Essential Thrombocythemia, or Polycythemia Vera
This trial is testing gandotinib (also known as LY2784544), a medication taken once daily as a capsule to treat various blood cancers collectively known as myeloproliferative neoplasms. The study aims to determine how effective gandotinib is in managing these conditions, which include myelofibrosis, essential thrombocythemia, and polycythemia vera.
Who can participate: Patients must be 18 years or older with a confirmed diagnosis according to World Health Organization criteria. They need to have stopped all previous therapies for at least 14 days, though hydroxyurea on a stable dose and low-dose aspirin are permitted. Participants must be able to swallow capsules and agree to use effective birth control during and for 3 months after the study. Women must have a negative pregnancy test and not be breastfeeding. Patients must have adequate organ function and a performance status of 0, 1, or 2 on a scale measuring ability to perform daily activities.
Who cannot participate: Those with other types of cancer unrelated to myeloproliferative neoplasms are excluded. Patients who had a heart attack or stroke within the past 6 months, have uncontrolled high blood pressure, severe liver or kidney disease, are pregnant or breastfeeding, or have allergies to the study medication cannot participate. Those with a recent history of drug or alcohol abuse or participating in another clinical trial are also excluded.
What the study involves: Gandotinib is taken orally once daily. The study will monitor response to treatment over time, comparing it with a placebo in some participants. Regular assessments will measure the objective response rate and characterize any side effects. The medication is a kinase inhibitor that targets specific pathways contributing to overproduction of blood cells. The study is estimated to conclude by December 31, 2024.
Study on Ropeginterferon Alfa-2b for Patients with Essential Thrombocythemia Intolerant or Refractory to Other Treatments
This study is evaluating ropeginterferon alfa-2b for patients with essential thrombocythemia, a rare blood disorder causing excessive platelet production. The medication is given as an injection under the skin using a pre-filled pen and is specifically designed for patients who cannot tolerate or do not respond to other blood cell-reducing treatments.
Who can participate: Patients must be 18 years or older with a confirmed diagnosis of essential thrombocythemia according to 2016 World Health Organization criteria, including a bone marrow biopsy not older than 5 years. They must need treatment to reduce blood cell counts but be intolerant to, not responding to, or ineligible for other approved treatments. There must be at least a 14-day break from previous treatments. Patients must not have received interferon treatment before and must have adequate liver function. They must score 0-7 on both parts of a questionnaire assessing anxiety and depression levels, or receive psychiatric clearance if scores are slightly higher.
Who cannot participate: Patients with conditions other than essential thrombocythemia are excluded. Those who are not intolerant, refractory, or ineligible for other blood cell-reducing therapies cannot participate. Vulnerable populations requiring special protection are also excluded.
What the study involves: After a screening phase lasting up to 28 days, participants receive ropeginterferon alfa-2b injections under the skin. The dosage is adjusted based on individual response and tolerance. Regular monitoring tracks blood counts and symptoms. The primary goal is to assess disease response at month 12, with additional evaluations at months 9, 18, 24, 30, and 36. The study also monitors for blood clots or bleeding events and uses questionnaires to assess quality of life and symptom improvement. Ropeginterferon alfa-2b is an interferon that modulates the immune system to reduce excessive blood cell production. The study is expected to conclude by December 28, 2027.
Summary
All three ongoing clinical trials focus on myeloproliferative neoplasms, with essential thrombocythemia being addressed in all studies. These trials are widely distributed across Europe, with the ropeginterferon alfa-2b study having the broadest geographic reach across 10 countries. The pelabresib study uniquely focuses on long-term safety for patients already receiving treatment, while the gandotinib and ropeginterferon studies test effectiveness in patients with limited previous treatment options. Each medication uses a different approach: pelabresib as a BET inhibitor, gandotinib as a kinase inhibitor, and ropeginterferon alfa-2b as an interferon therapy. These diverse treatment mechanisms offer hope for patients with varying needs and treatment histories.