Ongoing Clinical Trials for Myeloproliferative Neoplasm
There are currently 6 clinical trials investigating new treatments for myeloproliferative neoplasm, a group of diseases where the bone marrow produces too many blood cells. These studies are testing various medications including pelabresib, atorvastatin, apixaban, rivaroxaban, aspirin, gandotinib, givinostat, navitoclax, and ruxolitinib. The trials are being conducted across several European countries including Italy, Belgium, Netherlands, Denmark, France, Austria, Spain, Germany, Bulgaria, and Croatia.
Clinical trial locations
- Austria
- Belgium
- Bulgaria
- Croatia
- Denmark
- France
- Germany
- Italy
- Study on Long-Term Safety of Pelabresib for Patients with Myelofibrosis or Essential Thrombocythemia
- Study on the Long-term Effects of Givinostat for Patients with JAK2V617F Positive Chronic Myeloproliferative Neoplasms
- Study on the Safety and Effects of Navitoclax and Ruxolitinib for Patients with Myelofibrosis and Myeloproliferative Neoplasms
- Netherlands
- Spain
Study on Long-Term Safety of Pelabresib for Patients with Myelofibrosis or Essential Thrombocythemia
This trial is examining the long-term safety of pelabresib, a small molecule bromodomain inhibitor, in patients with myelofibrosis or essential thrombocythemia. Pelabresib works by targeting specific proteins involved in the growth of abnormal blood cells.
Main inclusion criteria: Participants must be at least 18 years old and currently receiving treatment with pelabresib in a previous clinical study. They must be experiencing clinical benefits from the treatment as determined by the study doctor. Participants must be willing to attend scheduled visits and follow treatment plans. They must also agree to use effective birth control during the study and for a specified period after the last dose. Women of childbearing potential must have a negative pregnancy test before joining.
Main exclusion criteria: Patients with medical conditions other than myelofibrosis or essential thrombocythemia cannot participate. Those not within the specified age range or not part of the previous clinical trial are excluded. Patients considered part of a vulnerable population are also excluded.
Trial focus: The study evaluates long-term safety, survival, and clinical benefits of pelabresib. Participants continue taking pelabresib tablets orally while undergoing regular monitoring to track any adverse events, survival rates, and duration of response. The study also assesses quality of life and progression-free survival.
Study on the Effects of Atorvastatin for Patients with Philadelphia-Negative Chronic Myeloproliferative Neoplasms
This trial investigates whether atorvastatin, a medication commonly used to lower cholesterol, can help reduce inflammation and improve blood cell counts in patients with essential thrombocythemia, polycythemia vera, or prefibrotic myelofibrosis.
Main inclusion criteria: Participants must have a diagnosis of essential thrombocythemia, polycythemia vera, or prefibrotic myelofibrosis as classified by the World Health Organization. They must be older than 18 years with an expected survival time of more than 3 years. If currently receiving treatment to reduce blood cell counts, this treatment must have started more than 3 months ago. Participants must not be taking statins before joining the study.
Main exclusion criteria: Patients with other types of blood cancer cannot participate.
Trial focus: The study examines whether adding atorvastatin to current treatment regimens can reduce inflammatory markers such as hs-CRP and inflammatory cytokines, as well as improve blood cell counts including leukocyte and platelet counts. Participants take atorvastatin tablets orally in doses ranging from 20mg to 80mg once daily, with regular blood tests to monitor effects over up to 24 months.
Study on Apixaban, Rivaroxaban, and Aspirin for Preventing Blood Clots in Patients with JAK2V617F-Positive Myeloproliferative Neoplasms
This trial compares the effectiveness of direct oral anticoagulants (apixaban and rivaroxaban) versus aspirin in preventing blood clots in high-risk patients with polycythemia vera, essential thrombocythemia, or primary myelofibrosis who have the JAK2V617F genetic mutation.
Main inclusion criteria: Participants must have a confirmed diagnosis of polycythemia vera, essential thrombocythemia, or prefibrotic myelofibrosis with the JAK2V617F mutation at a certain level. They must be considered high-risk due to being over 60 years old or having a history of blood clots. Participants must be at least 18 years old, and the time from diagnosis to study enrollment must not exceed 12 months.
Main exclusion criteria: Patients without the JAK2V617F mutation, those not considered high-risk for blood clots, and those under 18 or over 65 years of age are excluded. Patients with other types of blood cancer are also excluded.
Trial focus: The study monitors the time to blood clotting events and significant bleeding events over 24 months. Participants are randomly assigned to receive either apixaban (2.5mg), rivaroxaban (10mg), or aspirin (100mg) taken orally. The trial also evaluates overall survival, quality of life, and treatment adherence.
Study of Gandotinib for Patients with Myeloproliferative Neoplasms, Myelofibrosis, Essential Thrombocythemia, or Polycythemia Vera
This trial evaluates the effectiveness of gandotinib (also known as LY2784544), a medication being tested for treating myelofibrosis, essential thrombocythemia, and polycythemia vera.
Main inclusion criteria: Participants must have a diagnosis of polycythemia vera, essential thrombocythemia, or myelofibrosis as defined by the World Health Organization. They must have stopped all previous approved treatments for at least 14 days and recovered from any immediate effects, though hydroxyurea and low-dose aspirin are allowed. Participants must be at least 18 years old, able to swallow capsules, and have adequate organ function. They must have a performance status of 0, 1, or 2 on the ECOG scale and provide written informed consent.
Main exclusion criteria: Patients with other types of cancer unrelated to myeloproliferative neoplasms, those who have had a heart attack or stroke in the past 6 months, those with uncontrolled high blood pressure, severe liver or kidney disease, pregnant or breastfeeding women, and those with drug or alcohol abuse history in the past year are excluded.
Trial focus: The study assesses the objective response rate and characterizes the toxicity profile of gandotinib. Participants take gandotinib capsules once daily orally, with regular monitoring through physical exams and laboratory tests to evaluate treatment response and safety. Some participants may receive a placebo for comparison.
Study on the Long-term Effects of Givinostat for Patients with JAK2V617F Positive Chronic Myeloproliferative Neoplasms
This trial examines the long-term safety and effectiveness of givinostat, a histone deacetylase inhibitor, in patients with chronic myeloproliferative neoplasms who have the JAK2V617F genetic mutation.
Main inclusion criteria: Participants must have completed treatment with givinostat in at least one previous study or be part of a compassionate use program. They must have tolerated the previous treatment and shown improvement as judged by the doctor. Participants must be adults aged 18 years or older with a confirmed diagnosis of JAK2V617F positive chronic myeloproliferative neoplasm. They must have an ECOG performance status of less than 3 and acceptable organ function. Women of childbearing potential and men with partners who can have children must use effective birth control.
Main exclusion criteria: Patients without a diagnosis of chronic myeloproliferative neoplasm, those not within the specified age range, those unable to follow study procedures, pregnant or breastfeeding women, and those participating in another interfering clinical trial are excluded.
Trial focus: The study gathers information on the long-term safety and tolerability of givinostat, including tracking adverse events and evaluating treatment response. Participants take givinostat capsules orally in doses of 50mg, 75mg, or 100mg. The study also evaluates the reduction of the JAK2v617F allele burden and identifies potential markers of clinical benefit.
Study on the Safety and Effects of Navitoclax and Ruxolitinib for Patients with Myelofibrosis and Myeloproliferative Neoplasms
This trial studies the safety and effects of navitoclax, a BCL-2 family protein inhibitor, both alone and in combination with ruxolitinib. The study also examines how navitoclax interacts with celecoxib.
Main inclusion criteria: Participants must be at least 18 years old with a documented diagnosis of primary or secondary myelofibrosis, essential thrombocythemia, polycythemia vera, or chronic myelomonocytic leukemia as defined by the World Health Organization. They must require treatment and have failed or cannot tolerate at least one previous therapy. Participants must have an ECOG performance status of 2 or less and meet specific laboratory requirements for blood and organ function. For certain parts of the study, participants must have a QTc interval of 450 milliseconds or less.
Main exclusion criteria: Patients with different medical conditions than those being studied, those not within the specified age range, vulnerable populations, those not meeting specific health requirements, pregnant or breastfeeding women, and those who have recently participated in another clinical trial are excluded.
Trial focus: The study evaluates the safety, tolerability, and how the body processes navitoclax and ruxolitinib. It examines the medications’ effects on heart electrical activity through ECG monitoring. Participants take navitoclax and ruxolitinib tablets orally, with regular health checks including physical exams, laboratory tests, and electrocardiograms. The study includes different phases testing navitoclax alone, in combination with ruxolitinib, and its interaction with celecoxib.
Summary
The 6 ongoing clinical trials for myeloproliferative neoplasm represent a diverse range of therapeutic approaches. These studies are investigating different mechanisms of action, including bromodomain inhibition (pelabresib), cholesterol-lowering statins (atorvastatin), anticoagulation strategies (apixaban, rivaroxaban, aspirin), kinase inhibition (gandotinib), histone deacetylase inhibition (givinostat), and BCL-2 family protein inhibition (navitoclax and ruxolitinib).
The trials are spread across multiple European countries, with notable concentration in Italy, which hosts three trials, and France, Germany, and Spain, each hosting two trials. Several studies focus on patients with specific genetic mutations, particularly the JAK2V617F mutation, reflecting the growing emphasis on personalized medicine approaches.
Many of these trials are extension studies evaluating long-term safety and effectiveness of treatments that patients have already been receiving, suggesting that researchers are building on earlier promising results. The studies address various aspects of disease management, from preventing blood clots to reducing inflammation and improving blood cell counts. The expected completion dates range from late 2024 to 2029, indicating that results will become available gradually over the coming years.
