Myelofibrosis

Posted 8 months ago by Wales Gene Park

Brief Summary A Phase 3, multicenter, open-label, randomized study to evaluate the efficacy and safety of fedratinib compared to best available therapy (BAT) in subjects with DIPSS (Dynamic International Prognostic Scoring System)-intermediate or high-risk primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (post-PV MF), or post-essential thrombocythemia myelofibrosis (post-ET MF) and previously treated with ruxolitinib. The primary objective of the study is to evaluate the percentage of subjects with at least 35% spleen volume reduction in the fedratinib and the BAT arms. Detailed Description This Phase 3, multicenter, randomized, two-arm, open-label study will include subjects with intermediate or high-risk (as per the DIPSS score) primary myelofibrosis (PMF), postpolycythemia vera myelofibrosis (post-PV MF), or post-essential thrombocythemia myelofibrosis (post-ET MF). This study will be conducted in compliance with International Council for Harmonisation (ICH) Good Clinical Practices (GCPs). Study design includes: A 28-day Screening Period 2:1 Randomization to fedratinib or best available therapy (BAT) Stratification...

 Myelofibrosis

Posted 4 months ago by Wales Gene Park

Brief Summary: A Phase 3, randomized, blinded study comparing pelabresib (CPI-0610) and ruxolitinib with placebo and ruxolitinib in myelofibrosis (MF) patients that have not been previously treated with Janus kinase inhibitors (JAKi). Pelabresib is a small molecule inhibitor of bromodomain and extra-terminal (BET) proteins.

 Myelofibrosis /  Cardiff

Posted 4 months ago by Wales Gene Park

Summary: Myelofibrosis (MF) is a cancer of the bone marrow that disrupts the production of blood cells. Symptoms of MF include anaemia (low red cell levels), weakness, tiredness and often an enlarged spleen. Standard treatment can involve chemotherapy, radiotherapy, a stem cell transplant or treatment with a drug called ruxolitinib. Ruxolitinib targets the specific cells that play a part  in the development of MF, and is the only drug licenced to treat MF. Previous research has shown that ruxolitinib is good at reducing symptoms of MF and decreasing the size of the spleen. However, the majority of patients do not show a complete response to ruxolitinib, meaning that another treatment needs to be added to ruxolitinib to improve the outcome for patients. This study will test whether combining ruxolitinib with another drug called a bromodomain and extra terminal inhibitor (BETi) is safe for MF. Research has suggested that combining these...

 Myelofibrosis /  Cardiff

Posted 4 months ago by Wales Gene Park

Brief Summary: Phase 1 Part (Complete): Open-label, sequential dose escalation study of CPI-0610 in patients with previously treated Acute Leukemia, Myelodysplastic Syndrome, Myelodysplastic/Myeloproliferative Neoplasms, and Myelofibrosis. Phase 2 Part: Open-label study of CPI-0610 with and without Ruxolitinib in patients with Myelofibrosis. CPI-0610 is a small molecule inhibitor of bromodomain and extra-terminal (BET) proteins.

 Myelofibrosis /  Cardiff


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