A Safety Trial of Fedratinib in Subjects With DIPSS, Intermediate or High-Risk Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis and Previously Treated With Ruxolitinib With Concomitant Luspatercept for Subjects With Anemia

Official Title

A Phase 3b, Multicentre, Single-Arm, Open-Label Efficacy and Safety Study of Fedratinib 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 Including a Sub-study With Concomitant Luspatercept for Subjects With Anemia

Summary:

This is Single-Arm, Open-Label Efficacy and Safety Trial of Fedratinib 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 including a Sub-study with concomitant Luspatercept for subjects with anemia. The primary objective of the main study is to evaluate the percentage of subjects with at least a 35% reduction in spleen size and one of the secondary objectives is to evaluate the safety of fedratinib. The primary objective of the sub-study is to evaluate the safety and tolerability of Luspatercept when administered concomitantly with Fedratinib.

Trial Description

Primary Outcome:

  • Main study - Proportion of subjects who have a ≥ 35% SVR at end of Cycle 6
  • Sub study - Adverse Events (AEs)
Secondary Outcome:
  • Main study - Adverse Event(s)
  • Main study - Proportion of subjects who have ≥ 50% reduction in spleen size by palpation
  • Main study - Symptom response rate (SRR)
  • Main study - To evaluate durability of spleen volume response (DR)
  • Main study - To evaluate the durability of spleen response by palpation (DRP)
  • Main study - Durability of symptoms response (DSR)
  • Main study - Gastrointestinal Adverse Events
  • Main study - Wernicke encephalopathy (WE)
  • Main study - Wernicke encephalopathy (WE) thiamine monitoring
  • Sub study - Anemia response related to modified Hematological Improvement - Erythroid Response (HI-E)
  • Sub study - Anemia Response related to Reduction in Transfusion Burden
  • Sub study - Anemia Response related to Mean Hemoglobin Increase
  • Sub study - Anemia response related to RBC-transfusion independence
  • Sub study - Duration of anemia response
This is Single-Arm, Open-Label Efficacy and Safety Trial of Fedratinib 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 including a Sub-study with concomitant Luspatercept for subjects with anemia. The spleen volume reduction at the end of Cycle 6 as the primary objective. The secondary objectives of the study are to further evaluate the safety and to assess and implement mitigation strategies for WE and for gastrointestinal (GI) adverse events. The study will be at multiple centres to provide access to a broad population and have assurance the results are likely to have general applicability. This is also conducted as an open-label study to collect efficacy and safety data with fedratinib use, no randomization or stratification will occur. The primary objective of the sub-study is to evaluate the safety and tolerability of Luspatercept when administered concomitantly with Fedratinib for the treatment of anemia in subjects with MF who are red blood cell (RBC) transfusion dependent (Group A) or non-transfusion dependent (Group B).

View this trial on ClinicalTrials.gov

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Resources

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