Efficacy of Momelotinib Versus Best Available Therapy in Anemic or Thrombocytopenic Subjects With Primary Myelofibrosis (MF), Post-polycythemia Vera MF, or Post-essential Thrombocythemia MF (Simplify 2)
This study is to determine the efficacy of momelotinib (MMB) versus best available therapy (BAT) in anemic or thrombocytopenic adults with primary myelofibrosis (PMF), or post-polycythemia vera or post-essential thrombocythemia myelofibrosis (Post-PV/ET MF) who were treated with ruxolitinib as measured by splenic response rate at Week 24 (SRR24).
Participants will be randomized to receive either MMB or BAT for 24 weeks during the randomized treatment phase, after which they will be eligible to receive MMB in an extended treatment phase for up to an additional 168 weeks. After discontinuation of study medication, assessments will continue for 12 additional weeks, after which participants will be contacted for survival follow-up approximately every 6 months for up to 5 years from the date of enrollment.
|Primary Myelofibrosis (PMF) Post-polycythemia Vera (Post-PV) Post-essential Thrombocythemia Myelofibrosis (Post-ET MF)||Drug: Momelotinib Drug: Best Available Therapy (BAT)||Phase 3|
|Study Design:||Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
|Official Title:||A Phase 3, Randomized Study To Evaluate the Efficacy of Momelotinib Versus Best Available Therapy in Anemic or Thrombocytopenic Subjects With Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, or Post-essential Thrombocythemia Myelofibrosis Who Were Treated With Ruxolitinib|
- Splenic response rate at Week 24 [ Time Frame: Week 24 ]Splenic response rate at Week 24 is defined as the proportion of participants achieving a ≥ 35% reduction in spleen volume at Week 24 from baseline as measured by MRI or CT.
- Response rate in total symptom score at Week 24 [ Time Frame: Week 24 ]Total symptom score (TSS) is defined as the proportion of participants who achieve a ≥ 50% reduction in TSS from baseline to Week 24 as measured by the modified Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPNSAF TSS) v2.0 diary.
- Rate of red blood cell (RBC) transfusion through Week 24 [ Time Frame: Baseline to Week 24 ]Rate of RBC transfusion is defined as the average number of RBC units per participant per month.
- RBC transfusion independence rate at Week 24 [ Time Frame: Week 24 ]RBC transfusion independence is the proportion of participants who are transfusion independent at Week 24, defined as absence of RBC transfusions and no hemoglobin level below 8 g/dL in the prior 12 weeks.
- RBC transfusion dependence rate at Week 24 [ Time Frame: Week 24 ]RBC transfusion dependence is the proportion of participants who are transfusion dependent at Week 24, defined as at least 4 units of RBC transfusions, or a hemoglobin level below 8 g/dL in the prior 8 weeks.
|Actual Study Start Date:||June 19, 2014|
|Estimated Study Completion Date:||June 2018|
|Primary Completion Date:||July 28, 2016 (Final data collection date for primary outcome measure)|
Experimental: Arm 1: Momelotinib
Participants will receive momelotinib for 24 weeks during the randomized treatment phase, after which they will be eligible to receive momelotinib in an extended treatment phase for up to an additional 168 weeks.
Momelotinib tablet administered orally once daily
Active Comparator: Arm 2: Best Available Therapy (BAT)
Participants in the BAT treatment arm will receive treatment at doses and schedules determined by the investigator in accordance with standard of care. Therapy may be changed at any time during the study except during the screening period. After completion of the randomized treatment phase, participants will be eligible to receive momelotinib for the duration of the study during the extended treatment phase for up to 168 weeks.
Drug: Best Available Therapy (BAT)
Regimens for BAT may include but are not limited to chemotherapy (eg hydroxyurea), anagrelide, corticosteroid, hematopoietic growth factor, immunomodulating agent, androgen, interferon, and may include no myelofibrosis treatment.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02101268
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|Study Director:||Gilead Study Director||Gilead Sciences|