Momelotinib Versus Ruxolitinib in Subjects With Myelofibrosis (Simplify 1)
This study is to determine the efficacy of momelotinib (MMB) versus ruxolitinib in participants with primary myelofibrosis (PMF) or post-polycythemia vera or post-essential thrombocythemia myelofibrosis (post-PV/ET MF) who have not yet received treatment with a Janus kinase inhibitor (JAK inhibitor).
Participants will be randomized to receive either MMB or ruxolitinib for 24 weeks during a double-blind treatment phase, after which they will be eligible to receive open-label MMB for up to an additional 216 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 or until study termination. For those participants planning to continue treatment with MMB following the end of the study, the Early Study Drug Discontinuation (ESDD), 30-day, 12-Week, and survival follow-up visits are not required.
|Primary Myelofibrosis Post-Polycythemia Vera Myelofibrosis Post-Essential Thrombocythemia Myelofibrosis||Drug: Momelotinib Drug: Ruxolitinib Drug: Placebo to match momelotinib Drug: Placebo to match ruxolitinib||Phase 3|
|Study Design:||Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
|Official Title:||A Phase 3, Randomized, Double-blind Active-controlled Study Evaluating Momelotinib vs. Ruxolitinib in Subjects With Primary Myelofibrosis (PMF) or Post-Polycythemia Vera or Post-Essential Thrombocythemia Myelofibrosis (Post-PV/ET MF)|
- 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:||December 6, 2013|
|Estimated Study Completion Date:||June 2018|
|Primary Completion Date:||September 12, 2016 (Final data collection date for primary outcome measure)|
Participants will receive momelotinib plus placebo to match ruxolitinib.
Momelotinib tablet administered orally once daily
Other Names:Drug: Placebo to match ruxolitinib
Placebo to match ruxolitinib tablets administered orally twice daily
Active Comparator: Ruxolitinib
Participants will receive ruxolitinib plus placebo to match momelotinib.
Ruxolitinib tablets administered orally twice dailyDrug: Placebo to match momelotinib
Placebo to match momelotinib tablets administered orally once daily
Please refer to this study by its ClinicalTrials.gov identifier: NCT01969838
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|Study Director:||Gilead Study Director||Gilead Sciences|