Phase 3
N=156
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
Primary Myelofibrosis (PMF) · Post-polycythemia Vera (Post-PV) · Post-essential Thrombocythemia Myelofibrosis (Post-ET MF)
Bottom Line
View on ClinicalTrials.gov: NCT02101268 ↗Enrolled (actual)
156
Serious AEs
38.9%
Results posted
May 2023
Primary outcome: Primary: Splenic Response Rate at Week 24 — 7; 3; 97; 49 Participants — p=0.90
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Momelotinib (Drug); Best Available Therapy (BAT) (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Sierra Oncology LLC - a GSK company
- Primary completion
- Jul 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Splenic Response Rate at Week 24 |
7; 3; 97; 49 | 0.90 |
| SECONDARY Total Symptom Score (TSS) Response Rate at Week 24 |
27; 3; 76; 48 | < 0.001 sig |
| SECONDARY Rate of Red Blood Cell (RBC) Transfusion in the Randomized Treatment Phase, (the Average Number of RBC Units Transfused Per Month Not Associated With Overt Bleeding) |
0.5; 1.2 | 0.38 |
| SECONDARY RBC Transfusion Independence Rate at Week 24, (Defined as Absence of RBC Transfusions and no Hemoglobin Level Below 8 g/dL in the 12 Weeks Prior to Week 24, Excluding Cases Associated With Clinically Overt Bleeding) |
45; 11; 59; 41 | 0.001 sig |
| SECONDARY RBC Transfusion Dependence Rate at Week 24 |
52; 33; 52; 19 | 0.10 |
Summary
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 204 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 subjects planning to continue treatment with MMB following the end of the study, the End of Treatment, 30-day, 12-Week, and survival follow-up visits are not required.
Eligibility Criteria
Key Inclusion Criteria
- Palpable splenomegaly at least 5 cm below left costal margin
- Confirmed diagnosis of PMF in accordance, or Post-PV/ET MF
- Currently or previously treated with ruxolitinib for PMF or Post-PV/ET MF for at least 28 days, and characterized by
- Requirement for RBC transfusion while on ruxolitinib treatment, OR
- Dose adjustment of ruxolitinib to 0.75 x 10^9/L in the absence of growth factor in the prior 7 days
- Peripheral blood blast count 24 weeks
- Negative serum pregnancy test for female subjects (unless surgically sterile or greater than two years post-menopausal)
- Males and females of childbearing potential must agree to use protocol-specified method(s) of contraception
- Females who are nursing must agree to discontinue nursing before the first dose of MMB
- Able to understand and willing to sign informed consent form (ICF)
Key Exclusion Criteria
- Prior splenectomy
- Splenic irradiation within 3 months prior to Randomization
- Use of investigational agent within 28 days prior to Randomization
- Prior treatment with MMB
- Hematopoietic growth factor (granulocyte growth factor, erythropoiesis stimulating agent, thrombopoietin mimetic) within 28 days prior to Randomization
- Uncontrolled inter-current illness, per protocol
- Known positive status for human immunodeficiency virus (HIV)
- Chronic active or acute viral hepatitis A, B, or C infection, or hepatitis B or C carrier
- Presence of peripheral neuropathy ≥ CTCAE Grade 2
- Unwilling or unable to undergo a MRI or CT Scan per study protocol requirements
Note: Other protocol defined Inclusion/Exclusion criteria may apply.
Data sourced from ClinicalTrials.gov (NCT02101268). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.