TriangleNCT01969838 · CITED IN SOURCE DOCUMENTS
Momelotinib Versus Ruxolitinib in Subjects With Myelofibrosis
An NCI or FDA source cites this study. A citation does not establish that it applies to an individual diagnosis.
- Phase
- PHASE3
- Status at capture
- COMPLETED
- Registry last update
- 2023-05-12
This study is to determine the efficacy of momelotinib (MMB) versus ruxolitinib (RUX) 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.
Open the original ClinicalTrials.gov record → · Download preserved record
What did the study report?
Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.
Participant flow
Baseline characteristics
Splenic Response Rate at Week 24
Total Symptom Score (TSS) Response Rate at Week 24
Rate of Red Blood Cell (RBC) Transfusions in the Double-blind Phase, (the Average Number of RBC Units Transfused Per Month Not Associated With Overt Bleeding)
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)
RBC Transfusion Dependence Rate at Week 24. (Defined as Having Had at Least 4 Units of RBC Transfusions, or a Hemoglobin Level Below 8 g/dL in 8 Weeks Prior to Week 24 (Excluding Cases Associated With Clinically Overt Bleeding)).
Adverse events
Limitations, agreements, and source contact
Who could take part
- eligibility Criteria
- Key Inclusion Criteria: * Palpable splenomegaly at least 5 cm below the left costal margin * Confirmed diagnosis of PMF or post-PV/ET MF * Requires myelofibrosis therapy, in the opinion of the investigator * Classified as high risk OR intermediate-2 risk as defined by the International Prognostic Scoring System (IPSS) for PMF, or intermediate-1 risk (IPSS) associated with symptomatic splenomegaly, hepatomegaly, anemia (hemoglobin \< 10.0 g/dL), and/or unresponsive to available therapy * Acceptable laboratory assessment obtained within 14 days prior to the first dose of study drug: * Absolute neutrophil count (ANC) ≥ 0.75 x 10\^9/L in the absence of growth factor in the prior 7 days * Platelet Count ≥ 50 x 10\^9/L (≥ 100 x 10\^9/L if aspartate aminotransferase \[AST\] or alanine aminotransferase \[ALT\] is ≥ 2 x the upper limit of the normal range \[ULN\]) in the absence of platelet transfusion(s) or thrombopoietin mimetics in the prior 7 days * Peripheral blood blast count \< 10% * AST and ALT ≤ 3 x ULN (≤ 5 x ULN if liver is involved by extramedullary hematopoiesis as judged by the investigator or if related to iron chelator therapy that was started within the prior 60 days) * Calculated creatinine clearance (CrCL) of ≥ 45 mL/min * Direct bilirubin ≤ 2.0 x ULN * Life expectancy of \> 24 weeks * 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 study drug * Able to understand and willing to sign the informed consent form Key Exclusion Criteria: * Prior splenectomy * Splenic irradiation within 3 months prior to the first dose of study drug * Eligible for allogeneic bone marrow or stem cell transplantation * 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 a hepatitis B or C carrier * Prior use of a JAK1 or JAK2 inhibitor * Use of chemotherapy, immunomodulating therapy, biologic therapy, radiation therapy, or investigational therapy within 4 weeks of the first dose of study drug * Presence of peripheral neuropathy ≥ Common Terminology Criteria for Adverse Events (CTCAE) Grade 2 * Unwilling or unable to undergo a magnetic resonance imaging (MRI) or computed tomography (CT) scan Note: Other protocol defined Inclusion/Exclusion criteria may apply.
- healthy Volunteers
- false
- minimum Age
- 18 Years
- sex
- ALL
- std Ages
- ADULT
- OLDER_ADULT
Treatment arms and interventions
- arm Groups
- description
- Participants will receive momelotinib plus placebo to match ruxolitinib.
- intervention Names
- Drug: Momelotinib
- Drug: Placebo to match ruxolitinib
- label
- Momelotinib
- type
- EXPERIMENTAL
- description
- Participants will receive ruxolitinib plus placebo to match momelotinib.
- intervention Names
- Drug: Ruxolitinib
- Drug: Placebo to match momelotinib
- label
- Ruxolitinib
- type
- ACTIVE_COMPARATOR
- interventions
- arm Group Labels
- Momelotinib
- description
- Momelotinib tablet administered orally once daily
- name
- Momelotinib
- other Names
- GS-0387
- CYT387
- type
- DRUG
- arm Group Labels
- Ruxolitinib
- description
- Ruxolitinib tablets administered orally twice daily
- name
- Ruxolitinib
- type
- DRUG
- arm Group Labels
- Ruxolitinib
- description
- Placebo to match momelotinib tablets administered orally once daily
- name
- Placebo to match momelotinib
- type
- DRUG
- arm Group Labels
- Momelotinib
- description
- Placebo to match ruxolitinib tablets administered orally twice daily
- name
- Placebo to match ruxolitinib
- type
- DRUG
Study design
- allocation
- RANDOMIZED
- intervention Model
- PARALLEL
- masking Info
- masking
- QUADRUPLE
- who Masked
- PARTICIPANT
- CARE_PROVIDER
- INVESTIGATOR
- OUTCOMES_ASSESSOR
- primary Purpose
- TREATMENT
Enrollment
- count
- 432
- type
- ACTUAL
Registered outcome plans (not posted results)
- primary Outcomes
- description
- Splenic response rate at Week 24 is defined as the percentage of participants who achieved a spleen volume reduction of ≥ 35% from baseline at the Week 24 assessment as measured by MRI or CT.
- measure
- Splenic Response Rate at Week 24
- time Frame
- Week 24
- secondary Outcomes
- description
- Total symptom score (TSS) is defined as the percentage of participants who achieved a ≥50% reduction in TSS at Week 24 versus baseline as measured by the modified Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) v2.0 diary. Response rate was calculated using the average of the daily TSS from a consecutive 28-day period prior to Week 24, which had ≥20 daily TSS available. The modified MPN-SAF patient-reported outcome instrument consisted of 8 items assessing worst daily incidence of tiredness, filling up quickly, abdominal discomfort, night sweats, itching, bone pain, pain under ribs on left side, and inactivity. Scoring of the Total Symptom Score (TSS), in this study was based on 7 of these items, (range of 0-70,), excluding inactivity. These items assess the impact experienced by the participant in the 24 hours prior to completing the questionnaire. All items are measured using a 0 to 10 Numeric Rating Scale, with 0 corresponding to "Absent" and 10 being the worse
- measure
- Total Symptom Score (TSS) Response Rate at Week 24
- time Frame
- Week 24
- description
- Rate of RBC transfusion was defined as the average number of RBC units transfused not associated with clinically overt bleeding per subject-month during the double-blind phase.
- measure
- Rate of Red Blood Cell (RBC) Transfusions in the Double-blind Phase, (the Average Number of RBC Units Transfused Per Month Not Associated With Overt Bleeding)
- time Frame
- Baseline to Week 24
- description
- RBC transfusion independence is the percentage 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 12 weeks prior to Week 24, excluding cases associated with clinically overt bleeding.
- measure
- 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)
- time Frame
- Week 24
- description
- RBC transfusion dependence is the percentage of participants who are transfusion dependent at Week 24, defined as having had at least 4 units of RBC transfusions, or a hemoglobin level below 8 g/dL in 8 weeks prior to Week 24 (excluding cases associated with clinically overt bleeding). Participants with the last double-blind phase participation date prior to Day 162 (ie. missing at Week 24) were considered transfusion dependent at Week 24.
- measure
- RBC Transfusion Dependence Rate at Week 24. (Defined as Having Had at Least 4 Units of RBC Transfusions, or a Hemoglobin Level Below 8 g/dL in 8 Weeks Prior to Week 24 (Excluding Cases Associated With Clinically Overt Bleeding)).
- time Frame
- Week 24
Full study description
- brief Summary
- This study is to determine the efficacy of momelotinib (MMB) versus ruxolitinib (RUX) 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.
Source references
- references
- citation
- Palandri F, O'Connell C, Vachhani P, McLornan DP, Koschmieder S, Hernandez-Boluda JC, Shimoda K, Zhang S, Ellis C, Lim J, Sahni J, Gupta V. Impact of Hemoglobin Improvement on Survival in Patients With Myelofibrosis and Anemia Treated With Momelotinib in the SIMPLIFY-1 and MOMENTUM Trials. Eur J Haematol. 2026 Jul 14. doi: 10.1111/ejh.70237. Online ahead of print.
- pmid
- 42452788
- type
- DERIVED
- citation
- Mesa R, Palmer JM, Palandri F, Masarova L, Harrison CN, Mazerolle F, M'hari M, Liu T, Zhang S, Cardellino A, Wang Z, Ellis CE, Patnaik D, Regnault A, LeBlanc TW. Association Between Transfusion Status, Hemoglobin Levels, and Patient-Reported Outcomes in Myelofibrosis: A Post Hoc Clinical Trial Analysis. Cancer Med. 2026 Apr;15(4):e71729. doi: 10.1002/cam4.71729.
- pmid
- 41958363
- type
- DERIVED
- citation
- Mesa RA, Talpaz M, Mazerolle F, Gorsh B, M'Hari M, Regnault A, Ellis C, Wang Z, Purser M, Liu T, Strouse B, Patnaik D. Time Without Transfusion Reliance (TWiTR): Integrating Survival Quality Into Myelofibrosis Treatment Strategies Based on the Phase 3 SIMPLIFY-1, SIMPLIFY-2, and MOMENTUM Trials. EJHaem. 2025 Jun 18;6(3):e70075. doi: 10.1002/jha2.70075. eCollection 2025 Jun.
- pmid
- 40535755
- type
- DERIVED
- citation
- Harrison CN, Mesa R, Talpaz M, Gupta V, Gerds AT, Perkins A, Goh YT, Fox ML, McLornan D, Palmer J, Foltz L, Vannucchi A, Koschmieder S, Passamonti F, Lee SE, Ellis C, Strouse B, Gonzalez Carreras FJ, Oh ST. Longitudinal Assessment of Transfusion Intensity in Patients With JAK Inhibitor-Naive or -Experienced Myelofibrosis Treated With Momelotinib. Clin Lymphoma Myeloma Leuk. 2025 Mar;25(3):199-211. doi: 10.1016/j.clml.2024.10.001. Epub 2024 Oct 16.
- pmid
- 39516087
- type
- DERIVED
- citation
- Gupta V, Oh S, Devos T, Dubruille V, Catalano J, Somervaille TCP, Platzbecker U, Giraldo P, Kosugi H, Sacha T, Mayer J, Illes A, Ellis C, Wang Z, Gonzalez Carreras FJ, Strouse B, Mesa R. Momelotinib vs. ruxolitinib in myelofibrosis patient subgroups by baseline hemoglobin levels in the SIMPLIFY-1 trial. Leuk Lymphoma. 2024 Jul;65(7):965-977. doi: 10.1080/10428194.2024.2328800. Epub 2024 Mar 19.
- pmid
- 38501751
- type
- DERIVED
- citation
- Verstovsek S, Mesa R, Gupta V, Lavie D, Dubruille V, Cambier N, Platzbecker U, Hus M, Xicoy B, Oh ST, Kiladjian JJ, Vannucchi AM, Gerds A, Egyed M, Mayer J, Sacha T, Kawashima J, Morris M, Huang M, Harrison C. Momelotinib long-term safety and survival in myelofibrosis: integrated analysis of phase 3 randomized controlled trials. Blood Adv. 2023 Jul 25;7(14):3582-3591. doi: 10.1182/bloodadvances.2022009311.
- pmid
- 37042865
- type
- DERIVED
Source notices and limitations
Discovery and provenance
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- a Thirty‑six (27.7%) subjects treated with OJJAARA and 27 (41.5%) subjects treated with danazol discontinued treatment prior to Week 24. SIMPLIFY‑1 SIMPLIFY‑1 (NCT01969838) was a double‑blind, randomized, active‑controlled trial in 432 adults with MF who had not previously received a JAK inhibitor. Patients were treated with OJJA
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Preserved source evidence · Not medical advice