Not Yet RecruitingPhase 1/2

Revumenib in Patients With Myelofibrosis

United States32 participantsStarted 2026-12-01

Plain-language summary

This is a Phase Ib/II 2 study investigating the safety and efficacy of revumenib in two cohorts of participants with myelofibrosis. COHORT-1 will investigate the safety of revumenib as monotherapy in participants with myelofibrosis previously treated with a JAK inhibitor. Following confirmation of safety in COHORT-1, the study will proceed with enrollment in COHORT-2, which will evaluate the efficacy and safety of revumenib in combination with a JAK inhibitor.

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: In order to be eligible to participate in this study, an individual must meet all of the following criteria: * Adults ≥ 18 years of age at time of signing the informed consent * Participants must voluntarily sign informed consent form (ICF) and be willing and able to adhere to the study visit schedule and all protocol requirements. * Have an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2 * Participants must have a pathologically confirmed diagnosis of PMF, post-ET-MF or post-PV-MF as per the WHO diagnostic criteria, with intermediate-1 or higher risk disease by DIPSS (14.1). * Criteria for COHORT-1 (Monotherapy): Treated with at least one prior line of JAK inhibitor therapy to which they were refractory/resistant, lost response, or intolerant, or is not a candidate for approved JAK inhibitor therapy per investigator judgement, and with one or more of the following features of active disease: * Spleen palpable ≥ 5 cm below the left costal margin or \> 450cm3 by MRI/CT * MPN-SAF TSS ≥ 10 * Transfusion dependence (requiring at least 6 units of PRBCs in the 12 weeks prior to study enrollment, for a hemoglobin \< 8.5g/dL in the absence of bleeding or treatment-induced anemia) * Criteria for COHORT-2 (Combination therapy): Currently receiving treatment with an approved JAK inhibitor (including ruxolitinib, fedratinib, or momelotinib) with stable dose for at least 12 weeks prior to study enrollment, and with one or more of the following…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Dose limiting toxicity (DLT)

Timeframe: 2 months

Number of Treatment related adverse events (AEs)

Timeframe: 6 months

Overall response rate (ORR) for COHORT-2

Timeframe: 6 months

Trial details

NCT IDNCT07734077

SponsorJohn Mascarenhas

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2028-12-01

Contact for this trial

Gillian Sanchez

(212) 241-1391 gillian.sanchez@mssm.edu

View on ClinicalTrials.gov More Myelofibrosis trials