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.
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).
Treatment with any MF-directed therapy (including investigational therapies) within 2 weeks or 5 half-lives, whichever is shorter, of Cycle 1 Day 1
Participants in COHORT-1 should not have received a JAK inhibitor within 14 days prior to Cycle 1 Day 1. Participants who remain on JAK inhibitor at time of screening should be tapered off per investigator discretion.
Hydroxyurea is permitted until the day prior to C1D1, if needed for disease control.
Undergone allogeneic hematopoietic stem cell transplant (allo-HSCT) within the last 6 months prior to enrollment, or with active GVHD and/or on immunosuppressive therapy.
John Mascarenhas
Lead sponsor
Icahn School of Medicine at Mount Sinai
Sponsor institution
Syndax Pharmaceuticals
Collaborator
National Cancer Institute (NCI)
Collaborator