Menin-Inhibitor Targeted Maintenance in AML

NCT07563010 | Not Yet Recruiting Phase 2 DMC

• 1 other identifier: MENTAIN
• Study Type: interventional
• Target: 144
• Locations: 0 countries
• Sites: N/A

Brief Summary

Revumenib is a first in class oral menin inhibitor that targets a central oncogenic dependency shared across KMT2Ar, NPM1m, and NUP98r AML. In addition to suppressing leukemogenic transcriptional programs and promoting leukemic differentiation, menin inhibition has been shown to modulate epigenetic states linked to antigen presentation and immune recognition. These properties provide a strong biological rationale for evaluating revumenib as maintenance therapy following alloHCT, with the goal of suppressing residual leukemic clones while preserving or enhancing GVL activity during immune reconstitution.

Conditions

Relapsed Adult AML; Stem Cell Transplant Complications; Acute Myeloid Leukemia; Transplant-Related Hematologic Malignancy

Keywords

Allogenic Transplant; post-transplant relapse prevention

Outcome Measures

Primary Outcomes (1)

• Relapse Free Survival (RFS) in KMT2Ar, NPM1m, and NUP98r acute leukemias in the Intent-to-Treat (ITT) population with a minimum of 1 year of follow-up post-randomization

  RFS is defined as the time from randomization to the date of relapse or the date of death from any cause, whichever comes first. RFS is defined as the time from randomization to the date of relapse or the date of death from any cause, whichever comes first.

  From date of randomization until relapse, assessed up to 13 months

Secondary Outcomes (7)

• RFS in the modified ITT (mITT) population

  From date of randomization until relapse, assessed up to 13 months

• Overall survival (OS) in the ITT population

  From date of randomization until death, assessed up to 13months

• Relapse incidence in the ITT population

  From date of randomization to the first incident of relapse, assessed up to 13 months

• Event free survival (EFS) in the ITT population

  From date of randomization to first event of relapse or disease progression, assessed up to 13 months

• Non-relapse mortality (NRM) in the ITT population

  From date of randomization to death unrelated to relapse, assessed up to 13 months

Study Arms (2)

Revumenib BID

EXPERIMENTAL

160 mg/oral/q12h for patients not taking strong CYP3A4 inhibitor 110 mg/oral/q12h for patients taking strong CYP3A4 inhibitor

Drug: Revumenib

Placebo BID

PLACEBO COMPARATOR

160 mg/oral/q12h for patients not taking strong CYP3A4 inhibitor 110 mg/oral/q12h for patients taking strong CYP3A4 inhibitor

Drug: Placebo

Interventions

Revumenib DRUG

oral tablets

Also known as: Revuforj

Revumenib BID

Placebo DRUG

oral tablets

Placebo BID

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Aged ≥18 years at the time of signing informed consent
• Able to provide written informed consent personally or via a legally authorized representative in accordance with applicable regulatory and institutional requirements
• Willing and able to comply with all study procedures and available for the duration of the study
• Diagnosis of acute myeloid leukemia (AML) in complete morphologic remission with one of the following molecular abnormalities:
• KMT2A-rearranged (KMT2Ar) AML
• Excluding KMT2A partial tandem duplication (KMT2A-PTD)
• NPM1-mutated (NPM1m) AML
• Including FLT3-ITD or TKD co-mutation
• NUP98-rearranged (NUP98r) AML
• Planned first allogeneic hematopoietic cell transplantation (allo-HCT) for AML.
• Transplant Characteristics
• Planned allo-HCT using bone marrow or peripheral blood stem cell graft source.
• Planned reduced-intensity/non-myeloablative conditioning (RIC/NMA) or myeloablative conditioning (MAC), using a conditioning regimen permitted- by the protocol and consistent with standard clinical practice, meeting CIBMTR criteria for conditioning intensity
• Planned donor:
• HLA-matched related donor (5/6 or 6/6)

You may not qualify if:

• Disease Status
• a. Evidence of active AML prior to HCT, assessed within 42 days before transplant, defined as any of the following:
• ≥5% bone marrow blasts
• Circulating blasts within 14 days before conditioning
• CNS or other extramedullary disease
• Other active malignancy that, in the investigator's judgment, could interfere with safety or efficacy assessment
• Treatment with non-protocol antileukemic therapy (donor lymphocyte infusion for relapse prophylaxis or treatment will be considered an EFS event)
• Cardiac / QT Risk
• Requirement for concomitant medications known to prolong QT/QTc interval, except low-risk agents used as standard supportive care
• Diagnosis or suspicion of Long QT syndrome, or a family history of Long QT syndrome
• QTcF \>450 msec.
• History within 6 months of study entry of:
• Myocardial infarction
• Unstable angina
• Congestive heart failure (NYHA Class ≥ II)

Sponsors & Collaborators

• Center for International Blood and Marrow Transplant Research: lead
• Syndax Pharmaceuticals: collaborator
• Dana-Farber Cancer Institute: collaborator

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

revumenib

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Steven Devine, M.D

  NMDP

  STUDY CHAIR

Central Study Contacts

Paul Guo

CONTACT

7634064583; pguo@nmdp.org

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Masking Details: Double blind
• Purpose: SUPPORTIVE CARE
• Intervention Model: PARALLEL
• Sponsor Type: NETWORK
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 15, 2026
• First Posted: May 1, 2026
• Study Start (Estimated): December 1, 2026
• Primary Completion (Estimated): June 1, 2031
• Study Completion (Estimated): June 1, 2031
• Last Updated: May 4, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share