Expanded Access Program for Revumenib

August 28, 2025 updated by: Syndax Pharmaceuticals

Expanded Access Program for SNDX-5613 in Patients With Relapsed/Refractory Acute Leukemias With Genetic Alterations Associated With HOXA Overexpression

This expanded access program will provide an investigational treatment option in a controlled clinical setting for participants who are not otherwise eligible to participate in other Syndax-sponsored clinical studies and have no approved treatment options.

Study Overview

Status

No longer available

Conditions

Intervention / Treatment

Study Type

Expanded Access

Expanded Access Type

  • Treatment IND/Protocol

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • Alabama Center for Childhood Cancer And Blood Disorders, Children's of Alabama
    • Arizona
      • Phoenix, Arizona, United States, 85016
        • Phoenix Children's Hospital
    • California
      • Duarte, California, United States, 91010
        • City of Hope
      • Irvine, California, United States, 92618
        • City of Hope at Orange County Lennar Foundation Cancer Center
      • Los Angeles, California, United States, 90095
        • UCLA, UCLA RRMC, Drug Information Center, Department of Pharmaceutical Services Drug Supply Shipment
      • Palo Alto, California, United States, 94304
        • Lucile Packard Children's Hospital-Stanford
      • San Diego, California, United States, 92123
        • Rady Children's Hospital
      • San Francisco, California, United States, 94158
        • University of California, San Francisco
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Center for Cancer and Blood Disorders, Colorado Children's Hospital
      • Aurora, Colorado, United States, 80045
        • HCTU, Division of Hematology, University of Colorado, Anschutz Medical Center
    • Florida
      • Orlando, Florida, United States, 32804
        • Advent Health Orlando
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Children's Healthcare of Atlanta
      • Atlanta, Georgia, United States, 30322
        • Winship Cancer Institute at Emory University
    • Illinois
      • Chicago, Illinois, United States, 60637
        • University of Chicago Medical Center
      • Chicago, Illinois, United States, 60611
        • Lurie Children's Hospital of Chicago
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa Hospitals and Clinics
    • Louisiana
      • New Orleans, Louisiana, United States, 70118
        • Children's Hospital
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
        • Dana-Farber Cancer Institute, Boston Children's Cancer and Blood Disorders Center
    • Missouri
      • Kansas City, Missouri, United States, 64108
        • Children's Mercy Hospital-Kansas City
      • St Louis, Missouri, United States, 63110
        • Siteman Cancer Center - Washington University
    • New Jersey
      • Hackensack, New Jersey, United States, 07601
        • Hackensack University Medical Center
    • New York
      • Long Island City, New York, United States, 10065
        • Memorial Sloan Kettering Cancer Center
      • The Bronx, New York, United States, 10467
        • Montefiore Medical Center
    • North Carolina
      • Durham, North Carolina, United States, 27705
        • Duke University Medical Center
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital Medical Center
      • Cincinnati, Ohio, United States, 45267
        • Division of Hematology and Oncology, Division of Pulmonary, Critical Care and Sleep Medicine, Vontz Center for Molecular Studies
      • Columbus, Ohio, United States, 43210
        • OSU Medical Center
    • Oregon
      • Portland, Oregon, United States, 97239
        • Doernbecher Children's Hospital, Oregon Health & Science University
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Children's Hospital of Philadelphia
    • Tennessee
      • Memphis, Tennessee, United States, 38105
        • St. Jude Children's Research Hospital
    • Texas
      • Houston, Texas, United States, 77030
        • Md Anderson Cancer Center
      • Houston, Texas, United States, 77030
        • Texas Children's Hospital
      • Houston, Texas, United States, 77030
        • Memorial Hermann-Texas Medical Center
    • Utah
      • Salt Lake City, Utah, United States, 84112
        • Division of Hematology and Hematologic Malignancies, University of Utah-Huntsman Cancer Hospital
    • Washington
      • Seattle, Washington, United States, 98105
        • Seattle Children's Research Institute, Seattle Childrens Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

N/A

Description

Key Inclusion Criteria:

  • Male or female participant aged ≥6 months.
  • Not eligible for participation in an ongoing clinical study and have no approved treatment options.
  • Participant or participant's health care proxy is able and willing to provide written informed consent and able to follow study instructions.

  • Relapsed or refractory (R/R) acute leukemia, as defined by standardized criteria, after standard of care therapy, including but not limited to 1 or 2 cycles of intensive chemotherapy, or venetoclax combinations:

    1. R/R leukemia is defined by the presence of ≥5% blasts in the bone marrow and/or persistence or reappearance of peripheral blasts.
    2. Participants with persistent leukemia after initial therapy or recurrence of leukemia at any time after achieving a response during or after the course of treatment (including allogeneic hematopoietic stem cell transplant [HSCT]) are eligible.
    3. Participants with isolated extra-medullary disease are allowed if extramedullary disease was confirmed with biopsy.
    4. Participants previously treated on a revumenib clinical study who are entering the expanded access program for post-transplant maintenance because they are not eligible to receive revumenib on study or because the study is closed are not required to meet the R/R status.
    5. Participants who underwent HSCT and are eligible to resume treatment with revumenib will be dosed with the last revumenib tolerated dose before transplant.

  • Acute leukemia harboring a lysine (K) methyltransferase 2A gene rearrangement (KMT2Ar), nucleoporin 98 rearrangement, nucleophosmin 1 mutation (or mutated) or any other genetic alteration with overexpression of HOXA genes predicted to potentially respond to menin inhibitors.

    • Note: As revumenib is now approved in the United States, only participants with a KMT2Ar who are not included in the United States prescribing information indication or cannot be accurately dosed (within a 20% margin) with commercial supply and require use of oral solution will be allowed into the study.
  • Adequate liver, renal, and cardiac function.
  • Use of highly effective methods of contraception are required for females and males of childbearing potential from the time of enrollment through 120 days following the last study intervention dose.

For participants currently being treated with revumenib in a Syndax-sponsored clinical study or Syndax investigator-sponsored trial, the following criteria must be met:

  • In the opinion of the Investigator, participant demonstrated acceptable benefit from and tolerability of the study intervention.
  • Participant is considered compliant with study intervention and procedures.
  • Participant does not meet any criteria for study intervention discontinuation.
  • Investigator and participant agree to continue study intervention treatment.
  • Participant continues to experience clinical benefit.

Key Exclusion Criteria:

  • Evidence of uncontrolled infection.
  • Pregnant or nursing women.
  • Cardiac or gastrointestinal disease.
  • Graft-Versus-Host Disease (GVHD): Signs or symptoms of acute or chronic GVHD >Grade 1 within 4 weeks of enrollment. All transplant participants must have been off all systemic immunosuppressive therapy and calcineurin inhibitors for at least 1 week before enrollment, with the exception of steroids.
  • History of or any concurrent condition, therapy, laboratory abnormality, or allergy to excipients that, in the Investigator's opinion, either may interfere with the participant's participation or results in the conclusion that it is not in the best interest of the participant to participate.
  • Participants receiving other antileukemic therapy within 14 days of start of study drug and who have not recovered from previous adverse reactions.

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Syndax Pharmaceuticals

Investigators

  • Study Director: Medical Director, Syndax Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

June 15, 2023

First Submitted That Met QC Criteria

June 15, 2023

First Posted (Actual)

June 26, 2023

Study Record Updates

Last Update Posted (Actual)

September 5, 2025

Last Update Submitted That Met QC Criteria

August 28, 2025

Last Verified

August 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SNDX-5613-0707

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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