Expanded Access Program for Revumenib
Expanded Access Program for SNDX-5613 in Patients With Relapsed/Refractory Acute Leukemias With Genetic Alterations Associated With HOXA Overexpression
1 other identifier
expanded_access
N/A
1 country
35
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
35 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 15, 2023
CompletedFirst Posted
Study publicly available on registry
June 26, 2023
CompletedSeptember 5, 2025
August 1, 2025
June 15, 2023
August 28, 2025
Conditions
Keywords
Interventions
Eligibility Criteria
You may qualify if:
- 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:
- R/R leukemia is defined by the presence of ≥5% blasts in the bone marrow and/or persistence or reappearance of peripheral blasts.
- 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.
- Participants with isolated extra-medullary disease are allowed if extramedullary disease was confirmed with biopsy.
- 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.
- 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.
- +4 more criteria
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (35)
Alabama Center for Childhood Cancer And Blood Disorders, Children's of Alabama
Birmingham, Alabama, 35233, United States
Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
City of Hope
Duarte, California, 91010, United States
City of Hope at Orange County Lennar Foundation Cancer Center
Irvine, California, 92618, United States
UCLA, UCLA RRMC, Drug Information Center, Department of Pharmaceutical Services Drug Supply Shipment
Los Angeles, California, 90095, United States
Lucile Packard Children's Hospital-Stanford
Palo Alto, California, 94304, United States
Rady Children's Hospital
San Diego, California, 92123, United States
University of California, San Francisco
San Francisco, California, 94158, United States
Center for Cancer and Blood Disorders, Colorado Children's Hospital
Aurora, Colorado, 80045, United States
HCTU, Division of Hematology, University of Colorado, Anschutz Medical Center
Aurora, Colorado, 80045, United States
Advent Health Orlando
Orlando, Florida, 32804, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322, United States
Winship Cancer Institute at Emory University
Atlanta, Georgia, 30322, United States
Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
University of Chicago Medical Center
Chicago, Illinois, 60637, United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242, United States
Children's Hospital
New Orleans, Louisiana, 70118, United States
Dana-Farber Cancer Institute, Boston Children's Cancer and Blood Disorders Center
Boston, Massachusetts, 02215, United States
Children's Mercy Hospital-Kansas City
Kansas City, Missouri, 64108, United States
Siteman Cancer Center - Washington University
St Louis, Missouri, 63110, United States
Hackensack University Medical Center
Hackensack, New Jersey, 07601, United States
Memorial Sloan Kettering Cancer Center
Long Island City, New York, 10065, United States
Montefiore Medical Center
The Bronx, New York, 10467, United States
Duke University Medical Center
Durham, North Carolina, 27705, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
Division of Hematology and Oncology, Division of Pulmonary, Critical Care and Sleep Medicine, Vontz Center for Molecular Studies
Cincinnati, Ohio, 45267, United States
OSU Medical Center
Columbus, Ohio, 43210, United States
Doernbecher Children's Hospital, Oregon Health & Science University
Portland, Oregon, 97239, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States
MD Anderson Cancer Center
Houston, Texas, 77030, United States
Memorial Hermann-Texas Medical Center
Houston, Texas, 77030, United States
Texas Children's Hospital
Houston, Texas, 77030, United States
Division of Hematology and Hematologic Malignancies, University of Utah-Huntsman Cancer Hospital
Salt Lake City, Utah, 84112, United States
Seattle Children's Research Institute, Seattle Childrens Hospital
Seattle, Washington, 98105, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Medical Director
Syndax Pharmaceuticals
Study Design
- Study Type
- expanded access
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 15, 2023
First Posted
June 26, 2023
Last Updated
September 5, 2025
Record last verified: 2025-08