Last-Resort drug access for leukemia patients

NCT ID NCT05918913

Disease control Sponsor: Syndax Pharmaceuticals Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This program provides revumenib (SNDX-5613) to patients with relapsed or refractory acute leukemias who cannot join other studies and have no approved treatments. Participants take the drug orally every 12 hours. The goal is to offer a potential treatment option in a controlled setting.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: Revumenib (also called SNDX-5613)
What this could lead to: If it works, this could offer a treatment option for people with hard-to-treat acute leukemias who have run out of standard therapies.
What could go wrong: This is an expanded access program, not a formal trial, so results are not systematically collected. The drug is still investigational and may not work for everyone; side effects are possible.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages: 6 months and older
Sex: Anyone

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

leukemia Recurrence

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Relapsed/refractory acute leukemia

Contacts and locations

Locations

Advent Health Orlando

Orlando, Florida, 32804, United States
Alabama Center for Childhood Cancer And Blood Disorders, Children's of Alabama

Birmingham, Alabama, 35233, United States
Center for Cancer and Blood Disorders, Colorado Children's Hospital

Aurora, Colorado, 80045, United States
Children's Healthcare of Atlanta

Atlanta, Georgia, 30322, United States
Children's Hospital

New Orleans, Louisiana, 70118, United States
Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
Children's Mercy Hospital-Kansas City

Kansas City, Missouri, 64108, United States
Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, 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
Dana-Farber Cancer Institute, Boston Children's Cancer and Blood Disorders Center

Boston, Massachusetts, 02215, United States
Division of Hematology and Hematologic Malignancies, University of Utah-Huntsman Cancer Hospital

Salt Lake City, Utah, 84112, 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
Doernbecher Children's Hospital, Oregon Health & Science University

Portland, Oregon, 97239, United States
Duke University Medical Center

Durham, North Carolina, 27705, United States
HCTU, Division of Hematology, University of Colorado, Anschutz Medical Center

Aurora, Colorado, 80045, United States
Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States
Lucile Packard Children's Hospital-Stanford

Palo Alto, California, 94304, United States
Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States
MD Anderson Cancer Center

Houston, Texas, 77030, United States
Memorial Hermann-Texas Medical Center

Houston, Texas, 77030, 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
OSU Medical Center

Columbus, Ohio, 43210, United States
Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States
Rady Children's Hospital

San Diego, California, 92123, United States
Seattle Children's Research Institute, Seattle Childrens Hospital

Seattle, Washington, 98105, United States
Siteman Cancer Center - Washington University

St Louis, Missouri, 63110, United States
St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States
Texas Children's Hospital

Houston, Texas, 77030, United States
UCLA, UCLA RRMC, Drug Information Center, Department of Pharmaceutical Services Drug Supply Shipment

Los Angeles, California, 90095, United States
University of California, San Francisco

San Francisco, California, 94158, United States
University of Chicago Medical Center

Chicago, Illinois, 60637, United States
University of Iowa Hospitals and Clinics

Iowa City, Iowa, 52242, United States
Winship Cancer Institute at Emory University

Atlanta, Georgia, 30322, United States