Expanded Access Program for Revumenib

• Conditions: Relapsed/Refractory Acute Leukemia

• Registration Number: NCT05918913
• Lead Sponsor: Syndax Pharmaceuticals

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2023-06-15
• Study First Submitted QC: 2023-06-15
• Study First Posted: 2023-06-26
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-28
• Last Update Posted: 2025-05-01

Recruitment & Eligibility

• Status: AVAILABLE
• Sex: All
• Target Recruitment: Not specified

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.

Study & Design

• Study Type: EXPANDED_ACCESS
• Study Design: Not specified

Trial Locations

Locations (35)

UCLA, UCLA RRMC, Drug Information Center, Department of Pharmaceutical Services Drug Supply Shipment; 🇺🇸 Los Angeles, California, United States

University of California, San Francisco; 🇺🇸 San Francisco, California, United States

Rady Children's Hospital; 🇺🇸 San Diego, California, United States

Phoenix Children's Hospital; 🇺🇸 Phoenix, Arizona, United States

Alabama Center for Childhood Cancer And Blood Disorders, Children's of Alabama; 🇺🇸 Birmingham, Alabama, United States

City of Hope; 🇺🇸 Duarte, California, United States

City of Hope at Orange County Lennar Foundation Cancer Center; 🇺🇸 Irvine, California, United States

Advent Health Orlando; 🇺🇸 Orlando, Florida, United States

Children's Healthcare of Atlanta; 🇺🇸 Atlanta, Georgia, United States

Winship Cancer Institute at Emory University; 🇺🇸 Atlanta, Georgia, United States

Lurie Children's Hospital of Chicago; 🇺🇸 Chicago, Illinois, United States

Lucile Packard Children's Hospital-Stanford; 🇺🇸 Palo Alto, California, United States

Center for Cancer and Blood Disorders, Colorado Children's Hospital; 🇺🇸 Aurora, Colorado, United States

HCTU, Division of Hematology, University of Colorado, Anschutz Medical Center; 🇺🇸 Aurora, Colorado, United States

University of Chicago Medical Center; 🇺🇸 Chicago, Illinois, United States

University of Iowa Hospitals and Clinics; 🇺🇸 Iowa City, Iowa, United States

Children's Hospital; 🇺🇸 New Orleans, Louisiana, United States

Dana-Farber Cancer Institute, Boston Children's Cancer and Blood Disorders Center; 🇺🇸 Boston, Massachusetts, United States

Children's Mercy Hospital-Kansas City; 🇺🇸 Kansas City, Missouri, United States

Siteman Cancer Center - Washington University; 🇺🇸 Saint Louis, Missouri, United States

Hackensack University Medical Center; 🇺🇸 Hackensack, New Jersey, United States

Montefiore Medical Center; 🇺🇸 Bronx, New York, United States

Memorial Sloan Kettering Cancer Center; 🇺🇸 Long Island City, New York, United States

Duke University Medical Center; 🇺🇸 Durham, North Carolina, United States

Cincinnati Children's Hospital Medical Center; 🇺🇸 Cincinnati, Ohio, United States

Division of Hematology and Oncology, Division of Pulmonary, Critical Care and Sleep Medicine, Vontz Center for Molecular Studies; 🇺🇸 Cincinnati, Ohio, United States

OSU Medical Center; 🇺🇸 Columbus, Ohio, United States

Doernbecher Children's Hospital, Oregon Health & Science University; 🇺🇸 Portland, Oregon, United States

Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States

St. Jude Children's Research Hospital; 🇺🇸 Memphis, Tennessee, United States

MD Anderson Cancer Center; 🇺🇸 Houston, Texas, United States

Memorial Hermann-Texas Medical Center; 🇺🇸 Houston, Texas, United States

Texas Children's Hospital; 🇺🇸 Houston, Texas, United States

Division of Hematology and Hematologic Malignancies, University of Utah-Huntsman Cancer Hospital; 🇺🇸 Salt Lake City, Utah, United States

Seattle Children's Research Institute, Seattle Childrens Hospital; 🇺🇸 Seattle, Washington, United States