Relapsed/Refractory Acute LeukemiaNCT05918913
Expanded Access Program for Revumenib
A study testing Revumenib in people with Relapsed/Refractory Acute Leukemia, run by Syndax Pharmaceuticals at 35 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 months and older
- Conditions the study is for
- Relapsed/Refractory Acute Leukemia
- What is being tested?
- Revumenib
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- 35 sitesUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- 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.
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- 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.
- 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.
Who cannot join
- 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.
Trial locations and contacts
35 sites in 1 country.
35 sites in 1 country
United States35 sites
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
Winship Cancer Institute at Emory University
Atlanta, Georgia, 30322
Center for Cancer and Blood Disorders, Colorado Children's Hospital
Aurora, Colorado, 80045
HCTU, Division of Hematology, University of Colorado, Anschutz Medical Center
Aurora, Colorado, 80045
Alabama Center for Childhood Cancer And Blood Disorders, Children's of Alabama
Birmingham, Alabama, 35233
Dana-Farber Cancer Institute, Boston Children's Cancer and Blood Disorders Center
Boston, Massachusetts, 02215
Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
University of Chicago Medical Center
Chicago, Illinois, 60637
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Division of Hematology and Oncology, Division of Pulmonary, Critical Care and Sleep Medicine, Vontz Center for Molecular Studies
Cincinnati, Ohio, 45267
OSU Medical Center
Columbus, Ohio, 43210
City of Hope
Duarte, California, 91010
Duke University Medical Center
Durham, North Carolina, 27705
Hackensack University Medical Center
Hackensack, New Jersey, 07601
MD Anderson Cancer Center
Houston, Texas, 77030
Memorial Hermann-Texas Medical Center
Houston, Texas, 77030
Texas Children's Hospital
Houston, Texas, 77030
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242
City of Hope at Orange County Lennar Foundation Cancer Center
Irvine, California, 92618
Children's Mercy Hospital-Kansas City
Kansas City, Missouri, 64108
Memorial Sloan Kettering Cancer Center
Long Island City, New York, 10065
UCLA, UCLA RRMC, Drug Information Center, Department of Pharmaceutical Services Drug Supply Shipment
Los Angeles, California, 90095
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
Children's Hospital
New Orleans, Louisiana, 70118
Advent Health Orlando
Orlando, Florida, 32804
Lucile Packard Children's Hospital-Stanford
Palo Alto, California, 94304
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Phoenix Children's Hospital
Phoenix, Arizona, 85016
Doernbecher Children's Hospital, Oregon Health & Science University
Portland, Oregon, 97239
Division of Hematology and Hematologic Malignancies, University of Utah-Huntsman Cancer Hospital
Salt Lake City, Utah, 84112
Rady Children's Hospital
San Diego, California, 92123
University of California, San Francisco
San Francisco, California, 94158
Seattle Children's Research Institute, Seattle Childrens Hospital
Seattle, Washington, 98105
Siteman Cancer Center - Washington University
St Louis, Missouri, 63110
Montefiore Medical Center
The Bronx, New York, 10467
About this study
Official title: 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.
- Conditions
- Relapsed/Refractory Acute Leukemia
- Being tested
- Revumenib
- Keywords
- SNDX-5613, Acute Leukemia, Refractory leukemia, Relapsed leukemia, KMT2A, NPM1, UBTF tandem duplication, Revumenib, Revuforj
- Medical subject headings
- Recurrence, Leukemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RevumenibDrug
Also called SNDX-5613, Revuforj
Revumenib administered orally every 12 hours.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jun 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Relapsed/Refractory Acute LeukemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Syndax Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Syndax Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05918913
- Sponsor's protocol ID
- SNDX-5613-0707
- Status last verified
- Aug 2025
- First submitted
- 15 Jun 2023
- Last updated
- 5 Sep 2025
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access Program for SNDX-5613 in Patients With Relapsed/Refractory Acute Leukemias With Genetic Alterations Associated With HOXA Overexpression
- ClinicalTrials.gov ID
- NCT05918913
- Acronym / sponsor ID
- SNDX-5613-0707
- Conditions
- Relapsed/Refractory Acute Leukemia
- Keywords
- SNDX-5613, Acute Leukemia, Refractory leukemia, Relapsed leukemia, KMT2A, NPM1, UBTF tandem duplication, Revumenib
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Syndax PharmaceuticalsIndustry (pharma, biotech, devices)
- First posted
- 26 Jun 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
Who may qualify
- Ages
- 6 months and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (19 listed)
- 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:
- 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.
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RevumenibAlso: SNDX-5613, RevuforjRevumenib administered orally every 12 hours. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Sep 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Syndax Pharmaceuticals
35 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Revumenib in people with Relapsed/Refractory Acute Leukemia, run by Syndax Pharmaceuticals at 35 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 19 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 5 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 35 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Sep 2025.