New hope for tough leukemias: experimental pill targets genetic weakness

NCT ID NCT04065399

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

First seen Jun 27, 2026 · Last updated Sep 17, 2026 · Updated 2 times

Summary

This study tests an oral drug called revumenib in people with certain types of leukemia that have relapsed or not responded to standard therapy. The trial has two parts: first, finding the best dose, then testing that dose in four groups based on leukemia type. About 447 participants will take part to see if the drug can shrink the cancer and how safe it is.

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), taken orally
What this could lead to: If successful, this could provide a new treatment option for people with hard-to-treat leukemias that have specific genetic changes.
What could go wrong: This is an early-phase trial, so the drug may not work or could cause serious side effects. It is only for patients who have not responded to other treatments.

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.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 447 people

The number the study aims to enrol. It can still change while the study runs.

Started

Nov 2019

Expected to finish

Dec 2027

An estimate. End dates often move.

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

30 days and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

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: Participants must have active acute leukemia (bone marrow blasts ≥5% or reappearance of blasts in peripheral blood) as defined by the National Comprehensive Cancer Network (NCCN) in the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Acute Lymphoblastic Leukemia (Version 1.2020) and Acute Myeloid Leukemia (Version 3.2020), or acute leukemia harboring KMT2A rearrangement, NUP98 rearrangement, or NPM1 mutation that have detectable disease in the bone marrow. 1. Phase 1: * Arm A: Participants not receiving any strong CYP3A4 inhibitor/inducers or fluconazole. * Arm B: Participants receiving itraconazole, ketoconazole, posaconazole, or voriconazole (strong CYP3A4 inhibitors) for antifungal prophylaxis. * Arm C: Participants receiving revumenib in combination with cobicistat. * Arm D: Participants receiving fluconazole (moderate CYP3A4 inhibitor). * Arm E: Participants not receiving any weak, moderate, or strong CYP3A4 inhibitors/inducers. * Arm F: Participants receiving isavuconazole (moderate CYP3A4 inhibitor) for antifungal prophylaxis. 2. Phase 2: Documented R/R active acute leukemia (bone marrow blasts ≥5% or reappearance of blasts in peripheral blood) as defined by the NCCN Guidelines® for Acute Lymphoblastic Leukemia (Version 1.2020) and Acute Myeloid Leukemia (Version 3.2020). * Cohort 2A: Documented R/R ALL/MPAL with KMT2A rearrangement. * Cohort 2B: Documented R/R AML with KMT2A rearrangement. * Cohort 2C: Documented R/R AML with NPM1m. * Cohort 2D: Documented R/R acute leukemia with a genetic mutation expected to lead to HOX/MEIS upregulation (for example, KMT2Ar, NPM1m, and NUP98r), including participants who are MRD-positive by multiparametric flow cytometry or molecular methods only, and including participants with isolated extramedullary disease. 3. White blood cell count below 25,000/ microliter at time of enrollment. Participants may receive cytoreduction prior to enrollment per protocol-specified criteria. 4. Male or female participants aged ≥30 days old. Participants intended to receive SNDX-5613 in combination with cobicistat must weigh ≥35 kilograms (kg). Participants in Cohort 2D must be ≥18 years of age and have a body weight ≥40 kg. 5. Eastern Cooperative Oncology Group (ECOG) performance status score 0-2 or Karnofsky/Lansky score ≥50. 6. Any prior treatment-related toxicities resolved to ≤Grade 1 prior to enrollment, with the exception of ≤Grade 2 neuropathy or alopecia. Phase 1 and Phase 2 Cohorts 2A-2C only: 7. Radiation Therapy: At least 60 days from prior total body irradiation (TBI), craniospinal radiation and/or ≥50% radiation of the pelvis, or at least 14 days from local palliative radiation therapy (small port). 8. Stem Cell Infusion: At least 60 days must have elapsed from hematopoietic stem cell transplant and at least 4 weeks must have elapsed from donor lymphocyte infusion. 9. Immunotherapy: At least 42 days since prior immunotherapy, including tumor vaccines, and at least 21 days since receipt of chimeric antigen receptor therapy or other modified T or NK cell therapy. 10. Antileukemia Therapy: At least 14 days, or 5 half-lives, whichever is shorter, since the completion of antileukemic therapy. 11. Hematopoietic Growth Factors: At least 7 days since the completion of therapy with short-acting hematopoietic growth factors and 14 days with long-acting growth factors. 12. Biologics: At least 90 days, or 5 half-lives, whichever is shorter, since the completion of therapy with an antineoplastic biologic agent. 13. Steroids: At least 7 days since systemic glucocorticoid therapy, unless receiving physiologic dosing or cytoreductive therapy. Phase 2 Cohort 2D only: At least 14 days since any other investigational or commercially available antileukemic therapy, with the following exceptions: 1. Cytoreductive therapy with hydroxyurea, low-dose cytarabine (20 mg/square meter (m\^2)/day subcutaneously \[SC\] for 10 days) or low-dose etoposide (up to 200 mg/day orally for 10 days) may be administered concurrently with SNDX-5613. 2. Intrathecal chemotherapy for CNS prophylaxis is permitted at the treating physician's discretion. 3. Steroids at physiologic dosing (equivalent to ≤10 mg prednisone daily for participants ≥18 years or ≤10 mg/m\^2/day for participants \<18 years) or for cytoreductive therapy. 14. Adequate organ function. 15. If of childbearing potential, willing to use a highly effective method of contraception from the time of enrollment through 120 days following the last study drug dose. Key Exclusion Criteria: Participants meeting any of the following criteria are not eligible for study participation: 1. Diagnosis of active acute promyelocytic leukemia. 2. Isolated extramedullary relapse (Phase 2 Cohorts 2A-2C only). 3. Active central nervous system disease (cytologic, such as any blasts on cytospin, or radiographic). 4. Detectable human immunodeficiency virus (HIV) viral load within the previous 6 months. Participants with a known history of HIV 1/2 antibodies must have viral load testing prior to study enrollment. 5. Hepatitis B or C. 6. Pregnant or nursing women. 7. Cardiac Disease: * Any of the following within the 6 months prior to study entry: myocardial infarction, uncontrolled/unstable angina, congestive heart failure (New York Heart Association Classification Class ≥II), life-threatening, uncontrolled arrhythmia, cerebrovascular accident, or transient ischemic attack. * Corrected QT interval (QTc) \>450 milliseconds. 8. Gastrointestinal Disease: * any gastrointestinal issue of the upper GI tract that might affect oral drug absorption or ingestion (that is, gastric bypass and gastroparesis). * Cirrhosis with a Child-Pugh score of B or C. 9. Graft-Versus-Host Disease (GVHD): Signs or symptoms of acute or chronic GVHD \>Grade 0 within 4 weeks of enrollment. All transplant participants must have been off all systemic immunosuppressive therapy and calcineurin inhibitors for at least 4 weeks prior to enrollment. Participants may be on physiological doses of steroids. 10. Concurrent malignancy in the previous 2 years with the exception of basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or carcinoma in situ (for example, breast carcinoma, cervical cancer in situ, melanoma in situ) treated with potentially curative therapy, or concurrent low-grade lymphoma, that is asymptomatic and lacks bulky disease and shows no evidence of progression, and for which the participant is not receiving any systemic therapy or radiation. 11. In Phase 1 and Phase 2: Participants requiring the concurrent use of medications known or suspected to prolong the QT/QTc interval, with the exception of drugs with low risk of QT/QTc prolongation that are used as standard supportive therapies (for example, diphenhydramine, famotidine, ondansetron, Bactrim) and the azoles permitted in the relevant arms of Phase 1 and in Phase 2. Note: Other protocol defined inclusion/exclusion criteria may apply.

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Conditions

The condition(s) this trial relates to.

acute biphenotypic leukemia acute leukemia acute leukemia of ambiguous lineage acute lymphoblastic leukemia acute myeloid leukemia leukemia mixed phenotype acute leukemia Precursor Cell Lymphoblastic Leukemia-Lymphoma

As listed by the trial registrant

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

Acute leukemia of ambiguous lineage Acute lymphoblastic leukemia Acute myeloid leukemia Mixed lineage acute leukemia Mixed phenotype acute leukemia

How to take part

Only the study team decides who joins. These are the ways to reach them.

The study's own enquiry address

This study publishes an address for enquiries. See it below .
The places running it

39 sites in 11 countries. The list below names each one and where it is.
The official record

ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

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A doctor treating you

A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Show contact details

Study contacts

Contact

Email: •••••@•••••

Locations

Alfred Hospital
RECRUITING

Melbourne, 3004, Australia
Centre Hospitalier Lyon Sud
RECRUITING

Pierre-Bénite, 69495, France
Centre Hospitalier Universitaire (CHU) de Bordeaux
RECRUITING

Pessac, 33604, France
Children's Healthcare of Atlanta
COMPLETED

Atlanta, Georgia, 30329, United States
City of Hope Comprehensive Cancer Center
ACTIVE_NOT_RECRUITING

Duarte, California, 91010, United States
Dana Farber Cancer Institute
ACTIVE_NOT_RECRUITING

Boston, Massachusetts, 02215, United States
Duke University Medical Center
RECRUITING

Durham, North Carolina, 27110, United States
Emory Winship Cancer Institute
RECRUITING

Atlanta, Georgia, 30322, United States
Florida Cancer Specialists and Research Institute
ACTIVE_NOT_RECRUITING

Sarasota, Florida, 34232, United States
Galilee Medical Center
RECRUITING

Nahariya, 2210010, Israel
Hackensack University Medical Center
COMPLETED

Hackensack, New Jersey, 07601, United States
Hadassah Medical Center- Ein Kerem
RECRUITING

Jerusalem, 9112001, Israel
Hospital Centro Comprensivo de Cancer UPR
RECRUITING

San Juan, 00935, Puerto Rico
Hospital Saint-Louis - APHP
RECRUITING

Paris, 75010, France
Hospital Universitari i Politecnic La Fe de Valencia
RECRUITING

Valencia, 46026, Spain
Hospital Universitario Virgen del Rocio
RECRUITING

Seville, 41013, Spain
Huntsman Cancer Institute at the University of Utah
COMPLETED

Salt Lake City, Utah, 84112, United States
IRCCS Azienda Ospedaliero Universitaria di Bologna
RECRUITING

Bologna, 40138, Italy
IRCCS-Istituto Europeo di Oncologia
RECRUITING

Milan, 20141, Italy
Institut Catala d'Oncologia (ICO) - Hospital Duran i Reynals
RECRUITING

L'Hospitalet de Llobregat, 08908, Spain
Institut Gustave Roussy-Gustave Roussy Cancer Center -DITEP
RECRUITING

Villejuif, 94805, France
Istituto Romagnolo Per Lo Studio dei tumori Dino Amadori
RECRUITING

Meldola, 47014, Italy
Klinikum Nuernberg Nord
COMPLETED

Nuremberg, 90419, Germany
Memorial Sloan Kettering Cancer Center
ACTIVE_NOT_RECRUITING

New York, New York, 10065, United States
Moffitt Cancer Center
COMPLETED

Tampa, Florida, 33162, United States
Montefiore Medical Center
RECRUITING

New York, New York, 10467, United States
Ohio State University
RECRUITING

Columbus, Ohio, 43201, United States
Oregon Health & Science University
RECRUITING

Portland, Oregon, 97239, United States
Peter MacCallum Cancer Centre (PMCC)
ACTIVE_NOT_RECRUITING

Melbourne, Victoria, 3000, Australia
Princess Maxima Center for Pediatric Oncology
NOT_YET_RECRUITING

Utrecht, 3584 CS, Netherlands
Rabin Medical Center
RECRUITING

Petah Tikva, 4941492, Israel
Rambam Health Care Campus (RHCC)
RECRUITING

Haifa, 3109601, Israel
Royal Melbourne Hospital (RMH)
ACTIVE_NOT_RECRUITING

Parkville, Victoria, 3050, Australia
Royal North Shore Hospital
RECRUITING

Saint Leonards, 2065, Australia
S Bortolo Hospital AULSS 8 Berica
RECRUITING

Vicenza, 36100, Italy
Shaare Zedek Medical Center
RECRUITING

Jerusalem, 9103102, Israel
Sheba Medical Center
RECRUITING

Ramat Gan, 52621, Israel
Sir Charles Gairdner Hospital
RECRUITING

Nedlands, 6009, Australia
Stanford Cancer Institute
ACTIVE_NOT_RECRUITING

Palo Alto, California, 94305, United States
The Hospital for Sick Children
ACTIVE_NOT_RECRUITING

Toronto, Canada
The University of Chicago Medical Center
RECRUITING

Chicago, Illinois, 60637, United States
The University of Texas MD Anderson Cancer Center
ACTIVE_NOT_RECRUITING

Houston, Texas, 77030, United States
Universita Cattolica Fondazione Policlinico Agostino Gemelli
RECRUITING

Roma, 00168, Italy
Universitaetsklinikum Essen (AoR)
WITHDRAWN

Essen, 45147, Germany
Universitaetsklinikum Hamburg-Eppendorf
RECRUITING

Hamburg, 20246, Germany
Universitaetsmedizin Der Johannes
RECRUITING

Gutenberg, 55131, Germany
Universitaetsmedizin Greifswald
COMPLETED

Greifswald, 17475, Germany
University Health Network
RECRUITING

Toronto, M5G 2M9, Canada
University Hospital Of Ulm, Universitatsklinikum Ulm
RECRUITING

Ulm, Baden-Wurttemberg, 89081, Germany
University Of California Care Medical Group - Norris Comprehensive Cancer Center And Hospital
COMPLETED

Los Angeles, California, 90033, United States
University of Cincinnati
COMPLETED

Cincinnati, Ohio, 45267, United States
University of Colorado
RECRUITING

Aurora, Colorado, 80045, United States
University of Iowa Hospital
RECRUITING

Iowa City, Iowa, 52246, United States
University of Leipzig
RECRUITING

Leipzig, 04103, Germany
University of Pennsylvania
RECRUITING

Philadelphia, Pennsylvania, 19104, United States
Vilnius University Hospital Santaros Klinikos
RECRUITING

Vilnius, 08661, Lithuania
Washington University in St. Louis School of Medicine
RECRUITING

St Louis, Missouri, 63110, United States

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