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Study of Revumenib for Relapsed/Refractory Leukemias

This study is testing a drug called revumenib for people with acute leukemia that has come back or hasn't responded to previous treatments (relapsed/refractory). This includes specific types of leukemia with MLL/KMT2A gene rearrangements or NPM1 mutations. The study aims to find the safest and most effective dose of revumenib, and then to see how well it works and if it causes any side effects. You may be eligible if you have active acute leukemia and are at least 30 days old. The study will look at side effects and how much of the drug is in your body to determine the best dose, and then will assess the drug's effectiveness.

Study design
This is an interventional study with planned enrollment of 447 participants. It has a Phase 1 portion to find the right dose and a Phase 2 portion to evaluate effectiveness.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Primary endpoints for Phase 1, such as dose-limiting toxicities and adverse events, are measured at approximately 1 year.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT04065399

A Study of Revumenib in R/R Leukemias Including Those With an MLL/KMT2A Gene Rearrangement or NPM1 Mutation

Recruiting
PHASE1Ages 30+InterventionalTreatment
Syndax Pharmaceuticals
~447 participants
Updated 2026-03-18 on ClinicalTrials.gov
What's tested:revumenibcobicistat

At a glance

Recruiting sites
39 of 57 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Number of participants with dose-limiting toxicities (DLTs) (Phase 1)
Measured over Approximately 1 year
+8 more outcomes measured

Conditions

Acute Myeloid Leukemia
Acute Lymphoblastic Leukemia
Mixed Lineage Acute Leukemia
Mixed Phenotype Acute Leukemia
Acute Leukemia of Ambiguous Lineage

Explore related trials

Acute Biphenotypic LeukemiaAcute Leukemia of Ambiguous LineageAcute Lymphoblastic LeukemiaAcute Myeloid LeukemiaHematologic NeoplasmMixed Phenotype Acute LeukemiaKMT2A GeneNPM1 GeneNUP98 Gene

Where it's being run

57 sites across 28 states
Germany6
Israel6
Italy5
France4
California3
Australia3
Spain3
Florida2

Study leadership

  • Angela R Smith, M.D. · STUDY_DIRECTOR · Syndax Pharmaceuticals

Who to contact

Syndax Pharmaceuticals
[email protected]
781-419-1400
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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:
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.

Exclusion

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.

What this trial measures

  • Number of participants with dose-limiting toxicities (DLTs) (Phase 1)Approximately 1 year

    Assessed by the NCI CTCAE version 5.0 (Phase 1)

  • Number of participants with treatment-emergent adverse events (TEAEs) (Phase 1)Approximately 1 year

    Assessed by the NCI CTCAE version 5.0 (Phase 1)

  • Cmax (Phase 1)Approximately 1 year

    Maximum plasma concentration (Cmax) of revumenib and relevant metabolites (Phase 1)

  • Tmax (Phase 1)Approximately 1 year

    Time to observed maximum plasma concentration of revumenib and relevant metabolites (Phase 1)

  • AUC0-t (Phase 1)Approximately 1 year

    Area under the plasma concentration-time curve from time 0 to time of last measurable concentration (AUC0-t) of revumenib and relevant metabolites (Phase 1)

  • CR+CRh rate (Phase 2 [Cohorts 2A-2C])Approximately 3 years

    To assess the complete remission (CR) and complete remission with partial hematologic recovery (CRh) rate (Phase 2 \[Cohorts 2A-2C\])

  • Number of participants with TEAEs (Phase 2 [Cohorts 2A-2C])Approximately 3 years

    Assessed by the NCI CTCAE version 5.0 (Phase 2 \[Cohorts 2A-2C\])

  • Cmax (Phase 2 [Cohort 2D])Approximately 3 years

    Cmax of revumenib (Phase 2 \[Cohort 2D\])

  • AUC0-tau (Phase 2 [Cohort 2D])Approximately 3 years

    Area under the plasma concentration-time curve from time 0 to the end of the dosing interval (AUC0-tau) of revumenib (Phase 2 \[Cohort 2D\])