NCT04065399 · Syndax Pharmaceuticals
A Study of Revumenib in R/R Leukemias Including Those With an MLL/KMT2A Gene Rearrangement or NPM1 Mutation
(AUGMENT-101)
What this study is about
Phase 1 gradually increasing doses will determine the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) of revumenib in participants with acute leukemia. In Phase 2, participants will be enrolled in 3 indication-specific expansion cohorts to determine the effectiveness, short- and long-term safety, and tolerability of revumenib.
View original scientific description
Phase 1 dose escalation will determine the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) of revumenib in participants with acute leukemia. In Phase 2, participants will be enrolled in 3 indication-specific expansion cohorts to determine the efficacy, short- and long-term safety, and tolerability of revumenib.
Interventions
DRUG
revumenib
revumenib orally
DRUG
cobicistat
Phase 1 Arm C participants will receive 150 mg cobicistat daily.
Primary outcome measures
Number of participants with dose-limiting toxicities (DLTs) (Phase 1)
Time frame: Approximately 1 year
Assessed by the NCI CTCAE version 5.0 (Phase 1)
Number of participants with treatment-emergent adverse events (TEAEs) (Phase 1)
Time frame: Approximately 1 year
Assessed by the NCI CTCAE version 5.0 (Phase 1)
Cmax (Phase 1)
Time frame: Approximately 1 year
Maximum plasma concentration (Cmax) of revumenib and relevant metabolites (Phase 1)
Tmax (Phase 1)
Time frame: Approximately 1 year
Time to observed maximum plasma concentration of revumenib and relevant metabolites (Phase 1)
AUC0-t (Phase 1)
Time frame: 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])
Time frame: 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])
Time frame: Approximately 3 years
Assessed by the NCI CTCAE version 5.0 (Phase 2 \[Cohorts 2A-2C\])
Cmax (Phase 2 [Cohort 2D])
Time frame: Approximately 3 years
Cmax of revumenib (Phase 2 \[Cohort 2D\])
AUC0-tau (Phase 2 [Cohort 2D])
Time frame: 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 \[Cohort 2D\])
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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/ind
Where
- Duarte, California
- Los Angeles, California
- Palo Alto, California
- Aurora, Colorado
- Sarasota, Florida
- Tampa, Florida
- Atlanta, Georgia
- Chicago, Illinois
- Iowa City, Iowa
- Boston, Massachusetts
- St Louis, Missouri
- Hackensack, New Jersey
And 8 more locations — see the full list below.
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Mar 18, 2026 · Source of record for eligibility and locations