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Study of Revumenib, Azacitidine, and Venetoclax for Relapsed or Refractory AML/ALAL in Children and Young Adults

This research study is looking at whether adding a new drug called revumenib to two commonly used chemotherapy drugs, azacitidine and venetoclax, is safe and effective for children and young adults (ages 1 to 30) with acute myeloid leukemia (AML) or acute leukemia of ambiguous lineage (ALAL). These are types of blood cancer that have either not responded to previous treatments (refractory) or have come back after treatment (relapsed). The main goal is to find the safest dose of this three-drug combination. Revumenib and venetoclax are given by mouth, while azacitidine is given through an IV. Intrathecal (IT) chemotherapy, including cytarabine, may also be given directly into the spinal fluid. The study will measure the safety and tolerability of this combination after 43 days of treatment.

Study design
This is an interventional study with a planned enrollment of 24 participants. It will use a dose-escalation approach to find the safest dose of the drug combination.
What's involved
Participants will receive revumenib, azacitidine, and venetoclax. The specific doses of revumenib and venetoclax may change based on how well participants tolerate the treatment.
Compensation
Not stated in the trial record.
Follow-up
The safety and tolerability of the treatment will be measured at 43 days from the start of therapy. Participants may continue on revumenib, venetoclax, and azacitidine as long as it's beneficial and side effects are manageable, or until hematopoietic cell transplant (HCT).

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

NCT06177067

Study of Revumenib, Azacitidine, and Venetoclax in Pediatric and Young Adult Patients With Refractory or Relapsed Acute Myeloid Leukemia

Recruiting
PHASE1Ages 1–30InterventionalTreatment
St. Jude Children's Research Hospital
~24 participants
Updated 2026-08-03 on ClinicalTrials.gov
What's tested:RevumenibVenetoclaxAzacitidineintrathecal (IT) chemotherapyCytarabineMethotrexate

At a glance

Recruiting sites
10 of 10 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
The safety and tolerability of revumenib + azacitidine + venetoclax in pediatric patients with relapsed or refractory AML or ALAL
Measured over 43 days from the start of therapy.

Conditions

Refractory Acute Myeloid Leukemia
Relapsed Acute Myeloid Leukemia
Acute Leukemia of Ambiguous Lineage

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Where it's being run

10 sites across 9 states
Texas2
California1
Colorado1
Georgia1
Missouri1
New York1
Ohio1
Pennsylvania1

Study leadership

  • Hiroto Inaba, MD, PhD · PRINCIPAL_INVESTIGATOR · St. Jude Children's Research Hospital

Who to contact

Hiroto Inaba, MD, PhD
[email protected]
888-226-4343
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Refractory leukemia, defined as persistent leukemia after at least two courses of induction chemotherapy (one course for secondary AML), or relapsed leukemia, defined as the re-appearance of leukemia after the achievement of remission. Patients must have ≥5% blasts in the bone marrow as assessed by morphology or ≥1% blasts flow cytometry.
Presence of KMT2A rearrangement (KMT2Ar), NUP98 rearrangement (NUP98r), NPM1 mutation or fusion, PICALM::MLLT10, DEK::NUP214, UBTF-TD, KAT6A rearrangement (KAT6Ar), or SET::NUP214
Adequate organ function, defined as total bilirubin \< 1.5 × institutional upper limit of normal for age or normal conjugated bilirubin (for patients with known Gilbert's syndrome, total bilirubin \<3 × the ULN) unless attributed to leukemia, calculated creatinine clearance ≥60 mL/min/1.73 m\^2, and left ventricular ejection fraction ≥ 40%
QTcF \< 480 msec (average of triplicate)
Age ≥ 1 year and ≤ 30 years. The upper age limit may be defined by each institution, but may not exceed 30 years.
Lansky ≥ 60 for patients who are \< 16 years old and Karnofsky ≥ 60% for patients who are \> 16 years old.
At least 14 days or 5 half-lives (whichever is longer) must have elapsed since the completion of myelosuppressive therapy, with the exception of low-dose therapy used for cytoreduction according to institutional standards, such as hydroxyurea or low-dose cytarabine (up to 200 mg/m\^2/day). In addition, all toxicities must have resolved to grade 1 or less.
Patients must have a leukocyte count \<25,000 cells/uL. Low-dose therapy, such as hydroxyurea or cytarabine as described above, to achieve this limit is acceptable.
For patients who have received prior HCT, there can be no evidence of GVHD and greater than 60 days must have elapsed since the HCT, and patients should be off calcineurin inhibitors for at least 28 days prior to the start of protocol therapy. Physiologic prednisone for the treatment of adrenal insufficiency is acceptable..
Patients must be taking posaconazole or voriconazole, which must be started at least 24 hours prior to the start of therapy.
Patients of reproductive potential must agree to use effective contraception for the duration of study participation.

Exclusion

Patients who are pregnant or breastfeeding are not eligible.
Patients with Down syndrome, acute promyelocytic leukemia, juvenile myelomonocytic leukemia, or bone marrow failure syndromes are not eligible.
Patients with uncontrolled infection are not eligible. Patients with infections that are controlled on concurrent anti-microbial agents are eligible.

What this trial measures

  • The safety and tolerability of revumenib + azacitidine + venetoclax in pediatric patients with relapsed or refractory AML or ALAL43 days from the start of therapy.

    The primary endpoint is the recommended phase 2 dose (RP2D) of revumenib + azacitidine + venetoclax.