Actively Recruiting
A Phase 1 Study of Revumenib, Azacitidine, and Venetoclax in Pediatric and Young Adult Patients With Refractory or Relapsed Acute Myeloid Leukemia
Led by St. Jude Children's Research Hospital · Updated on 2026-05-04
24
Participants Needed
10
Research Sites
26 weeks
Total Duration
On this page
Sponsors
S
St. Jude Children's Research Hospital
Lead Sponsor
S
Syndax Pharmaceuticals
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are studying the safety and potential benefits of adding a new drug called revumenib to commonly used chemotherapy drugs for children and young adults aged 1 to 30 years who have acute myeloid leukemia (AML) or acute leukemia of ambiguous lineage (ALAL) that has either not responded to treatment or has returned after treatment. This Phase 1 study aims to find a safe total dose for the combination of revumenib, azacitidine, and venetoclax, especially when taken with an anti-fungal drug. The study also looks at rates of remission and overall survival for patients treated at the recommended dose. Participants will receive the three drugs: revumenib and venetoclax given by mouth or feeding tubes, azacitidine through intravenous infusion, and intrathecal chemotherapy (including cytarabine and methotrexate) directly into the spinal fluid. The treatment starts at a low dose and may be increased or decreased based on any side effects experienced. If a patient's doctor decides that revumenib alone is beneficial, it may be continued even after a stem cell transplant, although patients stop the study treatment at the time of transplant. Those who do not have a transplant may continue the combination therapy as long as it is helpful and safe. During the study, participants will be closely monitored for side effects and their response to treatment through regular assessments over about 43 days from the start of therapy. Researchers will evaluate safety, remission status, and survival, with overall survival followed for up to one year. Patients' organ function, heart activity, and blood counts will be checked, and safety will remain a focus throughout the study period.
CONDITIONS
Brief Title
Study of Revumenib, Azacitidine, and Venetoclax in Pediatric and Young Adult Patients With Refractory or Relapsed Acute Myeloid Leukemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of acute myeloid leukemia (AML) or acute leukemia of ambiguous lineage (ALAL)
- Refractory leukemia after at least two induction chemotherapy courses or relapsed leukemia with at least 5% blasts in bone marrow or 1% blasts by flow cytometry
- Presence of specific genetic markers such as KMT2A rearrangement, NUP98 rearrangement, NPM1 mutation, or others listed
- Adequate organ function including bilirubin, creatinine clearance, and heart ejection fraction
- QTcF less than 480 msec
- Age between 1 and 30 years
- Lansky performance score of 60 or higher if under 16 years old, Karnofsky score of 60% or higher if 16 years or older
- At least 14 days since last myelosuppressive therapy with resolved toxicities
- Leukocyte count below 25,000 cells/uL
- No evidence of graft-versus-host disease if prior stem cell transplant, and off calcineurin inhibitors for at least 28 days
- Taking posaconazole or voriconazole started at least 24 hours before therapy
- Agreement to use effective contraception if of reproductive potential
You will not qualify if you...
- Pregnancy or breastfeeding
- Diagnosis of Down syndrome, acute promyelocytic leukemia, juvenile myelomonocytic leukemia, or bone marrow failure syndromes
- Uncontrolled infection
- Patients with infections controlled by antimicrobial agents are eligible
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to approximately 6 weeks initially with possible continuation depending on clinical decisions
Participants receive revumenib, azacitidine, venetoclax, and intrathecal chemotherapy. Doses of revumenib and venetoclax may be adjusted based on tolerance and response. Treatment may continue as long as beneficial and without unacceptable side effects, or until hematopoietic cell transplant (HCT).
Multiple visits for dosing and monitoring during treatment cycles
Duration - Up to 1 year
Participants who undergo hematopoietic cell transplant (HCT) will stop therapy but remain on study for monitoring. Participants continuing treatment without HCT will be monitored for safety and outcomes.
Regular follow-up visits for safety and survival assessments
Trial Site Locations
Total: 10 locations
1
Rady Children's Hospital
San Diego, California, United States, 92132
Actively Recruiting
2
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
3
Children's Healthcare of Atlanta
Atlanta, Georgia, United States, 30329
Actively Recruiting
4
Children's Mercy Hospital of Kansas City
Kansas City, Missouri, United States, 64108
Actively Recruiting
5
Memorial Sloan- Kettering Cancer Center
New York, New York, United States, 10065
Actively Recruiting
6
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
7
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
8
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Actively Recruiting
9
UT Southwestern/Simmons Cancer Center
Dallas, Texas, United States, 75390
Actively Recruiting
10
Cook Children's Medical Center
Fort Worth, Texas, United States, 76104
Actively Recruiting
Research Team
H
Hiroto Inaba, MD, PhD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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