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Refractory Acute Myeloid LeukemiaPhase 1NCT06177067

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

A Phase 1 study testing Revumenib, Venetoclax and Azacitidine in people with Refractory Acute Myeloid Leukemia and Relapsed Acute Myeloid Leukemia, run by St. Jude Children's Research Hospital at 10 sites in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record

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Trial summary

This is a research study to find out if adding a new study drug called revumenib to commonly used chemotherapy drugs is safe and if they have beneficial effects in treating patients with acute myeloid leukemia (AML) or acute leukemia of ambiguous lineage (ALAL) that did not go into remission after treatment… Read more about this study

Who is it for?
Ages 1 year to 30 years
What is being tested?
Revumenib, Venetoclax and Azacitidine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
24 participants
Where
10 sitesUnited States
Study dates
19 Apr 2024to Feb 2029
Phase
Phase 1

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 1 year to 30 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

None listed.

Who cannot join

  • 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.

Trial locations and contacts

10 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Hiroto Inaba, MD, PhD contact

888-226-4343

referralinfo@stjude.org

10 sites in 1 country

United States10 sites, 10 recruiting

  • Children's Healthcare of Atlanta

    Atlanta, Georgia, 30329

    Himalee Sabnis, MD, 404-727-3285, hsabnis@emory.edu

    Himalee Sabnis, MD

    Recruiting

  • Children's Hospital Colorado

    Aurora, Colorado, 80045

    Kelly Faulk, MD, 720-777-6503, Kelly.Faulk@childrenscolorado.org

    Kelly Faulk, MD

    Recruiting

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229

    Lauren Pommert, MD, 513-803-9083, lauren.pommert@cchmc.org

    Lauren Pommert, MD

    Recruiting

  • UT Southwestern/Simmons Cancer Center

    Dallas, Texas, 75390

    Kathleen Ludwig, MD, 214-456-6927, kathleen.wiertel@utsouthwestern.edu

    Kathleen Ludwig, MD

    Recruiting

  • Cook Children's Medical Center

    Fort Worth, Texas, 76104

    Holly Pacenta, MD, 682-885-4007, Holly.Pacenta@cookchildrens.org

    Holly Pacenta, MD

    Recruiting

  • Children's Mercy Hospital of Kansas City

    Kansas City, Missouri, 64108

    Keith August, MD, 816-302-6808, kjaugust@cmh.edu

    Keith August, MD

    Recruiting

  • St. Jude Children's Research Hospital

    Memphis, Tennessee, 38105

    Hiroto Inaba, MD, PhD, 888-226-4343, referralinfo@stjude.org

    Hiroto Inaba, MD, PhD

    Recruiting

  • Memorial Sloan- Kettering Cancer Center

    New York, New York, 10065

    Maria-Luisa Sulis, MD, 212-639-5175, sulism@mskcc.org

    Maria-Luisa Sulis, MD

    Recruiting

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

    Sarah Tasian, MD, 267-425-0118, tasians@email.chop.edu

    Sarah Tasian, MD

    Recruiting

  • Rady Children's Hospital

    San Diego, California, 92132

    Dennis Kuo, MD, 858-966-5811, djkuo@rchsd.org

    Dennis Kuo, MD

    Recruiting

Other trials in United States

About this study

Official title: A Phase 1 Study of Revumenib, Azacitidine, and Venetoclax in Pediatric and Young Adult Patients With Refractory or Relapsed Acute Myeloid Leukemia

This is a research study to find out if adding a new study drug called revumenib to commonly used chemotherapy drugs is safe and if they have beneficial effects in treating patients with acute myeloid leukemia (AML) or acute leukemia of ambiguous lineage (ALAL) that did not go into remission after treatment (refractory) or has come back after treatment (relapsed), and to determine the total dose of the 3-drug combination of revumenib, azacitidine and venetoclax that can be given safely in participants also taking an anti-fungal drug.

Primary Objective

Read more
  • To determine the safety and tolerability of revumenib + azacitidine + venetoclax in pediatric patients with relapsed or refractory AML or ALAL.

Secondary Objectives

  • Describe the rates of complete remission (CR), complete remission with incomplete count recovery (CRi), and overall survival for patients treated with revumenib + azacitidine + venetoclax at the recommended phase 2 dose (RP2D).
Read the detailed description

Patients will receive revumenib + azacitidine + venetoclax in a dose-escalation fashion. The protocol starts at dose level 1. If there are no dose limiting toxicities at dose level 1, then patients will be treated at dose level 2, which equates to the dose of revumenib increasing from 65 to 95 mg/m^2 while the venetoclax exposure remains the same at 21 days. Alternatively, if there are dose limiting toxicities at dose level 1, then the dose level will be deescalated to dose level -1, which equates to the length of exposure of venetoclax being decreased from 21 days to 14 days, while the dose of revumenib stays at 65 mg/m^2. The doses of azacitidine will remain constant at all dose levels.

For patients whose primary physician considers that single agent revumenib is beneficial (e.g., transition to hematopoietic cell transplant), revumenib can be continued after discussing with study principal investigator. Patients who undergo HCT will be taken off therapy at the time of HCT, but will remain on study. Post-transplant therapy will be determined by the HCT physician.

Patients who do not go on to receive an HCT, may continue to receive revumenib, venetoclax and azacitidine as long as their primary physician considers it beneficial and there are no unacceptable side effects.

Being tested
Revumenib, Venetoclax, Azacitidine, intrathecal (IT) chemotherapy, Cytarabine
Medical subject headings
Leukemia, Myeloid, Acute, Leukemia, Biphenotypic, Acute

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • RevumenibDrug

    Also called SNDX-5613

    Given by mouth (capsule or liquid solution) or liquid solution by Nasogastric tube (NG) or Gastrostomy tube (G-tube)

  • VenetoclaxDrug

    Also called Venclextra®

    Given by mouth (tablet) or by NG or G-tube

  • AzacitidineDrug

    Also called VIDAZA®, 5-azacitidine

    Given intravenously (IV) infusion

  • intrathecal (IT) chemotherapyDrug

    Also called ITMHA, methotrexate/hydrocortisone/cytarabine

    Given intrathecal (IT)

  • CytarabineDrug

    Also called Ara-C, Cytosar®

    Given intrathecal (IT) as part of intrathecal (IT) chemotherapy.

  • MethotrexateDrug

    Also called MTX, Trexall®

    Given intrathecal (IT) as part of intrathecal (IT) chemotherapy.

Study groups in NCT06177067
Study groupRoleWhat they receive
All Eligible ParticipantsExperimentalAll eligible patients receive the following intervention: Revumenib, Venetoclax, Azacitidine, Intrathecal chemotherapy
Receives: Drug: Revumenib; Drug: Venetoclax; Drug: Azacitidine; Drug: intrathecal (IT) chemotherapy; Drug: Cytarabine; Drug: Methotrexate

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. The safety and tolerability of revumenib + azacitidine + venetoclax in pediatric patients with relapsed or refractory AML or ALAL

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

    Measured: 43 days from the start of therapy.

Secondary outcomes (3)

  1. The rates of complete remission (CR)

    CR is defined as bone marrow with < 5% blasts confirmed by flow cytometry, ANC ≥500/μL and platelets ≥50,000/μL without transfusions, and no evidence of extramedullary disease.

    Measured: 43 days from the start of therapy

  2. The rates of complete remission with incomplete count recovery (CRi)

    CRi is defined as bone marrow with <5% blasts confirmed by flow cytometry and ANC <500/μL or platelets <50,000/μL without transfusions

    Measured: 43 days from the start of therapy

  3. The overall survival of patients treated at the RP2D.

    Kaplan-Meier estimates with 95% confidence intervals will be used to describe overall survival.

    Measured: 1 year

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted20 Dec 2023
  2. Study start19 Apr 2024
  3. Primary completionJan 2027 (estimated)
  4. Study completionFeb 2029 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Model description
Participants with relapsed or refractory acute myeloid leukemia (AML) or acute leukemia of ambiguous lineage (ALAL) who meet the eligibility criteria.
Masking
None (open label)
Enrolment
24 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06177067
Sponsor's protocol ID
RAVAML
Other IDs
NCI-2023-10509
Status last verified
Oct 2026
First submitted
11 Dec 2023
Last updated
22 Oct 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: Individual participant de-identified datasets containing the variables analyzed in the published article will be made available (related to the study primary or secondary objectives contained in the publication). Supporting documents such as the protocol, statistical analyses plan, and informed consent are available through the CTG website for the specific study. Data used to generate the published article will be made available at the time of article publication. Investigators who seek access to individual level de-identified data will contact the computing team in the Department of Biostatistics (ClinTrialDataRequest@stjude.org) who will respond to the data request.

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