Study of Revumenib, Azacitidine, and Venetoclax in Pediatric and Young Adult Patients With Refractory or Relapsed Acute Myeloid Leukemia
NCT ID: NCT06177067
Last Updated: 2025-11-14
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1
24 participants
INTERVENTIONAL
2024-04-19
2027-04-30
Brief Summary
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Primary Objective
* 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).
Detailed Description
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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.
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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All Eligible Participants
All eligible patients receive the following intervention:
Revumenib, Venetoclax, Azacitidine, Intrathecal chemotherapy
Revumenib
Given by mouth (capsule or liquid solution) or liquid solution by Nasogastric tube (NG) or Gastrostomy tube (G-tube)
Venetoclax
Given by mouth (tablet) or by NG or G-tube
Azacitidine
Given intravenously (IV) infusion
intrathecal (IT) chemotherapy
Given intrathecal (IT)
Cytarabine
Given intrathecal (IT) as part of intrathecal (IT) chemotherapy.
Methotrexate
Given intrathecal (IT) as part of intrathecal (IT) chemotherapy.
Interventions
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Revumenib
Given by mouth (capsule or liquid solution) or liquid solution by Nasogastric tube (NG) or Gastrostomy tube (G-tube)
Venetoclax
Given by mouth (tablet) or by NG or G-tube
Azacitidine
Given intravenously (IV) infusion
intrathecal (IT) chemotherapy
Given intrathecal (IT)
Cytarabine
Given intrathecal (IT) as part of intrathecal (IT) chemotherapy.
Methotrexate
Given intrathecal (IT) as part of intrathecal (IT) chemotherapy.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
However, if an adequate bone marrow sample cannot be obtained (e.g., in a patient with acute megakaryoblastic leukemia with marrow fibrosis), patients may be enrolled if there is unequivocal evidence of leukemia with ≥5% blasts by morphology or ≥1% blasts flow cytometry in the blood.
* 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.
Patients who meet the criteria listed above are eligible for enrollment and treatment on the trial. However, patients in first relapse who are suitable for and willing to receive intensive remission induction therapy should be offered such therapy if deemed appropriate by the treating physician.
Exclusion Criteria
* 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.
1 Year
30 Years
ALL
No
Sponsors
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Syndax Pharmaceuticals
INDUSTRY
St. Jude Children's Research Hospital
OTHER
Responsible Party
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Principal Investigators
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Hiroto Inaba, MD, PhD
Role: PRINCIPAL_INVESTIGATOR
St. Jude Children's Research Hospital
Locations
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Rady Children's Hospital
San Diego, California, United States
Children's Mercy Hospital of Kansas City
Kansas City, Missouri, United States
Memorial Sloan- Kettering Cancer Center
New York, New York, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
St. Jude Children's Research Hospital
Memphis, Tennessee, United States
UT Southwestern/Simmons Cancer Center
Dallas, Texas, United States
Countries
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Central Contacts
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Facility Contacts
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Dennis Kuo, MD
Role: primary
Keith August, MD
Role: primary
Maria-Luisa Sulis, MD
Role: primary
Lauren Pommert, MD
Role: primary
Sarah Tasian, MD
Role: primary
Hiroto Inaba, MD, PhD
Role: primary
Kathleen Ludwig, MD
Role: primary
Related Links
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St. Jude Children's Research Hospital
Clinical Trials Open at St. Jude
Other Identifiers
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NCI-2023-10509
Identifier Type: REGISTRY
Identifier Source: secondary_id
RAVAML
Identifier Type: -
Identifier Source: org_study_id