Study of Revumenib, Azacitidine, and Venetoclax in Pediatric and Young Adult Patients With Refractory or Relapsed Acute Myeloid Leukemia
A Phase 1 Study of Revumenib, Azacitidine, and Venetoclax in Pediatric and Young Adult Patients With Refractory or Relapsed Acute Myeloid Leukemia
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 1 Year to 30 Years
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Not stated
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
Not stated in the study description — ask the study team about visits and procedures.
- Compensation
- Not stated
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
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 * 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).
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Official eligibility criteria
Inclusion Criteria: Participants must have a diagnosis of AML or ALAL and meet the criteria below: * 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. 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 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.
Study sites (10)
CA
- Rady Children's Hospital · San Diego, CA 92132Dennis Kuo, MD · Study contact · 858-966-5811 · djkuo@rchsd.orgDennis Kuo, MD · Principal investigatorMore trials in San Diego
CO
- Children's Hospital Colorado · Aurora, CO 80045Kelly Faulk, MD · Study contact · 720-777-6503 · Kelly.Faulk@childrenscolorado.orgKelly Faulk, MD · Principal investigatorMore trials in Aurora
GA
- Children's Healthcare of Atlanta · Atlanta, GA 30329Himalee Sabnis, MD · Study contact · 404-727-3285 · hsabnis@emory.eduHimalee Sabnis, MD · Principal investigatorMore trials in Atlanta
MO
- Children's Mercy Hospital of Kansas City · Kansas City, MO 64108Keith August, MD · Study contact · 816-302-6808 · kjaugust@cmh.eduKeith August, MD · Principal investigatorMore trials in Kansas City
NY
- Memorial Sloan- Kettering Cancer Center · New York, NY 10065Maria-Luisa Sulis, MD · Study contact · 212-639-5175 · sulism@mskcc.orgMaria-Luisa Sulis, MD · Principal investigatorMore trials in New York
OH
- Cincinnati Children's Hospital Medical Center · Cincinnati, OH 45229Lauren Pommert, MD · Study contact · 513-803-9083 · lauren.pommert@cchmc.orgLauren Pommert, MD · Principal investigatorMore trials in Cincinnati
PA
- Children's Hospital of Philadelphia · Philadelphia, PA 19104Sarah Tasian, MD · Study contact · 267-425-0118 · tasians@email.chop.eduSarah Tasian, MD · Principal investigatorMore trials in Philadelphia
TN
- St. Jude Children's Research Hospital · Memphis, TN 38105Hiroto Inaba, MD, PhD · Study contact · 888-226-4343 · referralinfo@stjude.orgHiroto Inaba, MD, PhD · Principal investigatorMore trials in Memphis
TX
- UT Southwestern/Simmons Cancer Center · Dallas, TX 75390Kathleen Ludwig, MD · Study contact · 214-456-6927 · kathleen.wiertel@utsouthwestern.eduKathleen Ludwig, MD · Principal investigatorMore trials in Dallas
- Cook Children's Medical Center · Fort Worth, TX 76104Holly Pacenta, MD · Study contact · 682-885-4007 · Holly.Pacenta@cookchildrens.orgHolly Pacenta, MD · Principal investigatorMore trials in Fort Worth