A Study of Venetoclax in Combination With Conventional Chemotherapy in Pediatric Patients With Acute Myeloid Leukemia

Paused · Phase 2

Conditions studied: Acute Myeloid Leukemia

In brief

This is a phase 2 study to test the hypothesis that venetoclax in combination with standard chemotherapy will be tolerable and active in pediatric patients with newly diagnosed acute myeloid leukemia (AML). Primary Objectives: * Establish the tolerability adding venetoclax to standard chemotherapy in pediatric patients with AML * Estimate the proportion of patients who become minimal residual disease (MRD) negative by flow cytometry after one course of venetoclax-based induction therapy Secondary Objectives: \- Estimate the rates of complete remission (CR), event-free survival (EFS), and overall survival (OS) in pediatric patients who receive venetoclax-based chemotherapy

Key facts

Study ID: NCT05955261
Run by: St. Jude Children's Research Hospital
People needed: 70
Starts: 2023-07-25
Expected to finish: 2034-03-01
Last updated by the study team: 2026-07-28

Who can join

Age: 0 and older, up to 21. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Diagnosis of AML fulfilling the criteria of the WHO classification of myeloid neoplasms or < 20% marrow myeloblasts and evidence of a clonal de novo AML genetic abnormality or myeloid sarcoma or primary myelodysplastic syndrome (MDS) with ≥ 10% blasts or a complete blood count with the presence of at least 1,000 blasts/μL (e.g., a WBC count ≥ 10,000/μL with ≥ 10% blasts or a WBC count ≥ 5,000/μL with ≥ 20% blasts
Age > 28 days and < 22 years
No prior therapy for this malignancy except for one dose of intrathecal therapy and hydroxyurea or low-dose cytarabine (≤ 200 mg/m\^2 per day for ≤ 7 days)
Female patients of childbearing potential must have a negative pregnancy test within 2 weeks prior to enrollment
Male and female participants of reproductive potential must agree to use an effective contraceptive method during the study and for 6 months after study treatment
Written informed consent from the patient and/or parent/legal guardian
Direct bilirubin ≤ 1.5 x institutional upper limit of normal

You may not qualify if…

Patients with treatment-related AML, Down syndrome, acute promyelocytic leukemia, chronic myeloid leukemia in blast crisis, juvenile myelomonocytic leukemia, Fanconi anemia, Kostmann syndrome, Shwachman syndrome, or other bone marrow failure syndromes are not eligible
Uncontrolled systemic fungal, bacterial, or viral infection or significant concurrent disease that would compromise patient safety or compliance, study participation, follow up, or interpretation of study results
Prior exposure to any dose of anthracycline or anthracenedione
Patients may not receive strong or moderate CYP3A inducers, such as rifampin, within 3 days of enrollment
Patients may not receive moderate or strong CYP3A inhibitors (e.g., ketoconazole, itraconazole, voriconazole, posaconazole) within 3 days of enrollment.

Where it is running

Valley Children's Hospital — Madera, California, United States
Children's Hospital of Orange County — Orange, California, United States
Rady Children's Hospital-San Diego — San Diego, California, United States
Children's National Medical Center — Washington D.C., District of Columbia, United States
Ann & Robert H. Lurie Children's Hospital — Chicago, Illinois, United States
Dana-Farber Cancer Institute — Boston, Massachusetts, United States
Novant Health Presbyterian Medical Center — Charlotte, North Carolina, United States
Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
St. Jude Children's Research Hospital — Memphis, Tennessee, United States
Cook Children's Medical Center — Fort Worth, Texas, United States

Full record on ClinicalTrials.gov

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