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

The film gives the main points. The full list below is copied from the registry; the study team makes the final decision.

• Age29 Days to 21 Years
• SexAll
• Healthy volunteersNo

You may be able to join if

• Patients with preexisting conditions that may affect chemotherapy tolerance (e.g., bone marrow failure syndrome, cardiac disease, pulmonary disease, and history of significant hepatic impairment) should be discussed with study PI in detail to enable review of the medical history prior to enrollment onto AML23 study.
• Inclusion Criteria:
• 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 cannot join 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.

This is how the treatment is meant to work. Whether it helps, and how safe it is, is what this trial is testing.

venetoclax. An orally bioavailable, selective small molecule inhibitor of the anti-apoptotic protein Bcl-2, with potential antineoplastic activity. Venetoclax mimics BH3-only proteins, the native ligands of Bcl-2 and apoptosis activators, by binding to the hydrophobic groove of Bcl-2 proteins thereby repressing Bcl-2 activity and restoring apoptotic processes in tumor cells. Bcl-2 protein is overexpressed in some cancers and plays an important role in the regulation of apoptosis; its expression is associated with increased drug resistance and tumor cell survival. Compared to the Bcl-2 inhibitor navitoclax, this agent does not inhibit bcl-XL and does not cause bcl-XL-mediated thrombocytopenia. NCI Drug Dictionary

Treatments listed in the registry

• Venetoclax (Drug): Venetoclax will be given with each course of therapy. Patients with low-risk AML will receive four courses of therapy, intermediate-risk patients will receive five courses of therapy, and high-risk patients will receive two or three courses of therapy followed by hematopoietic stem cell transplantation.
• Azacitidine (Drug): Given IV over 30 minutes on days 1-5
• Cytarabine (Drug): Given IV over 30 minutes q12 hours on days 1-8 (16 doses)
• Gemtuzumab Ozogamicin (Drug): Given IV
• Daunorubicin Hydrochloride (Drug): IV over 1 hour on days 1, 3, and 5
• Fludarabine Phosphate (Drug): Given IV over 30 minutes on days 1-5
• Idarubicin Hydrochloride (Drug): Given IV over 15 minutes on days 3-5
• Mitoxantrone Hydrochloride (Drug): IV over 1 hour on days 2-4
• Etoposide (Drug): Given IV over 1 hour on days 1-5
• Gilteritinib (Drug): PO on days 8-28 (21 doses)

Non Randomized · Parallel · masking: None

Main goals

• Minimal residual disease (MRD)-negativity rate (At day 29 after induction 1)
• Incidence of death or unacceptable adverse event (From initiation to completion of each course of therapy, an average of 6 weeks)

Other goals

• Event-free survival
• Overall survival

Planned enrollment: 70 (estimated).

• St. Jude Children's Research Hospital, Memphis, Tennessee

Site status as of Oct 30, 2026. Check the live record before contacting a site.

• ClinicalTrials.gov NCT05955261
• NCI Drug Dictionary: C103147

Registry record reviewed as fetched Oct 30, 2026, last updated by the sponsor Sep 16, 2026. Public record; not medical advice.