Clinical Trials / Research Studies
AML23: A Study of Venetoclax in Combination With Conventional Chemotherapy in Pediatric Patients With Acute Myeloid Leukemia
Why are we doing this research?
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
AML23: A Collaboration Phase 2 Study of Venetoclax in Combination With Conventional Chemotherapy in Pediatric Patients With Acute Myeloid Leukemia.
Who can participate?
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
Exclusion 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
Ages
29 Days to 21 Years
Conditions
- Leukemia AML New Diagnosis
- Adult - Leukemia AML New Diagnosis
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