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

Launched by ST. JUDE CHILDREN'S RESEARCH HOSPITAL · Jul 12, 2023

Keywords

ClinConnect Summary

This clinical trial is investigating the use of a medication called venetoclax along with standard chemotherapy to treat children and young adults with acute myeloid leukemia (AML), a type of blood cancer. The main goal is to see if this combination is safe and effective, particularly in helping patients achieve a state where very few cancer cells are detectable after treatment. The study is currently recruiting participants aged from just over 28 days to 22 years who have been diagnosed with AML and haven't received extensive treatment before.

Eligible participants will receive venetoclax and chemotherapy, and doctors will monitor how well the treatment works and whether it causes any side effects. Patients will need to meet specific health criteria, such as having a certain number of cancer cells in their blood or bone marrow. Additionally, participants who can become pregnant will need to confirm they are not pregnant and agree to use birth control during the study. This trial is a chance for young patients to receive a potentially promising treatment while contributing to important research that could improve future care for AML.

Gender

ALL

Eligibility criteria

• 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:
• • 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.