A Phase Ia/Ib Open-label, Multiple Dose, Study to Determine the Recommended Dose, Evaluate PKs, PDs, Safety, and Activity of Venetoclax in Combination With Oral Decitabine/Cedazuridine (ASTX727) in Pediatric Patients With Relapsed/Refractory Acute Myeloid Leukemia (AML)
M.D. Anderson Cancer Center
Summary
To find a recommended dose of ASTX727 (cedazuridine/decitabine) in combination with venetoclax for pediatric patients with relapsed AML.
Description
Primary Objectives 1. To determine the recommended dose of ASTX727 when given in combination with venetoclax in relapsed/refractory (R/R) pediatric participants with acute myeloid leukemia (AML). 2. To evaluate and describe the toxicities of ASTX727 and venetoclax administered in R/R AML pediatric population. 3. To characterize the pharmacokinetic (PK) and pharmacodynamic (PD) profile of ASTX727 alone and in combination with venetoclax in an R/R AML pediatric population. Secondary Objectives 1. To evaluate the potential for drug-drug interactions: effect of venetoclax on PK of ASTX727. 2. T…
Eligibility
- Age range
- 2–18 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Pediatric Participants with Relapsed/refractory AML by WHO criteria. 2. Participants must have ≥ 5% blasts in the bone marrow as assessed by morphology or flow cytometry. However, if an adequate bone marrow sample cannot be obtained, participants may be enrolled if there is unequivocal evidence of leukemia with ≥ 5% blasts in the peripheral blood or an AML defining genetic abnormality as specified by the WHO 2022 criteria. 3. Performance status: Lansky ≥ 50 for participants who are ≤ 16 years old and Karnofsky ≥ 50% for participants who are \> 16 years old. 4. Age ≥2 ye…
Interventions
- DrugASTX727
Given by PO
- DrugVenetoclax
Given by PO
Location
- MD Anderson Cancer CenterHouston, Texas