A Trial of Epigenetic Priming in Patients With Newly Diagnosed Acute Myeloid Leukemia

The overall aim of this study is to determine if epigenetic priming with a DNA methyltransferase inhibitor (DMTi) prior to chemotherapy blocks is tolerable and carries evidence of a clinical efficacy signal as determined by minimal residual disease (MRD), event-free survival (EFS), and overall survival (OS). Tolerability for each of the agents, as well as total reduction in DNA methylation and outcome assessments will be done to simultaneously obtain preliminary biological and clinical data for each DMTi in parallel. PRIMARY OBJECTIVES: * Evaluate the tolerability of five days of epigenetic priming with azacitidine and decitabine as a single agent DMTi prior to standard AML chemotherapy blocks. * Evaluate the change in genome-wide methylation burden induced by five days of epigenetic priming and the association of post-priming genome-wide methylation burden with event-free survival among pediatric AML patients. SECONDARY OBJECTIVES * Describe minimal residual disease levels following Induction I chemotherapy in patients that receive DMTi. * Estimate the event-free survival and overall survival of patients receiving a DMTi prior to chemotherapy courses.

NCT Number	NCT03164057
Lead Sponsor	St. Jude Children's Research Hospital
Conditions	Acute Myeloid Leukemia, Myelodysplastic Syndromes
Enrollment	206 participants
Start Date	2017-06-15
Primary Completion	2025-09-20 (estimated)
Study Completion	2027-06 (estimated)
Updated on ClinicalTrials.gov	2026-04-23