Phase 1 Trial of Oral Cedazuridine and Decitabine with Filgrastim as Maintenance Therapy After Stem Cell Transplant in Children and Young Adults with High-Risk Acute Myeloid Leukemia

What this Trial Is About

Researchers are evaluating the safety, side effects, and best dosing of ASTX727 combined with filgrastim for children and young adults with high-risk acute myeloid leukemia AML that has returned or not responded to treatment after allogenic hematopoietic stem cell transplantation. ASTX727 combines cedazuridine, which prevents the breakdown of decitabine, and decitabine itself, which helps bone marrow produce normal blood cells and kills abnormal ones. Filgrastim enhances the activity of decitabine, and this phase I trial aims to assess if this combination is safe and tolerable post-transplant. Participants receive filgrastim by injection once daily on days 1 through 6 and ASTX727 orally once daily on days 2 through 6 of each 28-day cycle, with up to 12 cycles unless disease progresses or unacceptable toxicity occurs. Some may receive decitabine alone without cedazuridine to reach the proper dose. During the study, participants may undergo bone marrow aspiration and biopsy, blood sample collection, lumbar puncture, and diagnostic imaging. Participants are monitored throughout treatment and followed for up to one year post-transplant or until relapse. Researchers assess the maximum tolerated dose, adverse events, drug pharmacokinetics, incidence and severity of graft versus host disease and graft rejection, and event-free survival. The study includes regular evaluations of blood and bone marrow, safety monitoring, and pharmacodynamic testing to understand the treatments impact on DNA methylation and immune response.

A Study to Find the Highest Dose of Cedazuridine and Decitabine Combination With Filgrastim as a Treatment Option After Hematopoietic Stem Cell Transplant in Children With High-Risk Acute Myeloid Leukemia