Phase 1 ASTX727 (oral decitabine + cedazuridine; DNA hypomethylating agent + cytidine deaminase inhibitor) with filgrastim (G‑CSF)

This Phase 1, post–allogeneic hematopoietic cell transplant (HCT) maintenance study tests whether ASTX727 plus filgrastim can be given safely, and at what dose, to help prevent relapse in children with high-risk AML who are in complete remission after HCT (including those who were refractory or relapsed before transplant). It uses 28‑day cycles for up to 12 cycles; patients receive filgrastim (granulocyte colony-stimulating factor that stimulates neutrophil production) on days 1–6 and ASTX727 (decitabine, a DNA methyltransferase–inhibiting hypomethylating agent, co-formulated with cedazuridine, a cytidine deaminase inhibitor that increases decitabine exposure) on days 2–6, with additional decitabine allowed to hit the intended dose. The sponsor is the National Cancer Institute; ASTX727 was developed by Astex Pharmaceuticals (Otsuka) with Taiho Oncology as the US commercial partner for the oral decitabine/cedazuridine program. The scientific angle is translating an “epigenetic maintenance” strategy into the pediatric post-HCT window using an oral hypomethylating backbone and growth-factor support. Today’s care after pediatric allo-HCT for high-risk AML is intensive surveillance with relapse-directed therapy (and, in select cases, donor lymphocyte infusion or targeted agents based on mutations), with no universally adopted maintenance standard.