Phase III Study of ASTX727, Venetoclax, and Gilteritinib for Treating Adults With FLT3-Mutated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

What this Trial Is About

Researchers are studying a combination of drugsASTX727 decitabine and cedazuridine, venetoclax, and gilteritinibin adults with FLT3-mutated acute myeloid leukemia AML or high-risk myelodysplastic syndrome MDS. This phase III trial aims to find the best dose of these drugs together and to evaluate their effects on patients with newly diagnosed, relapsed, or refractory AML, or high-risk MDS. The study also explores how genetic factors and disease markers affect treatment response and survival. Participants receive treatment in three phases. During induction cycle 1, they take oral decitabine and cedazuridine daily for five days, and venetoclax plus gilteritinib daily for 28 days. In consolidation cycles 2-24, decitabine and cedazuridine are given for five days, gilteritinib daily for 28 days, and venetoclax daily for 14 days per 28-day cycle. In maintenance cycles beyond 24, only gilteritinib is taken daily for 28 days per cycle. Treatment continues as long as the disease does not progress and side effects remain acceptable. Throughout the study, participants are monitored with various assessments, including response rates, survival, and safety up to two years. After completing treatment, follow-up visits occur 30 days later and then every six months to evaluate long-term effects. Researchers also study minimal residual disease, genetic changes, and leukemia stem cells to understand treatment impact better. The total study length depends on individual response and tolerance.

ASTX727, Venetoclax, and Gilteritinib for the Treatment of Newly Diagnosed, Relapsed or Refractory FLT3-Mutated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome