Phase II Study of Ivosidenib as Maintenance Treatment for Adults with IDH1-mutated Acute Myeloid Leukemia or High-risk Myelodysplastic Syndrome Who Cannot Have Transplant

What this Trial Is About

Researchers are evaluating the safety and effectiveness of ivosidenib as a maintenance treatment for patients with IDH1-mutated acute myeloid leukemia AML and high-risk myelodysplastic syndrome MDS who cannot undergo transplantation. This Phase II study also includes molecular biomarker research to better understand the treatment effects. The goal is to find an effective maintenance option for these transplant-ineligible patients. Participants will receive ivosidenib orally at a dose of 500 mg once daily in 28-day cycles. The treatment will continue until the disease progresses or unacceptable side effects occur. This open-label study involves only one treatment group receiving ivosidenib. Throughout the study, participants will be monitored regularly for disease progression, treatment response including complete remission, overall survival, and any adverse events. The main outcome measured is disease-free survival at 12 months. Participants will be followed for up to 36 months to assess overall survival and safety. The total duration of participation will depend on individual treatment response and tolerability.

Ivosidenib as Maintenance Therapy in Transplant-Ineligible IDH1-mutated AML and HR-MDS