Study of Avapritinib Combined With Standard Chemotherapy or VenetoclaxAzacitidine for Adults With Newly Diagnosed KIT Mutation Acute Myeloid Leukemia

What this Trial Is About

Researchers are investigating whether combining avapritinib with standard induction therapy can improve treatment outcomes for adults newly diagnosed with acute myeloid leukemia AML who have specific KIT mutations and chromosomal abnormalities such as t821, inv16, or t1616. The study aims to find the safest and most effective dose of avapritinib with chemotherapy and to assess if this combination increases the rate of minimal residual disease MRD negativity and long-term survival. This is a phase I-II trial conducted by the First Affiliated Hospital of Soochow University focusing on this patient population with historically poor prognosis. The trial includes two treatment groups Group A FIT receives avapritinib combined with an intensive chemotherapy regimen of cytarabine and idarubicin, while Group B UNFIT receives avapritinib combined with venetoclax and azacitidine. Avapritinib is administered orally on days 8 to 21 of a 28-day cycle in both groups, with chemotherapy or targeted agents given earlier in the cycle. Phase I focuses on dose escalation to determine the recommended dose for Phase II, which will then evaluate the efficacy and safety of this combination therapy including consolidation and maintenance schedules to prevent relapse. Participants will be closely monitored with regular assessments for disease response, side effects, and overall survival up to approximately four years from enrollment. Primary outcomes include determining the recommended phase II dose and measuring MRD negativity rates about two years after the first patient visit. Secondary outcomes include overall survival, remission rates, and adverse events. The study involves detailed evaluations of blood and bone marrow samples, and continuous safety monitoring to better understand how the combination therapy affects patients over time.

A New Treatment of Newly Diagnosed KIT Mutation CBF-Acute Myeloid Leukemia