Study of Avapritinib Treatment for Children Under 18 With Relapsed or Refractory Core Binding Factor Acute Myeloid Leukemia and KIT Mutation

What this Trial Is About

Researchers are evaluating the safety and effectiveness of avapritinib in children with relapsed or refractory core binding factor acute myeloid leukemia CBF-AML that has a KIT mutation. This phase 2, multicenter, single-arm clinical trial aims to address the challenge that about 30% of CBF-AML patients relapse despite current treatments. KIT mutations, especially the D816V variant, are linked to relapse and poorer survival, and avapritinib has shown promise in related conditions and preliminary reports. Participants will receive a combination treatment including avapritinib alongside hypomethylating agents and low-dose chemotherapy drugs such as decitabine or azacitidine, idarubicin, cytarabine, and granulocyte colony-stimulating factor. The avapritinib dose starts at 50mgm2 daily and may be increased to 100mgm2 based on blood counts, with treatment cycles lasting up to 28 days. For patients with molecular relapse after stem cell transplantation, avapritinib may be combined with demethylating agents, interferon, or donor lymphocyte infusion without chemotherapy. During the study, participants will be closely monitored for response to treatment, with the main outcome measured between days 28 and 35 after starting therapy. Researchers will also assess overall survival and progression-free survival for up to five years. Safety will be tracked through clinical evaluations and laboratory tests. The total study duration extends until March 2027, providing long-term follow-up to better understand the treatments impact.

Safety and Efficacy of Avapritinib in Relapsed or Refractory Pediatric CBF-AML With KIT Mutation