Phase 3, Open-Label, Randomized Study of Gilteritinib and Azacitidine Vs Azacitidine for Newly Diagnosed FLT3-Mutated Acute Myeloid Leukemia in Patients Ineligible for Intensive Induction Chemotherapy

Background: Gilteritinib (GIL), a FLT3 inhibitor, shows efficacy/safety in patients (pts) with FLT3mut+ relapsed/refractory (R/R) AML. For pts with newly diagnosed (ND) AML with FLT3 mutations unable to receive intensive induction chemotherapy (IIC), survival is limited, and therapy options are few. In preclinical studies, GIL plus azacitidine (AZA) impeded tumor growth and induced apoptosis/differentiation of FLT3-ITD AML cell lines. Antitumor effects appeared synergistic in xenografted mouse models (data on file). We investigated GIL+AZA vs AZA in adults with ND FLT3mut+ AML ineligible for IIC (NCT02752035).