An Innovative Therapies for Children with Cancer European consortium-led phase 1/2 dose escalation study aimed to establish the safety, tolerability, and efficacy of midostaurin in children and adolescents 3 months–18 years of age with either relapsed/refractory FLT3-mutated AML or KMT2A-rearranged ALL (which overexpresses wild-type FLT3). The gene discussed is FLT3; the disease is acute myeloid leukemia.