INTRODUCTION: Ivosidenib is an inhibitor of mutant isocitrate dehydrogenase 1 (mIDH1) that is approved alone or in combination with azacitidine in patients with mIDH1 acute myeloid leukemia (AML) that are ineligible to receive intensive chemotherapy. AIMS: Here we describe the design of ALIDHE, an international, multicenter, single-arm, open-label Phase 3b study of ivosidenib + azacitidine for treatment of newly diagnosed mIDH1 AML in usual clinical practice. METHODS: The primary endpoints are adverse events (AEs), clinical laboratory anomalies assessed as AEs, patients requiring transfusion and number of units transfused, and infection rate. The impact of ivosidenib + azacitidine treatment on health-related quality of life, healthcare resource utilization and measurable residual disease will also be assessed. CLINICAL TRIAL REGISTRATION NUMBER: NCT05907057 (ClinicalTrials.gov).
Journal article
2025-12-01T00:00:00+00:00
21
3721 - 3729
8
Acute myeloid leukemia, azacitidine, healthcare utilization, ivosidenib, mutant isocitrate dehydrogenase 1, quality of life, Humans, Isocitrate Dehydrogenase, Leukemia, Myeloid, Acute, Azacitidine, Mutation, Glycine, Pyridines, Antineoplastic Combined Chemotherapy Protocols, Adult, Clinical Trials, Phase III as Topic, Multicenter Studies as Topic, Quality of Life, Female, Male, Treatment Outcome, Middle Aged, Aged