ALIDHE phase 3b study design: ivosidenib + azacitidine in adults with newly diagnosed IDH1 mutant acute myeloid leukemia.
Vyas P., Salek S., Vives S., Recher C., Döhner H., Venditti A., Derrien H., Chatin S., De La Bigne A-M., Pelouchova J., Hills R., Nier S.
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).