Phase 3 Study of Ivosidenib or Azacitidine Alone in Adults With Untreated IDH1-Mutated Myelodysplastic Syndromes

What this Trial Is About

Researchers are evaluating treatments for adult patients with myelodysplastic syndromes MDS who have a specific IDH1 mutation and have not previously received treatment with hypomethylating agents. The study focuses on comparing two treatment options to understand their effects on this patient group. This phase 3, randomized, open-label trial aims to assess the responses and overall survival of participants receiving these therapies. Participants are randomized to receive either ivosidenib IVO alone or azacitidine AZA alone. Ivosidenib is taken orally once daily throughout each 28-day treatment cycle, while azacitidine is given by injection for the first 7 days of each 28-day cycle. Treatment continues until disease progression, unacceptable side effects, or other specified conditions. Study visits occur weekly during the first cycle and on the first day of each subsequent cycle. After treatment ends, participants attend a safety follow-up visit and are monitored for overall survival. During the study, participants will have regular appointments that may include physical exams, blood and urine tests, heart function monitoring with echocardiograms and electrocardiograms, bone marrow aspirates, and questionnaires about quality of life. Researchers will measure response rates at 4 months and other outcomes such as overall response, survival, disease progression, and quality of life over approximately four years. Safety will be monitored through adverse event reporting and follow-up visits.

Ivosidenib (IVO) Monotherapy and Azacitidine (AZA) Monotherapy in Patients With Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS) With an IDH1 Mutation