Study of Ivosidenib and Azacitidine with or without Venetoclax in Adults 18 and Older with Newly Diagnosed IDH1-Mutated Acute Myeloid Leukemia or MDSAML Who Cannot Have Intensive Chemotherapy

What this Trial Is About

Researchers are investigating treatments for adults diagnosed with acute myeloid leukemia AML or MDSAML that have a mutation in the IDH1 gene and who cannot undergo intensive chemotherapy. The study evaluates whether adding the drug venetoclax to the existing standard treatment of ivosidenib and azacitidine improves patient outcomes compared to a placebo. The safety and effectiveness of venetoclax are key focuses of this phase 3 trial. Participants receive either venetoclax or a placebo alongside ivosidenib and azacitidine. Treatments are given in continuous 28-day cycles and continue until disease relapse, progression, unacceptable side effects, death, withdrawal, or other protocol-defined reasons for stopping. The trial uses a triple-blind, randomized design to fairly compare the two treatment groups. During the study, researchers will monitor event-free survival as the primary outcome, assessing patients for signs of disease control or progression up to 12 months after the last patient is included. Secondary measures include overall survival, response rates, transfusion independence, and the duration and timing of responses. Participant involvement includes regular assessments, blood tests, and monitoring for side effects throughout the treatment period and follow-up.

Ivosidenib and Azacitidine With or Without Venetoclax in Adult Patients With Newly Diagnosed IDH1-Mutated AML or MDS/AML Considered Ineligible for Intensive Chemotherapy