Study of Revumenib With Azacitidine and Venetoclax to Treat Adults With Newly Diagnosed AML With NPM1 or KMT2A Gene Changes Ineligible for Intensive Chemotherapy

What this Trial Is About

Researchers are evaluating a new treatment approach for adults with newly diagnosed Acute Myeloid Leukemia AML who have mutations in the NPM1 or KMT2A genes and are not eligible for intensive chemotherapy. The study focuses on adding revumenib, a drug that blocks a molecule called menin important for leukemia cell survival, to standard treatment with azacitidine and venetoclax. This is a randomized, double-blind, placebo-controlled Phase 3 clinical trial aiming to improve outcomes for these patients. Participants will receive either revumenib or a placebo alongside azacitidine and venetoclax in continuous 28-day treatment cycles. Treatment continues until disease progression, unacceptable side effects, death, withdrawal, or other protocol-defined reasons for stopping. After the last patient is enrolled, there will be a 4-year follow-up period to observe survival and ongoing health. During the study, patients will be closely monitored with regular assessments to track overall survival, rates and duration of remission, event-free survival, and quality of life. Genetic and molecular tests will evaluate response at the bone marrow and blood levels. Safety and tolerability will also be monitored throughout treatment and the follow-up phase, which together may last several years.

Revumenib in Combination With Azacitidine + Venetoclax in Patients NPM1-mutated or KMT2A-rearranged AML