Phase 3 Double-Blind Studies Testing Ziftomenib with Standard Nonintensive or Intensive Treatments in Adults with Untreated NPM1 Mutated or KMT2A Rearranged Acute Myeloid Leukemia

What this Trial Is About

Researchers are studying ziftomenib, an investigational drug targeting the menin pathway, in patients with acute myeloid leukemia AML who have specific genetic mutations NPM1-m or KMT2A-r and have not yet received treatment. The study includes two separate phase 3, randomized, double-blind, placebo-controlled trials assessing ziftomenib combined with standard therapies. The goal is to evaluate the benefits and risks of adding ziftomenib to current standard treatments for AML. One study, called the Nonintensive Therapy Study, enrolls older patients or those with serious medical conditions to receive venetoclax and azacitidine standard care plus either ziftomenib or placebo. The second study, the Intensive Therapy Study, involves medically fit patients treated with cytarabine and daunorubicin known as 73 plus ziftomenib or placebo during induction, followed by cytarabine plus ziftomenib or placebo in consolidation, and a maintenance phase where patients receive ziftomenib or placebo alone. Patients are randomly assigned to these groups without knowing which treatment they receive. Participants will undergo regular assessments to monitor overall survival, event-free survival, remission rates, and measurable residual disease in bone marrow up to 36 months after enrollment. Safety is evaluated through adverse event reporting and drug concentration measurements. Patient-reported outcomes related to health are also collected. The study involves oral and intravenous drug administration with visits scheduled according to treatment phases. Participation lasts up to 36 months, including treatment and follow-up periods.

Studies to Assess Ziftomenib in Combination With Ven+Aza or 7+3 in Patients With Untreated NPM1-m or KMT2A-r AML