NCT07223814

Bleximenib in Combination With Standard Induction and Consolidation Therapy Followed by Maintenance for Treatment of Patients With Acute Myeloid Leukemia (AML)

Bleximenib or Placebo in Combination With Standard Induction and Consolidation Therapy Followed by Maintenance for the Treatment of Patients With Newly Diagnosed KMT2A-rearranged or NPM1-mutant Acute Myeloid Leukemia Eligible for Intensive Chemotherapy: a Double-blind Phase 3 Study

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 875 (estimated)
• Sponsor: Stichting Hemato-Oncologie voor Volwassenen Nederland · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The current standard of care treatment for adult patients with acute myeloid leukemia (AML) consists of chemotherapy and, if indicated, donor stem cell transplantation. Bleximenib blocks the interaction between a protein called menin and another protein called KMT2A in the leukemia cells. When this interaction is disrupted in AML with mutations in the NPM1 or KMT2A gene, bleximenib can cause leukemia cells to die. The main objective is to assess if treatment with bleximenib, when added to chemotherapy treatment will improve treatment outcome in adult participants with newly diagnosed AML who present with mutations in the NPM1 or KMT2A genes. This is a randomized, double-blind, placebo-controlled, phase 3 clinical trial. All of the participants will receive standard chemotherapy treatment, combined with either bleximenib or a placebo. A placebo is a substance that looks like the study medicine but has no active ingredients (e.g., a sugar pill). In a double blind trial neither the participant nor the doctor know if placebo or active study drug is given. After the end of the protocol treatment there will be an observational follow-up of 4 years from the time of inclusion of the last patient. The results of the different treatment groups will be compared. 875 previously untreated patients with AML with a specific change in the DNA of the leukemia cells (a KMT2A rearrangement or a NPM1 mutation) will be included. Participants must be 18 years or older and considered eligible for intensive chemotherapy.

Conditions

• Acute Myeloid Leukemia

Interventions

Type	Name	Description
DRUG	Bleximenib	Participants will receive bleximenib
DRUG	Cytarabine	Participants will receive Cytarabine
DRUG	Daunorubicin or Idarubicin	Participants will receive Daunorubicin or Idarubicin
DRUG	Placebo	Participants will receive Placebo

Timeline

• Start date: 2026-03-01
• Primary completion: 2030-06-01
• Completion: 2033-12-01
• First posted: 2025-11-03
• Last updated: 2026-04-06

Locations

8 sites across 3 countries: United States, Germany, Netherlands

Regulatory

• FDA-regulated drug study