NCT06652438

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

Randomized Study to Assess Revumenib in Combination With Azacitidine + Venetoclax in Adult Patients With Newly Diagnosed NPM1-mutated or KMT2A-rearranged AML Ineligible for Intensive Chemotherapy

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 415 (estimated)
• Sponsor: Stichting Hemato-Oncologie voor Volwassenen Nederland · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Treatment of patients with newly diagnosed AML who are not eligible for intensive chemotherapy has remained an area of high unmet medical need. The combination therapy with two medicines, azacitidine and venetoclax, is the usual plan of action. This has brought significant progress in the treatment, but it nevertheless is not curative and the disease does relapse over time. Revumenib blocks a specific molecule called menin in the cell nucleus. Some types of AML are reliant on menin working properly. These are leukemia cells with a change in the DNA, i.e. a mutation in the NPM1 or KMT2A gene. Revumenib can prevent the production of these types of leukemia cells by disrupting the production of this menin. The current study investigates whether adding revumenib to the combination therapy improves the prognosis for AML patients with a mutation in the NPM1 or KMT2A gene. This is a randomized, double-blind, placebo-controlled clinical study where subjects will be treated until disease progression, or development of side effects or death. From the moment of inclusion of the last patient, there will be a 4-year observational follow-up study in order to register survival duration and follow-up visits. Approximately 415 previously untreated patients with a mutation in the NPM1 or KMT2A gene and with newly diagnosed AML, who are not eligible for intensive chemotherapy. Patients must be ≥18 years of age.

Conditions

• Acute Myeloid Leukemia, Adult

Interventions

Type	Name	Description
DRUG	Revumenib	day 1- 28 per cycle
DRUG	Placebo	day 1- 28 per cycle

Timeline

• Start date: 2025-03-31
• Primary completion: 2029-12-13
• Completion: 2031-07-10
• First posted: 2024-10-22
• Last updated: 2025-04-04

Locations

67 sites across 3 countries: Germany, Netherlands, United Kingdom