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

Sponsor
Stichting Hemato-Oncologie voor Volwassenen Nederland
Study ID
NCT06652438
Phase
PHASE3
Status
Recruiting
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Conditions

  • Acute Myeloid Leukemia, Adult

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Revumenib — DRUG
    day 1- 28 per cycle
  • Placebo — DRUG
    day 1- 28 per cycle

Study Details

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.

Key Dates

Start date
Mar 31, 2025
Status verified
Mar 2025
Primary completion
Dec 13, 2029
Completion
Jul 10, 2031

Study Design

Enrollment
415 participants (estimated)
Allocation
RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Placebo Comparator: Revumenib-placebo
    day 1-28 Placebo Treatment will be on a continuous 28-day cycle schedule and continued until disease progression, development of unacceptable toxicity, death, withdrawal by subject or other protocol defined criteria for discontinuation (whichever comes first).
  • Experimental: Revumenib
    day 1-28 Revumenib Treatment will be on a continuous 28-day cycle schedule and continued until disease progression, development of unacceptable toxicity, death, withdrawal by subject or other protocol defined criteria for discontinuation (whichever comes first).

Primary Outcome Measure

Overall survival (OS) in adult patients with newly diagnosed NPM1-mutated AML ineligible for intensive chemotherapy. [ Time Frame: 58 months after last patient inclusion ]

Central Contacts

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