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 448 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.