Summary
The current standard of care treatment for adults with acute myeloid leukaemia (AML) consists of chemotherapy and, if indicated, donor stem cell transplantation.
Bleximenib is a drug that blocks the interaction between a protein called menin and another protein called KMT2A in the leukaemia cells. When this interaction is disrupted in AML with mutations in the NPM1 or KMT2A gene, bleximenib can cause leukaemia cells to die.
The main objective is to assess if treatment with bleximenib, when added to chemotherapy treatment, will improve treatment outcomes in people with newly diagnosed AML who present with mutations in the NPM1 or KMT2A genes.
How the study works
Eligible participants will receive standard chemotherapy treatment, which will be combined with either bleximenib or a placebo. People will be randomly assigned (by chance) to receive either bleximenib or the placebo.
A placebo is a substance that looks like the study medicine but has no active ingredients (e.g. a sugar pill). This is a double blind trial, which means neither the participants 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.