Phase 1b Study of Menin Inhibitor SNDX-5613 Combined With Daunorubicin and Cytarabine for Newly Diagnosed Acute Myeloid Leukemia with NPM1 Mutation, MLLKMT2A Rearrangement, or NUP98 Alterations

What this Trial Is About

Researchers are evaluating the safety, side effects, and best dose of the drug SNDX-5613 combined with standard chemotherapy in patients newly diagnosed with acute myeloid leukemia AML that has specific genetic changes in the NPM1, MLLKMT2A, or NUP98 genes. This phase Ib trial focuses on patients aged 18 to 75 who are eligible for intensive induction therapy and aims to find the recommended dose for further studies while assessing the treatments effects on cancer cell behavior and response. Participants receive a combination treatment starting with induction therapy where SNDX-5613 is given orally twice daily from days 2 to 28, along with daunorubicin intravenously on days 1 to 3, and cytarabine by continuous infusion on days 1 to 7. Those who respond move on to consolidation treatment with cycles of SNDX-5613 and cytarabine every 42 days for up to three cycles. Patients with persistent disease may receive re-induction therapy. Various scans, blood tests, and bone marrow biopsies are done throughout these treatment phases to monitor progress and safety. During the study, participants undergo regular evaluations including heart scans echocardiogram or MUGA, bone marrow aspiration and biopsy before, during, and after treatment phases, and blood collections at specific times. Researchers measure recommended doses, treatment responses, drug levels in the body, and explore biomarkers related to treatment effectiveness and resistance. Follow-up continues for up to two years after treatment to observe long-term outcomes such as disease relapse or survival.

A Phase 1b Study of Menin Inhibitor SNDX- 5613 in Combination With Daunorubicin and Cytarabine in Newly Diagnosed Patients With Acute Myeloid Leukemia and NPM1 Mutated/FLT3 Wildtype or MLL/KMT2A Rearranged or NUP98 Alterations Disease