Phase 1 Study of Oral ZE46-0134 to Assess Safety and Effectiveness in Adults with FLT3 or Spliceosome Mutated Relapsed or Refractory Acute Myeloid Leukemia

What this Trial Is About

Researchers are studying ZE46-0134, an investigational oral drug, in adults with relapsed or refractory acute myeloid leukemia AML who have specific gene mutations in FLT3 or spliceosome genes. This Phase 1, open-label study aims to assess the drugs safety, how it moves and works in the body pharmacokinetics and pharmacodynamics, and its preliminary effectiveness. AML is a serious blood cancer with poor survival rates, and mutations in FLT3 are common and contribute to disease progression. The study is divided into two parts Part 1 focuses on dose escalation to find the maximum tolerated dose, enrolling small groups of patients with either FLT3 or spliceosome mutations to receive up to 24 cycles of daily oral ZE46-0134 capsules. Part 2 involves dose expansion, with up to 30 patients in each mutation group receiving doses determined from Part 1. If patients benefit after 24 cycles, continuing treatment may be considered. Participants will undergo regular assessments including laboratory tests, safety monitoring, and pharmacokinetic sampling during the treatment cycles. Researchers will track adverse events, blood test results, and leukemia response up to six treatment cycles and beyond. The main outcome measured is the rate of dose-limiting toxicities during treatment, helping to evaluate the drugs safety profile. The study is expected to continue until December 2027.

Dose Escalation and Expansion Study to Evaluate the Safety, PK, PD and Efficacy of ZE46-0134 in Adults With FLT3 Mutated or Spliceosome Mutated Relapsed or Refractory Acute Myeloid Leukemia