Xitun District

Researchers are evaluating the safety, pharmacokinetics, pharmacodynamics, and preliminary effectiveness of an oral drug called ZE46-0134 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 includes patients who may be either outpatients or hospitalized due to AML. The trial focuses on those with FLT3-ITD, FLT3-TKD, or spliceosome mutations, conditions linked to aggressive blood cancers with generally poor prognosis. The study is conducted in two parts: Part 1 involves dose escalation and determination of the maximum tolerated dose (MTD), enrolling small groups of patients with either FLT3 mutations or spliceosome mutations to receive up to 24 cycles of treatment (each cycle lasting 28 days). Patients benefiting after 24 cycles may continue therapy. Part 2 is the dose expansion phase, enrolling up to 30 patients per mutation group across two dose cohorts to further evaluate treatment at selected doses from Part 1. ZE46-0134 is given as once-daily oral capsules throughout these phases. Participants will be monitored closely with clinical laboratory tests and safety assessments throughout the study. Researchers will measure outcomes including the occurrence of dose-limiting toxicities from the start of treatment up to a maximum of 24 cycles. The study also tracks pharmacokinetic and pharmacodynamic responses to understand how the drug behaves in the body. Participants may continue treatment if they benefit, and safety and efficacy data will guide future research directions.