Phase 12 Study of Oral Enzomenib DSP-5336 for Treating Acute Leukemia and Selected Blood Cancers With or Without MLL Rearrangement or NPM1 Mutation

What this Trial Is About

Researchers are evaluating Enzomenib DSP-5336, an oral drug, in patients with various types of acute leukemia, including relapsed or refractory acute myeloid leukemia AML, acute lymphocytic leukemia ALL, and acute leukemia of ambiguous lineage. The study also includes patients with high-risk myelodysplastic syndromes MDS and relapsed multiple myeloma MM in selected sites. This phase 12 trial aims to assess the safety, pharmacokinetics, pharmacodynamics, and clinical activity of DSP-5336 alone or combined with standard AML treatments, particularly in patients with specific genetic mutations like MLL rearrangement or NPM1 mutation. The study involves dose escalation and dose expansion of DSP-5336 administered orally. Participants may receive DSP-5336 alone or combined with standard AML regimens such as venetoclax plus azacitidine or intensive chemotherapy with cytarabine and daunorubicin 73. Different study arms include patients with or without certain medications like CYP3A4 inhibitor azoles, and those with specific genetic profiles. The trial evaluates recommended phase 2 doses for various patient groups and combination treatments. Participants will undergo assessments including monitoring for adverse events within 30 days after the last dose and evaluation of clinical responses approximately six months after treatment begins. Researchers will collect blood and bone marrow samples for genomic analysis and track drug levels in the body. Safety labs, ECGs, physical exams, and patient questionnaires will be performed throughout the study. The trial includes long-term follow-up of overall survival up to two years after treatment ends, with visits and tests scheduled to monitor health and treatment effects.

A Phase 1/2 Study of Enzomenib (DSP-5336) in Patients With Acute Leukemia (Horizen-1)