AZD3632 in Hematologic Malignancies is a modular, open-label, multicenter Phase I/II study investigating the HOX gene expression inhibitor AZD3632 as monotherapy or in combination. Primary endpoints for the dose-escalation (Phase I) module include the assessment of safety, tolerability, and determination of the Phase II dose. Eligible participants are adults with relapsed or refractory acute leukemia or MDS harboring KMT2Ar, NPM1m, or other genotypes associated with HOX overexpression. Participants receive oral AZD3632 as monotherapy (dose escalation) or in combination (Phase II). Secondary endpoints include pharmacokinetics, Objective Response Rate (ORR) to assess preliminary efficacy, and Overall Survival (OS).
This study is testing a new, orally administered drug called AZD3632 for patients with advanced blood cancers (such as acute leukemia or MDS). AZD3632 is designed to specifically target cancer cells that have certain genetic alterations (KMT2Ar or NPM1m). In this early Phase I/II study, doctors primarily evaluate how safe and well-tolerated the drug is, both alone and in combination with other therapies. Adults whose disease has progressed or relapsed despite standard treatments can participate. Another goal is to gather initial evidence of AZD3632's effectiveness, such as how long patients live with the treatment or whether they become independent of blood transfusions.
