Phase Ib Study of APG-115 Alone or With Azacitidine or Cytarabine in Adults With Relapsed or Refractory AML and High-Risk MDS

What this Trial Is About

Acute myeloid leukemia AML is a cancer characterized by the rapid and uncontrolled growth of immature blood cells in the bone marrow and circulation. This trial evaluates APG-115, an oral small-molecule drug that inhibits MDM2 protein and has shown strong anti-cancer activity in lab and animal models. The study aims to assess the safety, pharmacokinetics, and pharmacodynamics of APG-115 alone and in combination with azacitidine or cytarabine in patients with relapsed or refractory AML and high-risk myelodysplastic syndromes MDS. It is a phase 1b, open-label study sponsored by Ascentage Pharma Group Inc. The study is divided into three stages Stage 1 involves dose escalation of APG-115 alone given orally once daily from days 1 to 7 in a 28-day cycle to determine the maximum tolerated dose and dose-limiting toxicities. Stage 2 tests combinations of APG-115 with azacitidine or cytarabine in a similar dose escalation design. Stage 3 is a dose expansion phase for the combination treatments. Treatment continues for up to six cycles or until disease progression, unacceptable side effects, or other reasons for stopping. Patients benefiting after six cycles may continue treatment if it remains safe. Participants will undergo regular assessments including monitoring for toxicities during the first treatment cycle, evaluation of treatment response at the end of cycle 1 and 2 and every two months up to six cycles, and survival follow-up for six months after treatment ends. Safety and drug effects will be closely tracked throughout. The main measure is the occurrence of dose-limiting toxicities during cycle 1. Secondary measures include overall response rate and overall survival. The study follows patients until disease progression or withdrawal, with ongoing safety monitoring.

A Phase Ib Study of APG-115 Single Agent or in Combination With Azacitidine or Cytarabine in Patients With AML and MDS.