Phase 1 Study of APL-4098 Alone and with Azacitidine and Venetoclax for Adults with Acute Myeloid Leukemia or Myelodysplastic Syndromes

What this Trial Is About

Researchers are evaluating the safety, tolerability, and effectiveness of a drug called APL-4098 alone and combined with azacitidine, and with azacitidine plus venetoclax. This study focuses on adults with acute myeloid leukemia AML, myelodysplastic syndrome MDSAML, or MDS with excess blasts. The trial is an open-label Phase 1 study designed to find appropriate dosing and assess treatment effects in these blood cancers. The study includes different treatment groups where participants receive escalating doses of APL-4098 alone, APL-4098 combined with azacitidine, or APL-4098 combined with azacitidine and venetoclax. Azacitidine is given at a standard dose during the first seven days of each 28-day treatment cycle, while APL-4098 and venetoclax are taken orally. Participants may receive these treatments in cycles lasting 28 days, with dose levels adjusted during the escalation phase. Participants will be closely monitored throughout the study, including for side effects and drug levels in the body on specific days of treatment cycles. Researchers will assess safety through adverse event tracking and dose-limiting toxicities during the first cycles. They will also evaluate how the disease responds to treatment at several points up to two years. The study is expected to last approximately one year for primary safety and dosing outcomes, with longer follow-up for treatment response.

A Study to Investigate APL-4098 Alone and in Combination in Adults With AML or MDS