A Study of BTX-A51 in People With Relapsed or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome
Status:
Recruiting
Trial end date:
2025-03-01
Target enrollment:
Participant gender:
Summary
This is an open-label, dose escalation study to evaluate the safety, toxicity, and
pharmacokinetics (PK) as well as preliminary efficacy of BTX-A51 capsules in participants
with relapsed or refractory acute myeloid leukemia (AML) or high-risk myelodysplastic
syndrome (MDS).
The study will be done in two phases. Phase 1a of this study is designed to determine the
dose-limiting toxicities (DLTs) and maximum tolerated dose (MTD) of orally administered
BTX-A51 in up to 35 participants who are evaluable for toxicity. Once the MTD is determined,
it is planned that an additional 15 participants will be enrolled in Phase 1b of this study
for additional experience with safety and efficacy, and to determine the recommended Phase 2
dose (RP2D) which may or may not be different from the MTD.
Continued treatment will be available under this study protocol for up to eight 28-day cycles
(Continued Treatment Phase) if the Investigator judges the benefit outweighs the risk.
Once BTX-A51 treatment has completed, participants will be contacted by telephone every 3
months for up to 2 years after their last treatment for survival status and anticancer
therapy (Overall Survival Follow-up).