Study of Oral BTX-A51 Alone and With Azacitidine in Adults With Relapsed or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

What this Trial Is About

Researchers are studying BTX-A51, an oral capsule medicine, in people with relapsed or refractory acute myeloid leukemia AML or high-risk myelodysplastic syndrome MDS. This open-label, phase 1 study aims to evaluate the safety, side effects, how the drug moves through the body, and early signs of effectiveness. The study is sponsored by Edgewood Oncology Inc. and involves participants who have limited treatment options left. The study has three parts. Part 1a involves increasing doses of BTX-A51 alone to find the highest safe dose and dose-limiting toxicities during 28-day cycles of treatment, with 3 weeks of medicine followed by 1 week without. Part 1b expands this dose group to gather more safety and efficacy data. Part 1c explores combining BTX-A51 at the recommended dose with azacitidine, given by injection on days 1 to 7 of each 28-day cycle. Treatment can continue for up to eight 28-day cycles if benefits outweigh risks. Participants will have regular assessments including monitoring for side effects, lab tests, heart checks ECG and echocardiogram, and physical exams during treatment cycles. After completing treatment, they will be followed by phone every 3 months for up to 2 years to check survival and any further cancer therapies. The main outcomes include the highest tolerated dose, safety, drug levels in the blood, and response to treatment over time.

A Study of BTX-A51 in People With Relapsed or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome