A Phase I Study of BTX-A51 in People with Recurrent or Persistent Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndromes


Full Title

A First-In-Human, Open-Label, Escalating Multiple-Dose Study to Evaluate the Safety, Toxicity, and Pharmacokinetics of BTX-A51 Capsules in Patients with Relapsed or Refractory Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome


The purpose of this study is to find the highest dose of the investigational drug BTX-A51 that can be given safely in patients with acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (MDS, which can progress to leukemia) that have come back or continued to grow despite treatment. BTX-A51 works by increasing the levels of p53, a protein that normally causes cell death in response to stress. Some cancer cells survive because p53 is lost.

BTX-A51 also blocks two enzymes (called CDK7 and CDK9) that AML and high-risk MDS cells need to survive. Laboratory studies have shown that BTX-A51 can kill leukemia cells while sparing normal cells and was more effective than chemotherapy or targeted therapy. BTX-A51 is taken orally (by mouth).


To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients must have AML or high-risk MDS that has come back or continued to grow despite prior treatment.
  • Patients should recover from the serious side effects of prior treatments before entering the study.
  • Patients must be able to walk and do routine activities for more than half of their normal waking hours.
  • This study is for patients age 18 and older.

For more information about this study and to inquire about eligibility, please contact Dr. Eytan Stein at 212-639-3314.





Disease Status

Relapsed or Refractory