This is a multi-center, single-arm, Phase I research study for the study drug, BXCL701, for participants with refractory or relapsed Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome with Excess Blasts-2 (MDS-EB-2). Phase I clinical trials test the safety and effectiveness of an investigational drug to learn whether the drug works in treating a specific disease. Investigational means the drug is being studied. BXCL701 is a synthetic dipeptide that is in tablet form and is taken by mouth.~The U.S. Food and Drug Administration (FDA) has not approved BXCL701 as a treatment for any disease.~Research study procedures include screening for eligibility and study treatment including evaluations, blood collections, bone marrow biopsies, and follow up visits.~Participation in this study will last approximately 3 years.~It is expected that about 24 people will participate in this study.~BioXcel is supporting this research study by providing the study drug and funding research tests and procedures.
The goal of this research study is to find the safest and most effective dose of the study drug, BXCL701, for the treatment of Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS).~The names of the study drugs involved in this study are/is:~BXCL701