A Phase I Study of CPI-0610 in Patients with Acute Leukemia, Myelodysplastic Syndromes, or Myelodysplastic/Myeloproliferative Neoplasms

Full Title

A Phase 1 Study of CPI-0610, a Small Molecule Inhibitor of BET Proteins, in Patients with Acute Leukemia, Myelodysplastic Syndrome, Myelodysplastic/Myeloproliferative Neoplasms, or Myelofibrosis


CPI-0610 is an investigational drug designed to inhibit cancer growth by blocking a group of proteins called BET proteins. These proteins are known to regulate the cellular levels of different proteins that promote cancer cell growth and survival. CPI-0610 is taken orally (by mouth).

In this study, researchers want to find the highest dose of CPI-0610 that can be given safely in patients with hematologic cancers such as acute leukemia, myelodysplastic syndromes (MDS), or cancers with myeloproliferative features.


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

  • Patients must have one of the following cancers of the white blood cells: acute lymphocytic leukemia, acute myelogenous leukemia, mixed lineage leukemia, chronic myelogenous leukemia in blast crisis, MDS, chronic myelomonocytic leukemia, or a cancer with myeloproliferative and myelodysplastic features.
  • Patients must have recovered from the serious side effects of previous 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 and to inquire about eligibility for this study, please contact Dr. Eytan Stein at 212-639-3314.