A Phase I Study of CB-5339 for Recurrent or Persistent Acute Myeloid Leukemia or Myelodysplastic Syndromes


Full Title

A Phase I Study to Evaluate the Safety and Pharmacokinetic Profiles of CB-5339 in Participants with Relapsed/Refractory Acute Myeloid Leukemia, Intermediate or Higher-Risk Myelodysplastic Syndromes, bcr-abl negative Myeloproliferative, Myelodysplastic/Myeloproliferative Neoplasms and Multiple Myeloma


The purpose of this study is to find the highest dose of the investigational drug CB-5339 that can be given safely in patients with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) that came back or persisted despite prior treatment. CB-5339 blocks a protein called VCP/p97 that is believed to play an active role in the growth of cancer. Researchers believe that blocking VCP/p97 may stop or slow the growth of cancer cells. CB-5339 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 recurrent or persistent AML or high-risk MDS.
  • At least 2 weeks should pass since the completion of prior treatment and receipt of CB-5339.
  • 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 646-608-3749.





Disease Status

Relapsed or Refractory