A Phase I Study of H3B-8800 in Patients with Myelodysplastic Syndromes or Leukemia


Full Title

H3B-8800-G000-101: An Open-label, Multicenter Phase 1 Trial to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of Splicing Modulator H3B-8800 for Subjects With Myelodysplastic Syndromes, Acute Myeloid Leukemia, and Chronic Myelomonocytic Leukemia


Some cancers grow because of a type of genetic mutation called a “splicing” mutation, resulting in the production of abnormal proteins that promote cancer growth. H3B-8800 is an investigational drug designed to inhibit the splicing that leads to the production of these dysfunctional proteins.

In this study, researchers are finding the highest dose of H3B-8800 that can be given safely in patients with myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), or chronic myelomonocytic leukemia (CMML) that continues to grow despite standard therapies. H3B-8800 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 MDS, AML, or CMML that has continued to grow despite standard therapies.
  • At least 2 weeks must pass between the completion of prior treatment and entry into 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. Ellin Berman at 212-639-7762.






Klimek, Virginia, MD