A Phase II Study of E7820 in People with Bone Marrow (Myeloid) Cancers


Full Title

A Phase II Clinical Trial of E7820 for Patients with Relapsed/Refractory Myeloid Malignancies with Mutations in Splicing Factor Genes.


The purpose of this study is to assess the safety and effectiveness of the investigational drug E7820 in people with acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), or chronic myelomonocytic leukemia (CMML) that has come back or continued to grow despite treatment and has a change (mutation) in a “splicing factor” gene. Splicing factor genes send signals to cells about how to grow and divide. When there is a mutation in a splicing factor gene, cells can grow out of control, triggering the growth of cancer cells.

E7820 is designed to block a protein that is involved in splicing factor signaling and cancer cell growth. It 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, MDS, or CMML that has come back or continued to grow despite treatment and has a mutation in a splicing factor gene.
  • Participants should recover from the serious side effects of previous treatments before receiving E7820.
  • In addition to patients with good physical function, this study is also open to those who are capable of only limited self-care and are confined to bed or chair for more than half of their normal waking hours.
  • This study is for people 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