A Phase I Study of SP-2577 in Patients with Recurrent or Persistent Ewing Sarcoma


Full Title

Phase 1 Trial of the LSD1 Inhibitor Seclidemstat (SP-2577) with and without Topotecan and Cyclophosphamide in Patients with Relapsed or Refractory Ewing Sarcoma and Select Sarcomas


The purpose of this study is to find the highest dose of the investigational drug SP-2577 that can be given safely in patients with Ewing sarcoma that came back or continues to grow despite treatment. SP-2577 is designed to block the activity of a protein called LSD1. This protein plays a role in selecting which genes are turned on to make other proteins.

Blocking LSD1 may prevent the protein produced by the fusion gene responsible for Ewing sarcoma from binding to DNA and changing the function of other genes in the cell. This could allow the genes to make cells function like normal cells rather than cancer cells. SP-2577 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 Ewing sarcoma that came back or continues to grow despite prior therapy.
  • Patients must recover from the serious side effects of prior therapies before entering the study.
  • This study is for patients age 12 and older.

For more information about this study and to inquire about eligibility, please contact 1-833-MSK-KIDS.





Disease Status

Relapsed or Refractory