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TK216 in Patients With Relapsed or Refractory Ewing Sarcoma


Ewing sarcoma, including ESFT (Ewing sarcoma family of tumors)


This study is a first in human study of TK216, an inhibitor of protein-protein interactions of EWS-FLI1 fusion protein, in subjects with Ewing sarcoma. Ewing sarcoma is characterized by genomic rearrangements resulting in over-expression of ets family transcription factors driving tumor progression. TK216 is designed to inhibit this effect by inhibiting downstream effects of the EWS-FLI1 transcription factor. The study is designed to establish initial safety and efficacy data in monotherapy and in combination with vincristine to assess the potential of TK216 for further development.

Eligibility Criteria

Ages Eligible for Study: Children 10 years and older, Adult
Sexes Eligible for Study: All
Other inclusion and exclusion criteria as listed on


Jodi Muscal, MD   
Principal Investigator

Kathy McCarthy, RN