TK216 in Patients With Relapsed or Refractory 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. This study is a first in human study of TK216 in subjects with Ewing sarcoma. 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.

Inclusion Criteria

• Histologically or cytologically confirmed diagnosis of Ewing sarcoma (including ESFT) in subjects with relapsed or refractory disease who have failed standard therapy

Exclusion Criteria

• Symptomatic brain metastases

TK216 is administered as a continuous intravenous infusion over 7-21 days depending on the dosing group. Administration is in cycles with a 14 day resting period between cycles. In the dose escalation segment of the study vincristine can be added after completion of 2 cycles of treatment with TK216 alone. After a "recommended phase 2 dose" has been determined in the dose escalation segment an expansion cohort will be initiated. In the expansion cohort combination treatment of TK216 and vincristine will start with cycle 1.

Lead Organization
Oncternal Therapeutics, Inc

Primary ID TK216-01
Secondary IDs NCI-2016-00918
Clinicaltrials.gov ID NCT02657005