Clinical Trial: TK216 in Patients With Relapsed or Refractory Ewing Sarcoma

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Phase 1, Dose Escalation Study of Intravenous TK216 in Patients With Relapsed or Refractory Ewing Sarcoma

Brief Summary: 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 to assess the potential of TK216 for further development.

Detailed Summary:
Sponsor: Oncternal Therapeutics, Inc

Current Primary Outcome:

  • Dose-limiting toxicities (DLTs) [ Time Frame: 18 months ]
    Listing of dose-limiting toxicities by daily dose in mg/m^2
  • Maximum tolerated dose (MTD) [ Time Frame: 18 months ]
    Maximum daily dose in mg/m^2
  • Biologically effective and recommended Phase 2 dose (RP2D) [ Time Frame: 18 months ]
    Daily dose in mg/m^2


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Adverse Events [ Time Frame: 18 months ]
  • Antitumor activity as measured by Overall Response Rate (ORR) [ Time Frame: 18 months ]
  • Antitumor activity as measured by Duration of Response (DOR) [ Time Frame: 18 months ]
  • Duration of Disease Control [ Time Frame: 18 months ]
  • Assay methods to detect EWS-FLI1 (or EWS-ERG and EWS-ets) [ Time Frame: 18 months ]
  • Pharmacokinetics: Maximum Plasma Concentration [Cmax] [ Time Frame: 18 months ]
  • Pharmacokinetics: Area Under the Curve [AUC] [ Time Frame: 18 months ]
  • Pharmacokinetics: Halflife [T1/2] [ Time Frame: 18 months ]
  • Pharmacodynamics: serum miRNA profile [ Time Frame: 18 months ]
  • Pharmacodynamics: tumor tissue RNA assays [ Time Frame: 18 months ]
  • Pharmacodynamics: tumor tissue protein assays [ Time Frame: 18 months ]


Original Secondary Outcome: Same as current

Information By: Oncternal Therapeutics, Inc

Dates:
Date Received: January 12, 2016
Date Started: May 2016
Date Completion: June 2018
Last Updated: October 13, 2016
Last Verified: October 2016