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TK216 in Patients With Relapsed or Refractory Ewing Sarcoma

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT02657005
Recruitment Status : Recruiting
First Posted : January 15, 2016
Last Update Posted : May 26, 2021
Information provided by (Responsible Party):
Oncternal Therapeutics, Inc

Tracking Information
First Submitted Date  ICMJE January 12, 2016
First Posted Date  ICMJE January 15, 2016
Last Update Posted Date May 26, 2021
Actual Study Start Date  ICMJE August 2016
Estimated Primary Completion Date March 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 13, 2016)
  • 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 Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 13, 2016)
  • 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 Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
Descriptive Information
Brief Title  ICMJE TK216 in Patients With Relapsed or Refractory Ewing Sarcoma
Official Title  ICMJE 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 in monotherapy and in combination with vincristine to assess the potential of TK216 for further development.
Detailed Description The study is currently in the "expansion" phase after finding the recommended phase 2 dose in the dose/schedule escalation cohorts. Eighteen patients will be enrolled in the expansion phase. Vincristine is administered starting cycle 1, along with a 14 day continuous infusion of TK216.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Sarcoma, Ewing
Intervention  ICMJE Drug: TK216
Inhibitor of protein-protein interactions of EWS-FLI1 fusion protein
Study Arms  ICMJE Experimental: TK216 treatment
Dose escalation and expansion cohorts to determine dose-limiting toxicities, maximally tolerated dose, preliminary efficacy, and recommended phase 2 dose.
Intervention: Drug: TK216
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: January 13, 2016)
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE June 2022
Estimated Primary Completion Date March 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 10 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Oncternal Information 858-434-1113
Listed Location Countries  ICMJE United States
Removed Location Countries  
Administrative Information
NCT Number  ICMJE NCT02657005
Other Study ID Numbers  ICMJE TK216-01
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Oncternal Therapeutics, Inc
Study Sponsor  ICMJE Oncternal Therapeutics, Inc
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: James Breitmeyer, MD Oncternal Therapeutics
PRS Account Oncternal Therapeutics, Inc
Verification Date November 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP