Clinical Trials /

TK216 in Patients With Relapsed or Refractory Ewing Sarcoma

NCT02657005

Description:

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.

Related Conditions:
  • Ewing Sarcoma
Recruiting Status:

Recruiting

Phase:

Phase 1

Trial Eligibility

Document

Title

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

Clinical Trial IDs

  • ORG STUDY ID: TK216-01
  • NCT ID: NCT02657005

Conditions

  • Sarcoma, Ewing

Interventions

DrugSynonymsArms
TK216TK216 treatment

Purpose

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.

Trial Arms

NameTypeDescriptionInterventions
TK216 treatmentExperimentalDose escalation and expansion cohorts to determine dose-limiting toxicities, maximally tolerated dose, preliminary efficacy, and recommended phase 2 dose.
  • TK216

Eligibility Criteria

        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
      
Maximum Eligible Age:N/A
Minimum Eligible Age:12 Years
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:Dose-limiting toxicities (DLTs)
Time Frame:18 months
Safety Issue:
Description:Listing of dose-limiting toxicities by daily dose in mg/m^2

Secondary Outcome Measures

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

Details

Phase:Phase 1
Primary Purpose:Interventional
Overall Status:Recruiting
Lead Sponsor:Oncternal Therapeutics, Inc

Last Updated

October 13, 2016