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TK216 in Patients With Relapsed or Refractory Ewing Sarcoma

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ClinicalTrials.gov Identifier: NCT02657005
Recruitment Status : Recruiting
First Posted : January 15, 2016
Last Update Posted : August 12, 2019
Sponsor:
Information provided by (Responsible Party):
Oncternal Therapeutics, Inc

Study Description
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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.

Condition or disease Intervention/treatment Phase
Sarcoma, Ewing Drug: TK216 Phase 1

Detailed Description:
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.

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 45 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1, Dose Escalation Study of Intravenous TK216 in Patients With Relapsed or Refractory Ewing Sarcoma
Actual Study Start Date : August 2016
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : February 2020

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Arms and Interventions
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Arm Intervention/treatment
Experimental: TK216 treatment
Dose escalation and expansion cohorts to determine dose-limiting toxicities, maximally tolerated dose, preliminary efficacy, and recommended phase 2 dose.
 Drug: TK216
Inhibitor of protein-protein interactions of EWS-FLI1 fusion protein


Outcome Measures
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Primary Outcome Measures :
  1. Dose-limiting toxicities (DLTs) [ Time Frame: 18 months ]
    Listing of dose-limiting toxicities by daily dose in mg/m^2

  2. Maximum tolerated dose (MTD) [ Time Frame: 18 months ]
    Maximum daily dose in mg/m^2

  3. Biologically effective and recommended Phase 2 dose (RP2D) [ Time Frame: 18 months ]
    Daily dose in mg/m^2


Secondary Outcome Measures :
  1. Adverse Events [ Time Frame: 18 months ]
  2. Antitumor activity as measured by Overall Response Rate (ORR) [ Time Frame: 18 months ]
  3. Antitumor activity as measured by Duration of Response (DOR) [ Time Frame: 18 months ]
  4. Duration of Disease Control [ Time Frame: 18 months ]
  5. Assay methods to detect EWS-FLI1 (or EWS-ERG and EWS-ets) [ Time Frame: 18 months ]
  6. Pharmacokinetics: Maximum Plasma Concentration [Cmax] [ Time Frame: 18 months ]
  7. Pharmacokinetics: Area Under the Curve [AUC] [ Time Frame: 18 months ]
  8. Pharmacokinetics: Halflife [T1/2] [ Time Frame: 18 months ]
  9. Pharmacodynamics: serum miRNA profile [ Time Frame: 18 months ]
  10. Pharmacodynamics: tumor tissue RNA assays [ Time Frame: 18 months ]
  11. Pharmacodynamics: tumor tissue protein assays [ Time Frame: 18 months ]

Eligibility Criteria
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Ages Eligible for Study:   10 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02657005


Contacts
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Contact: Oncternal Information 858-434-1113 clinical@oncternal.com

Locations
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United States, California
UCLA Jonsson Comprehensive Cancer Center Recruiting
Los Angeles, California, United States, 90095
Contact: Marianne Bernardo    310-794-8086    mjbernardo@mednet.ucla.edu   
Principal Investigator: Noah Federman, MD         
United States, Colorado
Children's Hospital of Colorado Recruiting
Aurora, Colorado, United States, 80045
Contact: Melissa Widener       etp@childrenscolorado.org   
United States, New York
Memorial Sloan Kettering Cancer Center Recruiting
New York, New York, United States, 10174
Contact: Marilyn Winchester    646-888-5711    winchesm@mskcc.org   
Contact: Jennifer Bonura       DirenzoJ@mskcc.org   
Principal Investigator: Paul A Meyers, MD         
United States, North Carolina
Duke Cancer Institute Recruiting
Durham, North Carolina, United States, 27710
Contact: Colleen Piechocki    919-681-1883      
Principal Investigator: Richard Riedel, MD         
United States, Texas
Texas Children's Cancer & Hematology Centers, Baylor College Recruiting
Houston, Texas, United States, 77030
Contact: Kathy McCarthy, RN    832-824-4804    ksmccart@txch.org   
Principal Investigator: Jodi A Muscal, MD         
UT MD Anderson Cancer Center Recruiting
Houston, Texas, United States, 77030
Contact: Joseph A Ludwig, MD    713-792-3626      
Contact: Viju Bhadkamkar, MS       viju@mdanderson.org   
Principal Investigator: Joseph A Ludwig, MD         
Sponsors and Collaborators
Oncternal Therapeutics, Inc
Investigators
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Study Director: James Breitmeyer, MD Oncternal Therapeutics
More Information
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Responsible Party: Oncternal Therapeutics, Inc
ClinicalTrials.gov Identifier: NCT02657005     History of Changes
Other Study ID Numbers: TK216-01
First Posted: January 15, 2016    Key Record Dates
Last Update Posted: August 12, 2019
Last Verified: July 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Additional relevant MeSH terms:
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Sarcoma
Sarcoma, Ewing
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
 Neoplasms
Osteosarcoma
Neoplasms, Bone Tissue
Neoplasms, Connective Tissue