TK216 in Patients With Relapsed or Refractory Ewing Sarcoma

Study 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 in monotherapy and in combination with vincristine to assess the potential of TK216 for further development.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Interventional
Eligible Ages 8 Years and Over
Gender All
More Inclusion & Exclusion Criteria

1. Willing and able to provide written IRB/IEC-approved Informed Consent. For patients < 18 years of age, their parents or legal guardians must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines. 2. Have histologically or cytologically confirmed diagnosis of Ewing sarcoma (including ESFT) with relapsed or refractory disease. Patients with metastatic disease who had standard chemotherapy at the time of diagnosis Pathology reports and slides or blocks should be available for review or additional testing. If not available, site must discuss with Sponsor. 3. Measurable disease according to RECIST version 1.1. Measurable disease can be verified from a previously documented computed tomography (CT) scan or MRI as long as no anti-cancer treatments have been administered in the interim. 4. Must have a central venous catheter in place prior to initiating infusion of study drug. 5. Prior cancer therapy: Patients may have received no more than 5 prior systemic regimens. At the time of treatment initiation, at least 2 weeks or 5 half-lives, whichever is longer, must have elapsed since prior cytotoxic chemotherapy. At least 7 days must have elapsed since completion of any prior non-cytotoxic cancer therapy. 6. Prior radiotherapy is allowed If ≥ 4 weeks has elapsed for radiation therapy (RT); ≥ 6 months must have elapsed if prior total body irradiation, craniospinal RT or if > 50% radiation of the pelvis; > 6 weeks must have elapsed if other substantial bone marrow radiation. Patients who have received brain irradiation must have completed whole brain radiotherapy and/or gamma knife at least 4 weeks prior to enrollment. 7. Stem Cell Transplant or Rescue without TBI: No evidence of active graft vs. host disease and ≥ 3 months must have elapsed since transplant. 8. Patients with controlled asymptomatic CNS involvement are allowed (see Concomitant Medications). Patients not requiring steroids or requiring steroids at stable dose (≤ 4 mg/day dexamethasone or equivalent) for at least 2 weeks are eligible. 9. Resolution of all acute toxic effects (excluding alopecia) of any prior anti-cancer therapy to NCI CTCAE (Version 4.03) Grade < 1. Details can be provided by Sponsor. 10. Eastern Cooperative Oncology Group (ECOG) performance status (PS) ≤ 2 in patients ≥17 years old; or Karnofsky/Lansky >50 in patients <16 years old. 11. Life expectancy of at least 3 months.

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT02657005
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Phase 1/Phase 2
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Oncternal Therapeutics, Inc
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

James Breitmeyer, MD
Principal Investigator Affiliation Oncternal Therapeutics
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Industry
Overall Status Recruiting
Countries United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Sarcoma, Ewing
Additional Details

The study has been expanded to explore single agent TK216 for longer treatment duration. Approximately 26 patients will be enrolled in this Cohort.

Arms & Interventions

Arms

Experimental: TK216 treatment

Dose escalation and expansion cohorts to determine dose-limiting toxicities, maximally tolerated dose, preliminary efficacy, and recommended phase 2 dose.

Interventions

Drug: - TK216

Inhibitor of protein-protein interactions of EWS-FLI1 fusion protein

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

UCLA Jonsson Comprehensive Cancer Center, Los Angeles, California

Status

Recruiting

Address

UCLA Jonsson Comprehensive Cancer Center

Los Angeles, California, 90095

Site Contact

Rubi Arias

rubiarias@mednet.ucla.edu

310-794-0345

Children's Hospital of Colorado, Aurora, Colorado

Status

Recruiting

Address

Children's Hospital of Colorado

Aurora, Colorado, 80045

Site Contact

Matthew O'hern, BS

matthew.O'hern@childrenscolorado.org

720-777-8557

Children's National Hospital, Washington, District of Columbia

Status

Recruiting

Address

Children's National Hospital

Washington, District of Columbia, 20010

Site Contact

Ann Liew, MS

aliew@childrensnational.org

202-476-6755

Memorial Sloan Kettering Cancer Center, New York, New York

Status

Recruiting

Address

Memorial Sloan Kettering Cancer Center

New York, New York, 10174

Site Contact

Paul Meyers, MD

meyersp@mskcc.org

833-675-5437

Duke Cancer Institute, Durham, North Carolina

Status

Recruiting

Address

Duke Cancer Institute

Durham, North Carolina, 27710

Site Contact

Stephanie Weaver, RN, BSN

stephanie.weaver@duke.edu

919-681-1883

Cleveland Clinic Foundation, Cleveland, Ohio

Status

Recruiting

Address

Cleveland Clinic Foundation

Cleveland, Ohio, 44195

Site Contact

Matteo Trucco, MD

TRUCCOM@ccf.org

216-308-2706

Oregon Health & Science University, Portland, Oregon

Status

Recruiting

Address

Oregon Health & Science University

Portland, Oregon, 97239

Site Contact

Brett Rodgers

rodgerbr@ohsu.edu

503-494-4395

Houston, Texas

Status

Completed

Address

Texas Children's Cancer & Hematology Centers, Baylor College

Houston, Texas, 77030

UT MD Anderson Cancer Center, Houston, Texas

Status

Recruiting

Address

UT MD Anderson Cancer Center

Houston, Texas, 77030

Site Contact

Joseph A Ludwig, MD

clinical@oncternal.com

713-792-3626

Stay Informed & Connected