ClinicalTrials.gg
TerminatedNCT02657005Updated Feb 12, 2025Results posted

TK216 in Patients With Relapsed or Refractory Ewing Sarcoma

A Phase 1/2 interventional study of TK216 in Sarcoma, Ewing, sponsored by Oncternal Therapeutics, Inc. Terminated at 9 sites in United States. Open to participants aged 8 Years and older. Per ClinicalTrials.gov, last updated 2025-02-12.

Sponsored by Oncternal Therapeutics, Inc · Phase 1/2, Interventional, and Treatment

Why this study was terminated
Sponsor Decision
Phase
Phase 1/2
Study type
Interventional
Enrollment
85
Allocation
Not applicable
Ages
8 Years and older
Sex
All
01

Study 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.

Read the detailed description

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

Please note: This study has been terminated and is no longer enrolling patients.

02

Conditions studied

  • Sarcoma, Ewing

Browse trials for

03

In context

Sarcoma, Ewing

250 studies on the registry are indexed under Sarcoma, Ewing; 69 are open to participants now.

This study's enrollment of 85 is above the median of 36 across 204 interventional studies indexed under Sarcoma, Ewing.

Browse Sarcoma, Ewing studies →

Lead sponsor

Oncternal Therapeutics, Inc is the lead sponsor of 5 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
8 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

  1. Major Inclusion Criteria (for all Study Parts or protocol versions unless specifically noted):

Patients who meet the following inclusion criteria will be eligible to participate in this study:

  1. Willing and able to provide written IRB/IEC-approved . For patients \< 18 years of age, their parent or legal guardian must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines.
  2. Have or cytologically confirmed diagnosis of Ewing sarcoma (including ESFT) with disease

    1. who have failed standard therapy and for whom no known curative therapy exists (For Parts 1-3), OR
    2. patients with disease who had standard chemotherapy at the time of diagnosis (For Part 4) (NOTE: as of Protocol version 7, pathology reports and slides or blocks should be available for review or additional testing. If not available, site must discuss with Sponsor.)
  3. according to version 1.1. Measurable disease can be verified from a previously documented 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:

    1. Patients may have received any number of prior therapy regimens (For Parts 1-3) OR
    2. 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 (For Part 4).
  6. Prior radiotherapy is allowed

    1. If ≥ 2 weeks have elapsed for local palliative XRT (small port); ≥ 6 months must have elapsed if prior total body irradiation, craniospinal XRT 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 (For Parts 1-3) OR
    2. 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 (For Part 4).
  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.
  9. (ECOG) performance status (PS) ≤ 2 in patients ≥17 years old; or /Lansky >50 in patients \<16 years old.
  10. Age:

    a. Cohort 1: Patient's age >18 years old. b. Cohorts 2-6: Patients must be > 12 years old. c. Cohorts 7-10: Patients must be ≥ 10 years old. d. Cohort 11: Patient's age ≥ 8 years (For Part 4)

  11. of at least 3 months.
  12. Adequate organ function as measured by baseline laboratory values. 13. Cardiac > 50% or shortening fraction > 28%. 14. Females of must have a negative pregnancy test (within 7-days of starting treatment) during screening and subjects must be willing to use effective . be neither breastfeeding nor intending to become pregnant during study participation. Females of childbearing potential must agree to avoid pregnancy during the study and commit to abstinence from heterosexual intercourse or agree to use two methods of birth control (one highly effective method and one additional effective method) at least 4 weeks before the start of protocol therapy, for the duration of study participation, and for 6 months after the last dose of TK216. 15. Males with partner(s) of childbearing potential must take appropriate precautions to avoid fathering a child from the screening period until 90 days after receiving the last dose of TK216. They must commit to abstinence from heterosexual intercourse or agree to use appropriate barrier contraception.
  1. Willingness and ability to comply with scheduled visits, treatment plan, laboratory tests and other study procedures.

Major Exclusion Criteria:

Patients will not be enrolled if they meet any one of the following exclusion criteria:

  1. Current participation in another therapeutic clinical trial.
  2. Symptomatic .
  3. History of previous cancer (non ES), except squamous cell or basal-cell carcinoma of the skin or any in situ carcinoma that has been completely resected, which required therapy within the previous 3 years.
  4. Any of the following in the past 6 months: symptomatic congestive heart failure, cerebrovascular accident or , pulmonary embolism, deep vein thrombosis, symptomatic bradycardia, requirement for antiarrhythmic medication.
  5. History of prolonged (e.g., repeated demonstration of a QTc interval > 450 milliseconds, unless associated with the use of medications known to prolong the QTc interval). (NOTE: For Part 4, repeated demonstration of a QTc interval > 470 milliseconds) 6. History of additional risk factors for torsade de pointes (e.g., heart failure, family history of long QT syndrome
  1. Use of concomitant medications that increase or possibly increase the risk to prolong the QTc interval and/or induce torsades de pointes ventricular arrhythmia.
  1. Females who are breastfeeding/lactating. 9. Known active infections (bacterial, fungal, viral including hepatitis and positivity). 10. Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or study drug administration or may interfere with the interpretation of study results and, in the judgment of the Investigator, would make the patient inappropriate for entry into this study or could compromise protocol objectives in the opinion of the Investigator and/or the Sponsor.
Terms in these criteria, in plain language(16)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Histologically or cytologically confirmed

The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).

Your pathology report is the document that shows this.

Relapsed and refractory

Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.

Metastases

Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.

Measurable disease

The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.

The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.

RECIST

The standard rules for measuring tumours on CT or MRI scans, to tell whether a cancer is shrinking, stable or growing during treatment.

“Measurable disease per RECIST 1.1” usually means at least one tumour 10 mm or more at its longest on a CT scan, or a lymph node 15 mm or more across its short axis.

ECOG performance status

A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.

0
Fully active, no restrictions.
1
Limited in strenuous activity, but walking and able to do light work, such as housework or office work.
2
Walking and able to look after yourself, but unable to work; up and about more than half of waking hours.
3
Able to look after yourself only in part; in bed or a chair more than half of waking hours.
4
Unable to look after yourself at all; in bed or a chair all day.

Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.

Karnofsky performance status (KPS)

A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.

Studies often ask for 60 or 70 and above. Your doctor gives the score.

Life expectancy

The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.

LVEF (left ventricular ejection fraction)

The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.

Childbearing potential

Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).

Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

Brain metastases

Cancer deposits in the brain from a cancer that started elsewhere in the body (CNS metastases include the spinal cord).

Studies differ: some leave out any brain metastases, some accept ones treated and stable for some weeks. Check what this study allows.

TIA (transient ischaemic attack)

A “mini-stroke”: temporary stroke-like symptoms caused by reduced blood flow to part of the brain, spinal cord or eye, without signs of a new stroke on a scan.

Stroke symptoms need emergency care at once, even if they go away.

QT and QTc

On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.

The study may name a correction formula and a limit in milliseconds, often around 450–480.

HIV

The human immunodeficiency virus, which weakens the immune system.

Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.

05

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
85 participants (actual)

Study arms

  • 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

Interventions

  • DrugTK216

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

06

What researchers measure

Primary outcomes

  1. Overall Response Rate

    Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions and pathologic lymph nodes; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions, no new lesions, and no progression of non-target lesions; Overall Response (OR) = CR + PR.

    Time frame: 36 months

07

Results

Posted Nov 7, 2024
Limitations and caveats
TK216 was advanced as a novel inhibitor of EWS::FLI1 despite pharmacologic challenges to creating an oral formulation. In vivo studies showed that continuous exposure to TK216 was required for optimal efficacy.47 This continuous infusion was a logistical challenge and negatively affected study enrollment rates. Another limitation of this study was the lack of a pharmacodynamic readout of TK216 activity.

Participant flow

85 subjects were enrolled in this study across eight North American centers. The pathologic diagnoses were confirmed at each enrolling center. Most patients were heavily pretreated, with the median number of prior therapies being 3.0 (range, 1-10). The gender and ethnicity of the enrolled patients matched ES's prevalence in the U.S. population.

Participant flow — Overall Study
Milestone18 mg/m^2 (7 Days)36 mg/m^2 (7 Days)72 mg/m^2 (7 Days)144 mg/m^2 (7 Days)200 mg/m^2 (10 Days)200 mg/m^2 (14 Days)220 mg/m^2 (7 Days)220 mg/m^2 (10 Days)288 mg/m^2 (7 Days)Expansion Cohort175 mg/m^2 (28 Days)
Started333344337448
Completed00000000000
Not completed333344337448

Outcome measures

PrimaryOverall Response Rate

Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions and pathologic lymph nodes; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions, no new lesions, and no progression of non-target lesions; Overall Response (OR) = CR + PR.

Time frame:
36 months
Reported as:
Count of participants · Participants
Overall Response Rate
Participants18 mg/m^2 (7 Days)36 mg/m^2 (7 Days)72 mg/m^2 (7 Days)144 mg/m^2 (7 Days)200 mg/m^2 (10 Days)200 mg/m^2 (14 Days)220 mg/m^2 (7 Days)220 mg/m^2 (10 Days)288 mg/m^2 (7 Days)Expansion Cohort175 mg/m^2 (28 Days)
Overall Response Rate00000000030

Adverse events

Collected over Treatment emergent adverse events were collected from first treatment received through end of study for all patients. (i.e., 36 months). Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
18 mg/m^2 (7 Days)2/3 (66.7%)1/3 (33.3%)3/3 (100%)
36 mg/m^2 (7 Days)2/3 (66.7%)1/3 (33.3%)3/3 (100%)
72 mg/m^2 (7 Days)3/3 (100%)1/3 (33.3%)3/3 (100%)
144 mg/m^2 (7 Days)3/3 (100%)1/3 (33.3%)3/3 (100%)
200 mg/m^2 (10 Days)4/4 (100%)4/4 (100%)4/4 (100%)
200 mg/m^2 (14 Days)2/4 (50%)1/4 (25%)4/4 (100%)
220 mg/m^2 (7 Days)2/3 (66.7%)2/3 (66.7%)3/3 (100%)
220 mg/m^2 (10 Days)3/3 (100%)1/3 (33.3%)3/3 (100%)
288 mg/m^2 (7 Days)3/7 (42.9%)2/7 (28.6%)7/7 (100%)
Expansion26/44 (59.1%)21/44 (47.7%)44/44 (100%)
175 mg/m^21/8 (12.5%)5/8 (62.5%)8/8 (100%)
Most frequent serious events
Showing 10 of 36
Most frequent serious events
Event18 mg/m^2 (7 Days)36 mg/m^2 (7 Days)72 mg/m^2 (7 Days)144 mg/m^2 (7 Days)200 mg/m^2 (10 Days)200 mg/m^2 (14 Days)220 mg/m^2 (7 Days)220 mg/m^2 (10 Days)288 mg/m^2 (7 Days)Expansion175 mg/m^2
Febrile NeutropeniaBlood and lymphatic system disorders0/30/30/31/32/40/41/31/30/74/442/8
Aortobronchial fistulaRespiratory, thoracic and mediastinal disorders1/30/30/30/30/40/40/30/30/70/440/8
Device Related InfectionInfections and infestations0/31/30/30/30/40/40/30/31/72/440/8
Hypoglossal nerve paralysisNervous system disorders0/30/31/30/30/40/40/30/30/70/440/8
Influenza like illnessGeneral disorders0/30/30/30/30/40/41/30/30/70/440/8
HaematemesisGastrointestinal disorders0/30/30/30/31/40/40/30/30/70/440/8
Cancer PainNeoplasms benign, malignant and unspecified (incl cysts and polyps)0/30/30/30/31/41/40/30/30/71/440/8
Skin InfectionInfections and infestations0/30/30/30/31/40/40/30/30/70/440/8
Periorbital cellulitisInfections and infestations0/30/30/30/31/40/40/30/30/70/440/8
Abdominal PainGastrointestinal disorders0/30/30/30/30/40/40/30/31/70/441/8
Most frequent other events
Showing 10 of 36
Most frequent other events
Event18 mg/m^2 (7 Days)36 mg/m^2 (7 Days)72 mg/m^2 (7 Days)144 mg/m^2 (7 Days)200 mg/m^2 (10 Days)200 mg/m^2 (14 Days)220 mg/m^2 (7 Days)220 mg/m^2 (10 Days)288 mg/m^2 (7 Days)Expansion175 mg/m^2
NeutropeniaBlood and lymphatic system disorders0/30/30/31/33/42/40/33/34/726/446/8
AnaemiaBlood and lymphatic system disorders0/31/32/32/34/41/40/33/34/722/445/8
LeukopeniaBlood and lymphatic system disorders0/30/30/31/32/42/40/33/34/718/442/8
FatigueGeneral disorders0/30/31/32/32/42/41/33/33/718/441/8
PyrexiaGeneral disorders0/32/31/31/32/42/41/33/33/715/441/8
Lymphocyte cunt decreaseInvestigations1/30/30/30/33/42/40/30/31/75/442/8
AlopeciaSkin and subcutaneous tissue disorders0/30/30/30/31/43/40/32/32/716/443/8
DyspnoeaRespiratory, thoracic and mediastinal disorders0/30/31/31/30/43/40/31/30/79/440/8
ThrombocytopeniaBlood and lymphatic system disorders0/30/31/32/32/40/40/32/33/76/444/8
Febrile neutropeniaBlood and lymphatic system disorders0/30/30/31/31/40/40/32/33/74/442/8

Baseline characteristics

Safety Population

Age, Continuous
Age, Continuous(years)18 mg/m^2 (7 Days)36 mg/m^2 (7 Days)72 mg/m^2 (7 Days)144 mg/m^2 (7 Days)200 mg/m^2 (10 Days)200 mg/m^2 (14 Days)220 mg/m^2 (7 Days)220 mg/m^2 (10 Days)288 mg/m^2 (7 Days)Expansion Cohort175 mg/m^2 (28 Days)Total
Mean28.0 ± 5.338.7 ± 28.741.3 ± 12.733.3 ± 10.730.0 ± 9.525.5 ± 7.924.7 ± 8.124.0 ± 10.023.7 ± 6.329.2 ± 14.627.0 ± 10.028.9 ± 13.1
Sex: Female, Male
Sex: Female, Male(Participants)18 mg/m^2 (7 Days)36 mg/m^2 (7 Days)72 mg/m^2 (7 Days)144 mg/m^2 (7 Days)200 mg/m^2 (10 Days)200 mg/m^2 (14 Days)220 mg/m^2 (7 Days)220 mg/m^2 (10 Days)288 mg/m^2 (7 Days)Expansion Cohort175 mg/m^2 (28 Days)Total
Female13100121216229
Male20234312528656
Race (NIH/OMB)
Race (NIH/OMB)(Participants)18 mg/m^2 (7 Days)36 mg/m^2 (7 Days)72 mg/m^2 (7 Days)144 mg/m^2 (7 Days)200 mg/m^2 (10 Days)200 mg/m^2 (14 Days)220 mg/m^2 (7 Days)220 mg/m^2 (10 Days)288 mg/m^2 (7 Days)Expansion Cohort175 mg/m^2 (28 Days)Total
American Indian or Alaska Native000000000000
Asian000100005107
Native Hawaiian or Other Pacific Islander000000000112
Black or African American000000000000
White32324432041569
More than one race010000002104
Unknown or Not Reported000000010023
08

Study locations

9 sites
  • UCLA Jonsson Comprehensive Cancer Center
    Los Angeles, California 90095, United States
  • Children's Hospital of Colorado
    Aurora, Colorado 80045, United States
  • Children's National Hospital
    Washington, District of Columbia 20010, United States
  • Memorial Sloan Kettering Cancer Center
    New York, New York 10174, United States
  • Duke Cancer Institute
    Durham, North Carolina 27710, United States
  • Cleveland Clinic Foundation
    Cleveland, Ohio 44195, United States
  • Oregon Health & Science University
    Portland, Oregon 97239, United States
  • Texas Children's Cancer & Hematology Centers, Baylor College
    Houston, Texas 77030, United States
  • UT MD Anderson Cancer Center
    Houston, Texas 77030, United States
09

References and documents

Publications

  • Meyers PA, Federman N, Daw N, Anderson PM, Davis LE, Kim A, Macy ME, Pietrofeso A, Ratan R, Riedel RF, Trucco M, Breitmeyer JB, Toretsky JA, Ludwig JA. Open-Label, Multicenter, Phase I/II, First-in-Human Trial of TK216: A First-Generation EWS::FLI1 Fusion Protein Antagonist in Ewing Sarcoma. J Clin Oncol. 2024 Nov;42(31):3725-3734. doi: 10.1200/JCO.24.00020. Epub 2024 Jul 2. PubMed 38954782 ↗

Study documents

PDFs hosted by the registry, ClinicalTrials.gov.

Individual participant data

Plan to share: No

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Feb 12, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT02657005
Lead sponsor
Oncternal Therapeutics, Inc
Responsible party
Sponsor
First posted
Jan 15, 2016
Start date
Aug 2016
Primary completion
May 2022
Completion
Jun 2022
Results posted
Nov 7, 2024
Last update
Feb 12, 2025

Study contacts

James Breitmeyer, MD
study director · Oncternal Therapeutics

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is terminated, as verified in Jan 2025. You cannot join it, but the record below documents what was studied.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Studies of the drug tested here: Ets-family Transcription Factor Inhibitor TK216

Start the discussion