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
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.
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.
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 →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.
Patients who meet the following inclusion criteria will be eligible to participate in this study:
Have or cytologically confirmed diagnosis of Ewing sarcoma (including ESFT) with disease
Prior cancer therapy:
Prior radiotherapy is allowed
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)
Major Exclusion Criteria:
Patients will not be enrolled if they meet any one of the following exclusion criteria:
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.
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.
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: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Metastases
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
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.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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
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
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.
| Milestone | 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 Cohort | 175 mg/m^2 (28 Days) |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Started | 3 | 3 | 3 | 3 | 4 | 4 | 3 | 3 | 7 | 44 | 8 |
| Completed | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Not completed | 3 | 3 | 3 | 3 | 4 | 4 | 3 | 3 | 7 | 44 | 8 |
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.
| 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 Cohort | 175 mg/m^2 (28 Days) |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Response Rate | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 3 | 0 |
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.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| 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%) |
| Expansion | 26/44 (59.1%) | 21/44 (47.7%) | 44/44 (100%) |
| 175 mg/m^2 | 1/8 (12.5%) | 5/8 (62.5%) | 8/8 (100%) |
| Event | 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 | 175 mg/m^2 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Febrile NeutropeniaBlood and lymphatic system disorders | 0/3 | 0/3 | 0/3 | 1/3 | 2/4 | 0/4 | 1/3 | 1/3 | 0/7 | 4/44 | 2/8 |
| Aortobronchial fistulaRespiratory, thoracic and mediastinal disorders | 1/3 | 0/3 | 0/3 | 0/3 | 0/4 | 0/4 | 0/3 | 0/3 | 0/7 | 0/44 | 0/8 |
| Device Related InfectionInfections and infestations | 0/3 | 1/3 | 0/3 | 0/3 | 0/4 | 0/4 | 0/3 | 0/3 | 1/7 | 2/44 | 0/8 |
| Hypoglossal nerve paralysisNervous system disorders | 0/3 | 0/3 | 1/3 | 0/3 | 0/4 | 0/4 | 0/3 | 0/3 | 0/7 | 0/44 | 0/8 |
| Influenza like illnessGeneral disorders | 0/3 | 0/3 | 0/3 | 0/3 | 0/4 | 0/4 | 1/3 | 0/3 | 0/7 | 0/44 | 0/8 |
| HaematemesisGastrointestinal disorders | 0/3 | 0/3 | 0/3 | 0/3 | 1/4 | 0/4 | 0/3 | 0/3 | 0/7 | 0/44 | 0/8 |
| Cancer PainNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 0/3 | 0/3 | 0/3 | 0/3 | 1/4 | 1/4 | 0/3 | 0/3 | 0/7 | 1/44 | 0/8 |
| Skin InfectionInfections and infestations | 0/3 | 0/3 | 0/3 | 0/3 | 1/4 | 0/4 | 0/3 | 0/3 | 0/7 | 0/44 | 0/8 |
| Periorbital cellulitisInfections and infestations | 0/3 | 0/3 | 0/3 | 0/3 | 1/4 | 0/4 | 0/3 | 0/3 | 0/7 | 0/44 | 0/8 |
| Abdominal PainGastrointestinal disorders | 0/3 | 0/3 | 0/3 | 0/3 | 0/4 | 0/4 | 0/3 | 0/3 | 1/7 | 0/44 | 1/8 |
| Event | 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 | 175 mg/m^2 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NeutropeniaBlood and lymphatic system disorders | 0/3 | 0/3 | 0/3 | 1/3 | 3/4 | 2/4 | 0/3 | 3/3 | 4/7 | 26/44 | 6/8 |
| AnaemiaBlood and lymphatic system disorders | 0/3 | 1/3 | 2/3 | 2/3 | 4/4 | 1/4 | 0/3 | 3/3 | 4/7 | 22/44 | 5/8 |
| LeukopeniaBlood and lymphatic system disorders | 0/3 | 0/3 | 0/3 | 1/3 | 2/4 | 2/4 | 0/3 | 3/3 | 4/7 | 18/44 | 2/8 |
| FatigueGeneral disorders | 0/3 | 0/3 | 1/3 | 2/3 | 2/4 | 2/4 | 1/3 | 3/3 | 3/7 | 18/44 | 1/8 |
| PyrexiaGeneral disorders | 0/3 | 2/3 | 1/3 | 1/3 | 2/4 | 2/4 | 1/3 | 3/3 | 3/7 | 15/44 | 1/8 |
| Lymphocyte cunt decreaseInvestigations | 1/3 | 0/3 | 0/3 | 0/3 | 3/4 | 2/4 | 0/3 | 0/3 | 1/7 | 5/44 | 2/8 |
| AlopeciaSkin and subcutaneous tissue disorders | 0/3 | 0/3 | 0/3 | 0/3 | 1/4 | 3/4 | 0/3 | 2/3 | 2/7 | 16/44 | 3/8 |
| DyspnoeaRespiratory, thoracic and mediastinal disorders | 0/3 | 0/3 | 1/3 | 1/3 | 0/4 | 3/4 | 0/3 | 1/3 | 0/7 | 9/44 | 0/8 |
| ThrombocytopeniaBlood and lymphatic system disorders | 0/3 | 0/3 | 1/3 | 2/3 | 2/4 | 0/4 | 0/3 | 2/3 | 3/7 | 6/44 | 4/8 |
| Febrile neutropeniaBlood and lymphatic system disorders | 0/3 | 0/3 | 0/3 | 1/3 | 1/4 | 0/4 | 0/3 | 2/3 | 3/7 | 4/44 | 2/8 |
Safety Population
| 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 Cohort | 175 mg/m^2 (28 Days) | Total |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Mean | 28.0 ± 5.3 | 38.7 ± 28.7 | 41.3 ± 12.7 | 33.3 ± 10.7 | 30.0 ± 9.5 | 25.5 ± 7.9 | 24.7 ± 8.1 | 24.0 ± 10.0 | 23.7 ± 6.3 | 29.2 ± 14.6 | 27.0 ± 10.0 | 28.9 ± 13.1 |
| 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 Cohort | 175 mg/m^2 (28 Days) | Total |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Female | 1 | 3 | 1 | 0 | 0 | 1 | 2 | 1 | 2 | 16 | 2 | 29 |
| Male | 2 | 0 | 2 | 3 | 4 | 3 | 1 | 2 | 5 | 28 | 6 | 56 |
| 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 Cohort | 175 mg/m^2 (28 Days) | Total |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 | 1 | 0 | 0 | 0 | 0 | 5 | 1 | 0 | 7 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 1 | 1 | 2 |
| Black or African American | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| White | 3 | 2 | 3 | 2 | 4 | 4 | 3 | 2 | 0 | 41 | 5 | 69 |
| More than one race | 0 | 1 | 0 | 0 | 0 | 0 | 0 | 0 | 2 | 1 | 0 | 4 |
| Unknown or Not Reported | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 1 | 0 | 0 | 2 | 3 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
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Studies of the drug tested here: Ets-family Transcription Factor Inhibitor TK216
Oncternal Therapeutics, Inc