ClinicalTrials.gg
TerminatedNCT03485729Updated Dec 24, 2024Results posted

ONC201 in Recurrent or Metastatic Type II Endometrial Cancer Endometrial Cancer

A Phase 2 interventional study of Dordaviprone (ONC201) in Endometrial Cancer Recurrent, sponsored by Chimerix. Terminated at 3 sites in United States. Open to female participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-12-24.

Sponsored by Chimerix (part of Jazz Pharmaceuticals) · Phase 2, Interventional, and Treatment

Why this study was terminated
This study was terminated due to a change in the Sponsor's corporate priorities. The decision to terminate the study was not based on any safety concerns.

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Phase
Phase 2
Study type
Interventional
Enrollment
27
Allocation
Non-randomized
Ages
18 Years and older
Sex
Female
01

Where this study stands

Counted across the records on this site · 2026-11-14

Among 714 studies in Endometrial Neoplasms with a site in United States — 197 open to participants now, 287 under way, 22 of them phase 3. Endometrial Neoplasms · United States.

Enrollment
435th of 590
27 · median 60
Started
2018
with 32 others
Phase 2
178 of 714
35 open now
Registered
319th of 714
by date

Among the 714 studies in Endometrial Neoplasms with a site in United States, this one is the 435th largest by enrollment of the 590 interventional ones that give a count, is one of 33 that started in 2018, is one of 178 in phase 2, and was the 319th registered.

Endometrial Neoplasms

1,426 studies on the registry are indexed under Endometrial Neoplasms; 471 are open to participants now.

Browse Endometrial Neoplasms studies →

Open to participants now

197 of the 714 studies in Endometrial Neoplasms with a site in United States are open now. Recruiting first, the newest first:

192 more — sign in →

All 197 open studies →

A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →

Who these studies take

Of the 357 Endometrial Neoplasms studies recruiting now, 267 (75%) set no upper age limit and 54 (15%) stop at 75 or younger; 26 take children under 18; 23 take healthy volunteers.

This study: 18 Years and older · female · no healthy volunteers.

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02

Study summary

This was a Phase 2, Simon two-stage, non-randomized, open-label, 2-arm trial of dordaviprone (ONC201) in women with metastatic or recurrent Type II endometrial cancer who failed at least 1 prior chemotherapy regimen. Patients with histologically confirmed Type II endometrial cancer, including but not limited to serous, clear cell, carcinosarcoma, adenosquamous, and mixed histologies were eligible.

The primary objective of this study was to determine the efficacy of dordaviprone (ONC201) in metastatic type II endometrial cancer.

Note: This study was completed by predecessor company, Oncoceutics, Inc.

Read the detailed description

This study included 3 arms: Arm A, Arm B, and Arm C. Dordaviprone (ONC201) was to be administered as a dose of 625 mg by mouth, once or twice each week until disease progression, unacceptable toxicity, or if the patient discontinued for any other reason.

For Arm A and Arm B, ONC201 administration occurred on Days 1, 8, and 15 of each 3-week cycle. For Arm C, ONC201 administration occurred on Days 1, 2, 8, 9, and 15, and 16 of each 3-week cycle.

All patients in Arm A had a biopsy of their tumor one day after the second dose of dordaviprone (ONC201) on Cycle 1, Day 9. All patients in Arm C had a biopsy of their tumor one day after the fourth dose of dordaviprone (ONC201) on Cycle 1, Day 10.

Assessments of objective tumor response were conducted using RECIST version 1.1. Safety was assessed through the reporting of adverse events, measurement of vital signs, electrocardiograms, and clinical laboratory results.

Before the study was terminated, a total of 27 patients were enrolled and received at least 1 dose of dordaviprone (ONC201): 10 patients in Arm A, 14 patients in Arm B, and 3 patients in Arm C.

03

Conditions studied

  • Endometrial Cancer Recurrent
04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
Female
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

A patient had to meet all of the following criteria to be eligible to participate in the study:

  1. Had confirmed or recurrent Type II endometrial cancer (serous, clear cell, carcinosarcoma, adenosquamous, and mixed histologies). For patients with tumors that had mixed histologies, the tumor should have had evidence of some tumor cells with Type II endometrial cancer features.
  2. Must have had , defined as at least 1 lesion that could be accurately measured in at least 1 dimension in accordance with (RECIST) version 1.1.
  3. Had availability of at least 12 unstained slides from archival formalin-fixed paraffin-embedded (FFPE) tumor tissue. Available archived tissue were provided for correlative studies.
  4. For Arm A and Arm C, patients must have had disease that was amendable to biopsy and must have been willing to provide consent for a tumor biopsy at baseline (within 30 days of beginning ONC201) an at least 1 on-treatment tumor biopsy.
  5. Must have had radiographic disease progression after at least 1 line of systemic cytotoxic therapy for metastatic disease or with progression within 12 months of completing chemotherapy.
  6. Were aged ≥18 years.
  7. Had an (ECOG) performance status of 0, 1, or 2.
  8. Must have had adequate bone marrow, hepatic and renal function, as defined below:

    • Leukocytes: ≥3000/mcL
    • : ≥1500/mcL
    • : ≥100,000/mcL
    • Total : ≤1.5 (ULN)
    • /Alanine aminotransferase (SGOT/SGPT): ≤2 ULN
    • Creatinine: ≤1.5 ULN OR
    • : ≥60 mL/min/1.732 for patients with creatinine levels above ULN calculated using Calvert formula
  9. Had a of at least 3 months.
  10. Had the ability to understand and willingness to sign a written and Health Insurance Portability and Accountability Act (HIPAA) consent document.
  11. Must have been surgically sterile or or must have agreed to use effective during the period of the trial and for at least 90 days after completion of treatment.

Exclusion criteria

Exclusion Criteria:

A potential patient who met any of the following criteria was ineligible to participate in the study:

  1. Had any prior treatment with ONC201.
  2. Had chemotherapy or radiotherapy within 4 weeks prior to entering the study or those who had not recovered from adverse events (AEs) due to agents administered more than 4 weeks earlier.
  3. Patients who had not recovered to baseline or (CTCAE) Grade ≤2 from related toxicity to all prior therapies were excluded. Patients with non-serious AEs such as alopecia, fatigue, weakness, loss of appetite, and nausea that were non-significant were not excluded.
  4. Had any other prior from which the patient had been disease-free for less than 3 years, with the exception of adequately treated and cured basal or squamous cell skin cancer, superficial bladder cancer, carcinoma in situ of any site.
  5. Were unable to swallow capsules.
  6. Had impairment of gastrointestinal (GI) function or GI disease that may have significantly altered the absorption of ONC201 (uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection).
  7. Were receiving any other agents.
  8. Patients with symptomatic were excluded. Patients with asymptomatic and treated central nervous system (CNS) may have participated in this trial. The patient must have completed any prior treatment for CNS metastases >28 days prior to study entry including radiotherapy or surgery. for the treatment of brain metastasis were not permitted, and patients must have been stable off steroid treatment for 4 weeks prior to enrollment.
  9. Had uncontrolled intercurrent illness including but not limited to ongoing or active infection. Or any of the following in the 6 months previous to enrollment: myocardial infarction, severe/unstable angina, coronary/peripheral artery bypass graft, cerebrovascular accident, , or symptomatic pulmonary embolism.
  10. Had active inflammatory GI disease, chronic diarrhea (unless related to underlying malignancy or prior related treatment) or history of abdominal fistula, GI perforation, peptic ulcer disease, or intra-abdominal abscess within 6 months prior to study enrollment. Gastroesophageal reflux disease under treatment with proton pump inhibitors was allowed.
  11. Known (HIV)-positive patients on combination antiretroviral therapy.
  12. Had active virus (HBV) or virus (HCV) infection. Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may have increased the risk associated with study participation or study drug administration or may have interfered with the interpretation of study results, or in the judgement of the Investigator, would have made the patient inappropriate for entry into the study.
  13. Were pregnant or breast feeding.
  14. Had known history of cardiac arrhythmias including atrial fibrillation, tachyarrhythmias, or bradycardia, unless arrhythmia was controlled and after cardiology had cleared patient to receive ONC201. Was receiving therapeutic agents known to prolong ; however, the use of Zofran was permitted. Had a history of congestive heart failure or myocardial infarction or stroke in the 3 months prior to enrollment.
  15. Concomitant use of potent cytochrome P450 (CYP) A4/5 inhibitors or inducers during the treatment phase of the study and within 2 hours prior to starting study drug administration.
Terms in these criteria, in plain language(27)

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.

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.

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.

Biopsy and archival tissue

A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.

Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.

Adjuvant and neoadjuvant

Neoadjuvant treatment is given before the main treatment (usually surgery), often to shrink a tumour. Adjuvant treatment is given after it, to lower the chance of the cancer coming back.

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.

Absolute neutrophil count (ANC)

The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.

It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.

Platelets

Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.

Bilirubin

A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.

Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.

ULN (upper limit of normal)

The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.

“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.

ALT and AST (liver enzymes)

Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.

Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.

Creatinine clearance (CrCl)

How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.

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.

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.

Postmenopausal

Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).

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.

CTCAE grade

The US National Cancer Institute’s scale of how severe an adverse event is — any unwanted medical event during a study, such as a symptom, an illness or an abnormal test result, whether or not a treatment caused it. Whether it did is judged separately.

1
Mild.
2
Moderate.
3
Severe.
4
Life-threatening.
5
Death due to the event.

“Side effects of earlier treatment resolved to grade 1 or less” means they have settled to mild or gone.

Cancer (malignancy)

Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.

Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

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.

Corticosteroids (steroids)

Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.

Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.

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.

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.

Hepatitis B tests

Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.

Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.

Hepatitis C tests

Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.

After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.

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.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
27 participants (actual)

Study arms

  • Experimental
    Arm A

    Patients received 625 mg dordaviprone (ONC201) once weekly on Days 1, 8, and 15 of each 3-week cycle. Patients were required to undergo a biopsy of their tumor one day after their second dose of dordaviprone (ONC201) on Cycle 1, Day 9.

    Drug: Dordaviprone (ONC201)

  • Experimental
    Arm B

    Patients received 625 mg dordaviprone (ONC201) once weekly on Days 1, 8, and 15 of each 3-week cycle. Patients were not required to undergo a biopsy of their tumor.

    Drug: Dordaviprone (ONC201)

  • Experimental
    Arm C

    Patients received 625 mg dordaviprone (ONC201) twice weekly on consecutive days on Days 1, 2, 8, 9, 15, and 16 of each 3-week cycle. Patients were required to undergo a biopsy of their tumor one day after their fourth dose of dordaviprone (ONC201) on Cycle 1, Day 10.

    Drug: Dordaviprone (ONC201)

Interventions

06

What researchers measure

Primary outcomes

  1. Progression-Free Survival Rate at 2 Months

    Progression-free survival rate at 2 months was defined as the percentage of participants who exhibited progression-free survival for \>8 weeks (\>56 days) following treatment initiation; only Arm B participants were analyzed for this outcome.

    Time frame: 2 months (8 weeks); from treatment initiation to 2 months (8 weeks) following treatment initiation

07

Results

Posted Dec 24, 2024
Limitations and caveats
Note: This study was terminated due to a change in corporate priorities. The decision to terminate the study was not based on any safety concerns with dordaviprone (ONC201).

Participant flow

Participant flow — Overall Study
MilestoneArm AArm BArm C
Started10143
Completed001
Not completed10142
Withdrew: Withdrawal by subject001
Withdrew: Disease progression8131
Withdrew: Hospice care100
Withdrew: Poor performance status100
Withdrew: Illness010

Outcome measures

PrimaryProgression-Free Survival Rate at 2 Months

Progression-free survival rate at 2 months was defined as the percentage of participants who exhibited progression-free survival for \>8 weeks (\>56 days) following treatment initiation; only Arm B participants were analyzed for this outcome.

Time frame:
2 months (8 weeks); from treatment initiation to 2 months (8 weeks) following treatment initiation
Reported as:
Count of participants · Participants
Progression-Free Survival Rate at 2 Months
ParticipantsArm B
Progression-Free Survival Rate at 2 Months3

Adverse events

Collected over From the time/date of initiation of study treatment through 30 days following cessation of study treatment (32 months). Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Arm A5/10 (50%)7/10 (70%)10/10 (100%)
Arm B4/14 (28.6%)4/14 (28.6%)14/14 (100%)
Arm C2/3 (66.7%)3/3 (100%)3/3 (100%)
Most frequent serious events
Showing 10 of 28
Most frequent serious events
EventArm AArm BArm C
DyspnoeaRespiratory, thoracic and mediastinal disorders1/101/142/3
Atrial fibrillationCardiac disorders0/100/141/3
Cardiac failureCardiac disorders0/100/141/3
AscitesGastrointestinal disorders0/101/141/3
Disease progressionGeneral disorders1/100/141/3
Oedema peripheralGeneral disorders0/100/141/3
HyponatraemiaMetabolism and nutrition disorders0/100/141/3
Mental status changesPsychiatric disorders0/100/141/3
Pelvic painReproductive system and breast disorders0/100/141/3
Respiratory failureRespiratory, thoracic and mediastinal disorders0/100/141/3
Most frequent other events
Showing 10 of 112
Most frequent other events
EventArm AArm BArm C
HyponatraemiaMetabolism and nutrition disorders0/101/142/3
DyspnoeaRespiratory, thoracic and mediastinal disorders2/106/142/3
FatigueGeneral disorders6/109/141/3
NauseaGastrointestinal disorders4/108/140/3
AnaemiaBlood and lymphatic system disorders4/106/141/3
VomitingGastrointestinal disorders4/103/140/3
ConstipationGastrointestinal disorders3/105/141/3
Atrial fibrillationCardiac disorders0/101/141/3
Cardiac failureCardiac disorders0/100/141/3
AscitesGastrointestinal disorders0/101/141/3

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)Arm AArm BArm CTotal
<=18 years0000
Between 18 and 65 years65011
>=65 years49316
Sex: Female, Male
Sex: Female, Male(Participants)Arm AArm BArm CTotal
Female1014327
Male0000
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Arm AArm BArm CTotal
Hispanic or Latino2002
Not Hispanic or Latino813324
Unknown or Not Reported0101
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Arm AArm BArm CTotal
American Indian or Alaska Native0000
Asian0101
Native Hawaiian or Other Pacific Islander0000
Black or African American4419
White58215
More than one race0000
Unknown or Not Reported1102
Histologically confirmed metastatic or recurrent Type II endometrial cancer
Histologically confirmed metastatic or recurrent Type II endometrial cancer(Participants)Arm AArm BArm CTotal
Count of participants1014327
08

Study locations

3 sites
  • Rutgers, The State University of New Jersey
    New Brunswick, New Jersey 08903, United States
  • UNC Lineberger Comprehensive Cancer Center
    Chapel Hill, North Carolina 27599, United States
  • Fox Chase Cancer Center
    Philadelphia, Pennsylvania 19111, United States
09

References and documents

Study documents

PDFs hosted by the registry, ClinicalTrials.gov.

10

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Ran from Mar 2018 to primary completion in Jan 2023, 57 months.

Results were first posted in Dec 2024, 1 year 11 months after primary completion.

How these figures are worked out, and which studies are left out of them.

Registered
Mar 25, 2018
First posted
Apr 2, 2018
StartActual
Mar 21, 2018
Primary completionActual
Jan 19, 2023
CompletionActual
Jan 19, 2023
Results first posted
Dec 24, 2024
Last update
Dec 24, 2024
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Dec 24, 2024, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT03485729
Lead sponsor
Chimerix
Collaborators
Oncoceutics, Inc.
Responsible party
Sponsor
First posted
Apr 2, 2018
Start date
Mar 21, 2018
Primary completion
Jan 19, 2023
Completion
Jan 19, 2023
Results posted
Dec 24, 2024
Last update
Dec 24, 2024

Oversight

FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is terminated, as verified in Dec 2024. You cannot join it, but the record below documents what was studied.

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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.

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Studies of the drug tested here: Dordaviprone

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