ONC201 in Recurrent/Refractory Metastatic Breast Cancer and Advanced Endometrial Carcinoma
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03394027|
Recruitment Status : Recruiting
First Posted : January 9, 2018
Last Update Posted : February 6, 2018
The new drug ONC201 have been shown to kill breast cancer and endometrial cancer cells in the laboratory. The exact mechanism of action is not completely clear yet, but the ONC201 destroys the mitochondria inside the cells. Blocking mitochondrial activity may kill tumor cells, which would shrink tumors. Researchers want to see if ONC201 helps shrink tumors of certain breast or endometrial cancers and if that effect is maintained.
To see if ONC201 shrinks tumors with a lasting effect.
Adults ages 18 and older who have metastatic breast cancer (hormone-positive or triple-negative) or metastatic endometrial cancers.
Participants will be screened with:
<TAB>Heart, blood, and urine tests
<TAB>CT and bone scans
<TAB>Review of medical report and tumor sample
Participants will have a tumor biopsy before starting treatment and after 5 weeks taking the study drug. A scan or ultrasound may be used to guide the biopsy. Patients will receive local anesthetic and a needle will remove a small piece of tumor.
The study will be done in 28-day cycles. Every day 1 of each cycle participants will repeat most screening tests, will be seen by the physician and receive a supply of the study drug.
Participants will take the study drug by mouth once every 7 days. They will keep a diary of when they take the drug and any side effects. During cycle 1, participants will get weekly calls to discuss their health and symptoms. Images will be repeated every 2 cycles to evaluate reponse to the treatment.
|Condition or disease||Intervention/treatment||Phase|
|Triple Negative Breast Cancer Endometrial Cancer Hormone Receptor Positive, HER2 Negative Breast Cancer||Drug: ONC201||Phase 2|
National Cancer Institute (NCI) has indicated that access to an investigational treatment associated with this study is available outside the clinical trial.
Show Detailed Description
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||94 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 2 Study of ONC201 in Recurrent/Refractory Metastatic Breast Cancer and Advanced Endometrial Carcinoma|
|Actual Study Start Date :||January 17, 2018|
|Estimated Primary Completion Date :||August 1, 2022|
|Estimated Study Completion Date :||December 30, 2027|
Experimental: Arm 1
Single arm divided in three cohorts, each cohort with a different type of metastatic disease: ER + breast cancer, triple negative breast cancer, and endometrial cancer
625 mg by mouth every 7 days; each cycle = 28 days. Patients will receive ONC201 as long as they derive clinical benefit or toxicity becomes impeditive.
- Cohort 1 - Progression-free survival (PFS) [ Time Frame: at 8 months ]Cohort 1 HR+BC - To determine the progression free survival at 8 months (PFS by RECIST) of ONC201 in patients with refractory, metastic hormone receptor positive breast cancer
- Cohort 2 - Overall Response Rate (ORR) [ Time Frame: every 8 weeks ]To determine the overall response rate (ORR; CR + PR by RECIST) of ONC201 in patients with metastic triple negative breast cancer
- Cohort 3 - Overall Response Rate (ORR) [ Time Frame: every 8 weeks ]To determine the ORR of ONC201 in patients with advanced or metastatic endometrial cancer
- Cohorts 1,2, and 3 - tabulation of adverse event type and grade [ Time Frame: every 4 weeks ]tabulation of adverse event type and grade every 4-week cycle
- Cohort 1 - Overall Response Rate [ Time Frame: every 8 weeks ]To determine the overall response rate (ORR; CR + PR by RECIST) of ONC201 in patients with refractory, metastatic hormone receptor positive breast cancer
- Cohorts 1,2, and 3 - Clinical Benefit Rate [ Time Frame: every 8 weeks ]CR + PR + SD by RECIST
- Cohorts 2 and 3 - Progression-free Survival [ Time Frame: every 8 weeks ]PFS by RECIST
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): NCT03394027
|Contact: Nicole D. Houston, R.N.||(240) firstname.lastname@example.org|
|United States, Maryland|
|National Institutes of Health Clinical Center||Recruiting|
|Bethesda, Maryland, United States, 20892|
|Contact: For more information at the NIH Clinical Center contact National Cancer Institute Referral Office 888-624-1937|
|Principal Investigator:||Alexandra S Zimmer, M.D.||National Cancer Institute (NCI)|