Actively Recruiting
Testing for Safety and Colorectal Cancer Preventive Effects of ONC201
Led by National Cancer Institute (NCI) · Updated on 2026-05-13
36
Participants Needed
5
Research Sites
125 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The purpose of this phase I trial is to test the safety and cancer preventive effects of different doses of ONC201 in people with familial adenomatous polyposis (FAP) or a history of multiple polyps. People with familial adenomatous polyposis (FAP) or a history of multiple polyps are at higher than average risk of developing colorectal cancer. ONC201, now known as dordaviprone, is a drug that may stop cancer cells from growing. This drug has been shown in previous studies to cause cancer cell death but not harm normal cells. If successful, this study may help us develop a new option for colorectal cancer prevention.
CONDITIONS
Official Title
Testing for Safety and Colorectal Cancer Preventive Effects of ONC201
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Be identified as high risk for recurrent colorectal adenomas by having a diagnosis of familial adenomatous polyposis (FAP) and/or findings of either more than 5 small adenomas (<1 cm) or at least 3 adenomas with one ≥10 mm on a recent colonoscopy within 5 years
- Be 18 years of age or older at the time of signing informed consent
- Have an ECOG performance status of 0 or 1 (Karnofsky ≥ 70%)
- Have leukocytes count ≥ 3,000/microliter
- Have absolute neutrophil count ≥ 1,000/microliter
- Have platelet count ≥ 100,000/microliter
- Have total bilirubin within normal institutional limits
- Have AST (SGOT)/ALT (SGPT) levels ≤ 1.5 times the institutional upper limit of normal
- Have creatinine ≤ 1.5 times the institutional upper limit of normal
- Be scheduled for a standard lower gastrointestinal colonoscopy for detection and removal of colorectal polyps, with at least two adenomatous polyps of at least 5 mm size
- Have at least one polyp within reach of a flexible sigmoidoscope that will be retained and marked
- Agree to have six biopsies of normal colonic mucosa taken at least 1 cm from a collected polyp
- Willing to undergo a second research endoscopic procedure (sigmoidoscopy or colonoscopy) about 12 weeks after starting ONC201
- Willing and able to comply with all study visits, treatments, lab tests, and procedures
- Have a life expectancy of at least 5 years
- Men and women of childbearing potential must agree to use effective contraception during the study and for 4 weeks after treatment ends
- Ability to understand and sign informed consent
You will not qualify if you...
- Prior history of hereditary nonpolyposis colorectal cancer (HNPCC) or Lynch syndrome
- Currently receiving or have received any other investigational agents within 4 weeks prior to study
- Prior history of invasive colorectal cancer
- Prior invasive active cancer progressing or requiring treatment within 3 years, except certain treated skin or cervical cancers
- Prior exposure to cytotoxic chemotherapy or ONC201
- History of allergic reaction to compounds similar to ONC201
- Uncontrolled illness including active infection, symptomatic heart failure, unstable angina, arrhythmia, or psychiatric/social conditions limiting compliance
- Pregnant or nursing women
- Use of strong or moderate CYP3A4/5 inducers or inhibitors within 72 hours before starting ONC201
- Cardiac conditions including prolonged QTc interval >480 ms, history of Torsades de pointes, heart failure, or family history of prolonged QT syndrome
- Use of drugs known to prolong QT interval unless willing to stop or substitute at least 72 hours before starting ONC201
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 5 locations
1
University of Michigan Rogel Cancer Center
Ann Arbor, Michigan, United States, 48109
Actively Recruiting
2
Washington University School of Medicine
St Louis, Missouri, United States, 63110
Actively Recruiting
3
Cleveland Clinic Foundation
Cleveland, Ohio, United States, 44195
Actively Recruiting
4
Ohio State University Comprehensive Cancer Center
Columbus, Ohio, United States, 43210
Actively Recruiting
5
Rhode Island Hospital
Providence, Rhode Island, United States, 02903
Not Yet Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
PREVENTION
Number of Arms
1
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