Testing for Safety and Colorectal Cancer Preventive Effects of ONC201
Eligible age
18+ yrs
Accepts
All genders
Locations
4 states
Healthy volunteers
No
See if you qualify for this study
Answer a few quick questions about your location and health. Takes about a minute.
About this study
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.
Sponsor: National Cancer Institute (NCI)
You may qualify if…
- ✓ Be identified as high risk for recurrent colorectal adenomas, as defined by:
- ✓ A diagnosis of FAP AND/OR
- ✓ Findings of either \> 5 small (less than 1 cm) adenomas OR \>= 3 with at least one \>= 10 mm on most recent colonoscopy performed in the past 5 years
- ✓ Be \>= 18 years of age on day of signing informed consent
- ✓ Have an Eastern Cooperative Oncology Group (ECOG) performance status =\< 1 (Karnofsky \>= 70%)
- ✓ Leukocytes \>= 3,000/microliter
- ✓ Absolute neutrophil count \>= 1,000/microliter
- ✓ Platelets \>= 100,000/microliter
You may not qualify if…
- ✕ Prior history of hereditary nonpolyposis colorectal cancer (HNPCC), also known as Lynch syndrome
- ✕ Participants may not be currently receiving any other investigational agents or have received any investigational agents within the past four weeks
- ✕ Prior history of invasive colorectal cancer
- ✕ Prior invasive active neoplasm that is progressing or requires active treatment within 3 years from registration. Exceptions include basal cell carcinoma of the skin or squamous cell carcinoma of the skin that has undergone potentially curative therapy or in situ cervical cancer. Participants with a history of prior invasive neoplasm diagnosed and treated greater than 3 years form registration may be considered with consultation of the primary investigator
- ✕ Prior history of exposure to cytotoxic chemotherapy or ONC201
- ✕ History of allergic reactions attributed to compounds of similar chemical or biologic composition to ONC201
- ✕ Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
- ✕ Pregnant and women who are nursing are excluded from this study because ONC201 is an imipridone agent with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events (AEs) in nursing infants secondary to treatment of the mother with ONC201, breastfeeding should be discontinued if the mother is treated with ONC201
Where it's recruiting
Source: ClinicalTrials.gov · NCT05630794 · last updated 2026-06-12