Back to search results

Testing ONC201 for Colorectal Cancer Prevention

This study is testing the safety and cancer-preventing effects of a drug called ONC201 (also known as dordaviprone hydrochloride). It's for people who are at high risk for colorectal cancer, specifically those with Familial Adenomatous Polyposis (FAP) or a history of many polyps. Researchers want to see if ONC201 can stop cancer cells from growing by causing them to die (a process called apoptosis), without harming healthy cells. The study aims to find out if this drug could be a new way to prevent colorectal cancer. About 36 people are planned to join this study.

Study design
This is an interventional study, meaning participants will receive a specific treatment. It is designed to test the safety and effects of different doses of ONC201.
What's involved
You would undergo a biopsy, blood collection, and a colonoscopy. You would also take the drug ONC201 by mouth and complete questionnaires.
Compensation
Not stated in the trial record.
Follow-up
Your safety will be monitored for up to 35 days after your last dose of ONC201.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT05630794

Testing for Safety and Colorectal Cancer Preventive Effects of ONC201

Recruiting
PHASE1Ages 18+InterventionalPrevention
National Cancer Institute (NCI)
~36 participants
Updated 2026-09-11 on ClinicalTrials.gov
What's tested:Biopsy ProcedureBiospecimen CollectionColonoscopyDordaviprone HydrochlorideQuestionnaire AdministrationSigmoidoscopy

At a glance

Recruiting sites
3 of 5 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Proportion of participants with unacceptable toxicity
Measured over Up to 35 days post last dose of ONC201

Conditions

Colorectal Adenomatous Polyp
Colorectal Carcinoma
Familial Adenomatous Polyposis
Multiple Adenomatous Polyps

Explore related trials

Colorectal Adenomatous PolypFamilial Adenomatous Polyposis

NCT05630794

Where you'd take part

This study runs at 5 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Cleveland Clinic Foundation

    Cleveland, Ohiostudy coordinator listed

    Recruiting

  • Ohio State University Comprehensive Cancer Center

    Columbus, Ohiostudy coordinator listed

    Recruiting

  • Rhode Island Hospital

    Providence, Rhode Islandstudy coordinator listed

    Not yet recruiting

  • University of Michigan Rogel Cancer Center

    Ann Arbor, Michiganstudy coordinator listed

    Recruiting

  • Washington University School of Medicine

    St Louis, Missouristudy coordinator listed

    Not yet recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Alexander G Raufi, MD · PRINCIPAL_INVESTIGATOR · Brown University Health/ Rhode Island Hospital

Who to contact

Elena M. Stoffel
[email protected]
734-936-0781
Email the study team

Opens a ready-to-send draft in your own email app — review before sending.

Want this trial checked against your situation?

Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.

Check eligibility for this trial ~2 min · HIPAA-protected · delete anytime
Eligibility criteria

Inclusion

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
Total bilirubin within normal institutional limits
Aspartate aminotransferase (AST) (serum (glutamic-oxaloacetic transaminase \[SGOT\])/alanine aminotransferase (ALT) (serum glutamic pyruvic transaminase (\[SGPT\]) =\< 1.5 x institutional upper limit of normal
Creatinine =\< 1.5 x institutional upper limit of normal
Participant is due to undergo a standard of care lower gastrointestinal (GI) colonoscopy for detection and removal of colorectal polyps. On this colonoscopy, participant is required to have:
Two (2) adenomatous polyps of at least five (5) mm in size
At least one (1) polyp within reach of a flexible sigmoidoscope (which will be retained in the colon or rectum and marked)
In addition to polypectomy, six (6) biopsies of normal colonic mucosa \>= 1 cm from a collected polyp will also be collected
Willing to undergo a second, research intent endoscopic procedure (either sigmoidoscopy or colonoscopy), approximately 12 weeks after initiating ONC201 treatment
Willingness and ability to comply with scheduled visits, treatment plans, laboratory tests, and other study procedures
Life expectancy of at least 5-years
ONC201 is an imipridone agent with the potential for teratogenic or abortifacient effects. For this reason and because imipridones potential teratogenic effects are unknown, men and women of child-bearing potential must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation, and for four weeks after study treatment is completed. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should STOP the study medication and inform her study physician immediately
Ability to understand and the willingness to sign a written informed consent document

Exclusion

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
Concomitant use of strong/moderate CYP3A4/5 inducers/inhibitors. These agents must be discontinued at least 72-hours prior to beginning ONC201
Any of the following cardiac criteria:
Prolongation of corrected QT (QTc) interval (QTc interval \> 480 milliseconds, preferably using Frederica's QT correction formula), confirmed on electrocardiogram (ECG) tracings performed during screening
A history of Torsades de pointes, heart failure, or family history of prolonged QT Syndrome
Concomitant use of drugs that are known to prolong QT and have a known risk of torsade de pointes (TdP) unless they are willing to stop these medications and possibly change to an alternative non-excluded medication to treat the same condition at least 72 hours prior to beginning ONC201

What this trial measures

  • Proportion of participants with unacceptable toxicityUp to 35 days post last dose of ONC201

    Will be assessed using National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0.