ONC201 for Colorectal Cancer Prevention

Recruiting at 4 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: National Cancer Institute (NCI)

Must not be taking: Investigational agents

Disqualifiers: Lynch syndrome, Invasive cancer, Uncontrolled illness, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 12 weeks

24 Participants Needed

Trial Summary

What is the purpose of this trial?

This phase I trial tests the safety, side effects, and best dose of Akt/ERK Inhibitor ONC201 (ONC201) in preventing colorectal cancer in patients with familial adenomatous polyposis (FAP) or a history of multiple polyps. ONC201 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Do I need to stop my current medications to join the trial?

The trial information does not specify if you need to stop taking your current medications. However, you cannot be on any other investigational agents or have taken them in the past four weeks.

How is the drug ONC201 unique for preventing colorectal cancer?

ONC201 is unique because it represents a novel approach to colorectal cancer prevention, potentially offering a new mechanism of action compared to traditional chemoprevention agents like aspirin or statins, which target inflammation and cholesterol pathways. While existing treatments focus on reducing inflammation or cholesterol, ONC201 may work through different biological pathways, although specific details about its mechanism in colorectal cancer prevention are not yet well-documented. ^{1 2 3 4 5}

Research Team

Alexander G Raufi

Principal Investigator

Rhode Island Hospital

Eligibility Criteria

This trial is for adults at high risk of colorectal adenomas, specifically those with familial adenomatous polyposis (FAP) or a history of multiple polyps. Participants must be in good health with an ECOG performance status <=1 and have normal organ function. They should not have had invasive cancer treatments within the last 3 years, except for certain skin cancers or cervical cancer in situ.

Inclusion Criteria

I am willing and able to follow the study's schedule and procedures.

I am at high risk for colorectal adenomas due to FAP or having more than 5 small adenomas or at least 3 with one being larger than 10mm.

I am 18 years old or older.

Exclusion Criteria

I have a history of Lynch syndrome.

I am not pregnant or nursing, as the treatment may harm the baby.

I am not currently on any experimental drugs and haven't been for the last 4 weeks.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive ONC201 orally once weekly or once every 3 weeks for 12 weeks. Blood, tissue biopsy, and sigmoidoscopy/colonoscopy are conducted throughout the study.

12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• ONC201

Trial Overview The trial is testing ONC201's safety and optimal dosage to prevent colorectal cancer in patients prone to developing polyps. It involves procedures like colonoscopy, biopsy collection, and questionnaires to monitor effects on tumor cell growth inhibition by targeting specific enzymes.

Participant Groups

Group I: Prevention (ONC201, biopsy, sigmoidoscopy, colonoscopy) Experimental Treatment 6 Interventions

Patients receive ONC201 PO QW or Q3W for 12 weeks. Patients also undergo collection of blood, tissue biopsy, and sigmoidoscopy/colonoscopy throughout the study.

Who Is Running the Clinical Trial?

National Cancer Institute (NCI)

Lead Sponsor

Trials

14,080

Recruited

41,180,000+

References

1. United States pubmed.ncbi.nlm.nih.gov

Chemoprevention of Colorectal Cancer. [2021]

2. Russia (Federation) pubmed.ncbi.nlm.nih.gov

CANCERPREVENTIVE IN ULCERATIVE COLITIS. [2018]

3. England pubmed.ncbi.nlm.nih.gov

Cancer prevention in inflammatory bowel disease and the chemoprophylactic potential of 5-aminosalicylic acid. [2019]

4. England pubmed.ncbi.nlm.nih.gov

Chemoprevention of colorectal cancer: Past, present, and future. [2021]

5. United Arab Emirates pubmed.ncbi.nlm.nih.gov

The potential of statins for individualized colorectal cancer chemoprevention. [2019]