Testing Obeticholic Acid for Familial Adenomatous Polyposis

Major Program
Cancer Prevention Clinical Trials Network
Research Group
Gastrointestinal and Other Cancers
Sponsor
National Cancer Institute (NCI)
Status
Recruiting
ClinicalTrials.gov ID
For more information, see ClinicalTrials.gov NCT05223036
This phase IIa trial investigates if giving obeticholic acid (OCA) is safe and has a beneficial effect on the number of polyps in the small bowel and colon in patients with familial adenomatous polyposis (FAP). FAP is a rare gene defect that increases the risk of developing cancer of the intestines and colon. OCA is a drug similar to a bile acid the body makes. It is fluid made and released by the liver. OCA binds to a receptor in the intestine that is believed to have a positive effect on preventing cancer development. OCA has been effective in treating primary biliary cholangitis (PBC), a liver disease, and is approved by the Food and Drug Administration (FDA) for use at a lower dose (10 mg). There have been studies showing that OCA decreases inflammation and fibrosis. However, it is not yet known whether OCA works on reducing the number of polyps in patients with FAP.
Intervention
Biopsy, Biospecimen Collection, Gastrointestinal Endoscopy, Obeticholic Acid, Placebo Administration, Questionnaire Administration
Condition
Attenuated Familial Adenomatous Polyposis, Colorectal Carcinoma, Duodenal Carcinoma, Familial Adenomatous Polyposis
Investigators
Eduardo Vilar-Sanchez, Niloy J. Samadder, Ramona M. Lim, Elena M. Stoffel, Carol A. Burke, Marcia R. Cruz-Correa

See list of participating sites