Testing Obeticholic Acid for Familial Adenomatous Polyposis

Summary

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.

Conditions

• Attenuated Familial Adenomatous Polyposis
• Colorectal Carcinoma
• Duodenal Carcinoma
• Familial Adenomatous Polyposis

Interventions

PROCEDURE

Biopsy Procedure

Undergo biopsy

PROCEDURE

Biospecimen Collection

Undergo collection of blood samples

PROCEDURE

Gastrointestinal Endoscopy

Undergo GI endoscopy

BIOLOGICAL

Obeticholic Acid

Given PO

DRUG

Placebo Administration

Given PO

OTHER

Questionnaire Administration

Ancillary studies

Sponsors & Collaborators

• National Cancer Institute (NCI)

  lead NIH

Principal Investigators

• Eduardo Vilar-Sanchez · M.D. Anderson Cancer Center

Study Design

Allocation

RANDOMIZED

Purpose

PREVENTION

Masking

DOUBLE

Model

PARALLEL

Eligibility

Min Age

18 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2023-01-19

Primary Completion

2026-03-17

Completion

2026-04-17

FDA Drug

Yes

Countries

• United States
• Puerto Rico

Study Locations