Testing Obeticholic Acid for Familial Adenomatous Polyposis

Sponsor: National Cancer Institute (NCI)

Sponsor score: 0
Inactive

ClinicalTrials ID:NCT05223036

Provider View

This study is exploring whether the drug obeticholic acid (OCA) is safe and effective in reducing the number of polyps in the intestines and colon for patients with familial adenomatous polyposis (FAP), a genetic condition that increases cancer risk.

Patient Parameters

Program Overview

This phase IIa trial is investigating if obeticholic acid (OCA) can safely reduce the number of polyps in the small bowel and colon in patients with familial adenomatous polyposis (FAP). FAP is a rare genetic condition that raises the risk of developing cancer in the intestines and colon. OCA is similar to a bile acid produced by the liver and is believed to help prevent cancer. It is already approved for treating a liver disease called primary biliary cholangitis (PBC) at a lower dose. This study aims to find out if OCA can also reduce polyps in FAP patients.

Description

PRIMARY OBJECTIVE:

  • To see if OCA can reduce the number of polyps in the small intestine in people with FAP compared to a placebo.

SECONDARY OBJECTIVES:

  • To check the safety of OCA compared to a placebo in people with FAP.
  • To see if OCA can reduce the number of polyps in the rectum and rectal pouch compared to a placebo.
  • To measure the total number of polyps in the small intestine and rectum with OCA versus placebo.
  • To evaluate changes in certain blood markers and gene expressions in polyps and normal tissue with OCA versus placebo.
  • To study the effects of OCA on the diversity of gut bacteria in polyps and normal tissue.

STUDY DESIGN:

Participants are randomly assigned to one of two groups:

  • Group 1: Takes OCA 25 mg by mouth daily for 6 months, with regular check-ups and tests.
  • Group 2: Takes a placebo by mouth daily for 6 months, with regular check-ups and tests.

After finishing the treatment, participants will have follow-up visits within 14-21 days.

Eligibility Criteria

Inclusion Criteria

  • Must have a diagnosis of FAP with polyps in the small intestine and rectum.
    • Genetic diagnosis: APC gene mutation or family history of FAP.
    • Clinical diagnosis: More than 100 polyps in the large intestine and family history of FAP.
    • Clinical diagnosis: Had surgery for polyps, family history of FAP, and agreement by two experts.
    • Attenuated FAP: APC gene mutation required.
  • No active or returning cancer for 6 months before screening.
  • Age 18 years or older.
  • Good overall health with specific blood counts and organ function.
  • Negative tests for HIV, hepatitis B, and hepatitis C.
  • Willing to use contraception during and after the study.
  • Willing to limit alcohol intake.
  • Able to understand and sign a consent form.

Exclusion Criteria

  • Previous use of the study drug.
  • Polyps that cannot be measured.
  • High-grade dysplasia or cancer found during screening.
  • Active or suspected chronic liver disease.
  • Gallstones or bile duct issues.
  • History of pancreatitis or pancreatic problems.
  • Uncontrolled high cholesterol.
  • Severe or uncontrolled medical conditions.
  • Pregnant or breastfeeding women.
  • Allergies to the study drug.
  • Serious medical or psychiatric conditions that could interfere with the study.
  • Taking certain medications that cannot be stopped before the study.

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