Testing the Drug Obeticholic Acid for Familial Adenomatous Polyposis

About the Study

This study is looking at whether obeticholic acid (OCA) is safe and has a beneficial effect on the number of polyps in the small bowel and colon in people with familial adenomatous polyposis (FAP). OCA is a drug similar to a bile acid the body's liver makes. Researchers believe OCA may help keeping cancer from developing. OCA is already FDA approved to treat a type of liver disease. 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.

What the Study Involves

Participants will be divided into two groups:

• Group 1 will receive OCA for 6 months
• Group 2 will receive a placebo for 6 months

All participants will undergo GI endoscopy with biopsy and collection of blood samples at screening and on study.

After completion of the study treatment, patients are followed within 14-21 days.

Study Locations

Massachusetts 

Boston, MA
Dana-Farber Cancer Institute
Contact: Ramona M. Lim
617-582-7777 ColonScreening@DFCI.harvard.edu 

Michigan 

Ann Arbor, MI
University of Michigan Comprehensive Cancer Center
Contact: Elena M. Stoffel
734-615-9712 estoffel@med.umich.edu

Ohio 

Cleveland, OH
Cleveland Clinic Foundation
Contact: Carol A. Burke
216-444-7000 BURKEC1@ccf.org

Texas 

Houston, TX
MD Anderson Cancer Center
Contact: Eduardo Vilar-Sanchez
713-563-4743 EVilar@mdanderson.org

Puerto Rico

San Juan, PR
University of Puerto Rico
Contact: Marcia R. Cruz-Correa
787-758-2525 marcia.cruz1@upr.edu

This study is open to people over 18 years who: 

• have a diagnosis of FAP with disease involvement of the duodenum and rectum.
• APC mutation (with or without family history).
• FAP with more than 100 adenomas in large intestine and participant has a family history of FAP.
• Attenuated FAP (AFAP) with an APC mutation.
• participants must have no evidence of active or recurrent invasive cancer for 6 months prior to screening and must be at least 6 months from any prior cancer-directed treatment (such as surgery, chemotherapy, immunotherapy, hormonal therapy or radiation).
• presence of intact rectum or ileo-rectal anastomosis (IRA) or ileal pouch-anal anastomosis (IPAA) or end ileostomy.
• must have a negative test result for human immunodeficiency virus (HIV); if tested within the past 6 months, result will be accepted without repeating the test.
• must have negative test for hepatitis C virus (HCV) and B virus (HBV).
• must be willing not to get pregnant and use birth control.

See clinicaltrials.gov for the full list of inclusion criteria. 

People with the following are not eligible:

• prior use of study drug.
• not enough polyps in the duodenal or rectal/pouch to measure.
• high-grade dysplasia or cancer on biopsy at screening.
• chronic liver disease including cirrhosis, nonalcoholic steatohepatitis (NASH) with liver fibrosis, NASH with cirrhosis, primary sclerosing cholangitis, biliary atresia.
• history of gallbladder, liver, kidney blood or heart disease.
• people with a history of pancreatitis or presence of pancreatic abnormalities suggestive of increased risk of pancreatitis.
• pregnant, breast-feeding, or women of childbearing potential unwilling to use a reliable contraceptive method. 
• any serious and/or unstable pre-existing medical disorder (aside from malignancy exception above), psychiatric disorder, or other conditions that could interfere with participant's safety, obtaining informed consent, or compliance to the study procedures.
• the use of other research agents or certain drugs or drug classes is prohibited while on the study. 

See clinicaltrials.gov for the full list of exclusion criteria.