SuspendedPhase 2

Testing Obeticholic Acid for Familial Adenomatous Polyposis

Stopped: FDA Partial Clinical Hold

United States80 participantsStarted 2023-01-19

Plain-language 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.

Who can participate

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Participants must have a diagnosis of phenotypic FAP with disease involvement of the duodenum and rectum as defined by: * Genetic diagnosis: APC germline mutation (with or without family history) or obligate carrier * Clinical diagnosis: FAP phenotype with \> 100 adenomas in large intestine and participant has a family history of FAP * Clinical diagnosis: FAP phenotype who are status post colectomy for polyposis, participant has a family history of FAP, and 2 FAP experts agree to the diagnosis * Attenuated FAP diagnosis: APC germline mutation required * 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 surgical resection, chemotherapy, immunotherapy, hormonal therapy or radiation) * Age \>= 18 years. Because no dosing or adverse event (AE) data are currently available on the use of OCA in participants \< 18 years of age, children are excluded from this study but will be eligible for future pediatric trials, if applicable * Eastern Cooperative Oncology Group (ECOG) performance status =\< 1 (Karnofsky \>= 70%) * Hemoglobin \>= 10 g/dL or hematocrit \>= 30% * Leukocyte count \>= 3,500/microliter * Platelet count \>= 100,000/microliter * Absolute neutrophil count \>= 1,500/microliter * Creatinine clearance (calculated if measured is not available) \>= 30 mL/min/1.73m\^2 * Aspartate aminotransferase (AST) (serum glu…

What they're measuring

Percentage change in duodenal polyp burden

Timeframe: Baseline to 6 months

Trial details

NCT IDNCT05223036

SponsorNational Cancer Institute (NCI)

Sponsor typeNIH

Study typeINTERVENTIONAL

Primary completion2029-01-31