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Bile Acid Analog

Obeticholic Acid for Familial Adenomatous Polyposis

Phase 2

Recruiting

Led By Eduardo Vilar-Sanchez

Research Sponsored by National Cancer Institute (NCI)

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Participants must have no evidence of active or recurrent invasive cancer for 6 months prior to screening

Presence of Spigelman stage II or III duodenal polyposis at screening

Must not have

Uncontrolled hyperlipidemia

Uncontrolled intercurrent illnesses

Timeline

Screening 3 weeks

Treatment Varies

Follow Up at 6 months

Summary

This trial is investigating if a drug similar to a bile acid can help prevent the development of cancer in patients with a gene defect that increases their risk.

Who is the study for?

Adults with familial adenomatous polyposis (FAP), a condition causing many polyps in the intestine, can join this trial. They must have an APC gene mutation or clinical diagnosis of FAP, be in good general health (ECOG <=1), and not have had cancer treatment for at least 6 months. Participants need functioning major organs as indicated by specific blood tests and agree to use contraception.

What is being tested?

The trial is testing Obeticholic Acid (OCA) to see if it's safe and effective at reducing intestinal polyps in FAP patients. OCA mimics a bile acid made by the liver that may help prevent cancer growth. Patients will either receive OCA or a placebo while undergoing endoscopies, biopsies, and questionnaires to monitor effects.

What are the potential side effects?

Possible side effects of OCA include itching, fatigue, abdominal pain, rash, digestive issues like constipation or diarrhea, joint pain and swelling. There might also be more serious risks such as liver problems which would be closely monitored during the trial.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I haven't had signs of cancer coming back for at least 6 months.

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My duodenal polyps are classified as stage II or III.

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I have FAP affecting my duodenum and rectum, confirmed by genetic or clinical diagnosis.

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I am fully active or can carry out light work.

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My liver function tests are normal.

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I am 18 years old or older.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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My cholesterol levels are very high and not under control.

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I do not have any uncontrolled illnesses.

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I have had pancreatitis or issues with my pancreas.

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I have an active hepatitis C or B infection that hasn't been treated.

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I have a liver condition such as cirrhosis, NASH, or a biliary disorder.

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I have previously used the drug being studied.

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My biopsy shows high-grade dysplasia or cancer.

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I have too many polyps in my duodenum or rectum to count.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ at 6 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at 6 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and at 6 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Percentage change in duodenal polyp burden

Secondary study objectives

Absolute differences in the levels of serum FGF19 and C4

Changes in gene expression (messenger ribonucleic acid) profiles of adenomas and normal mucosa

Changes in the microbiome of adenomas and normal mucosa

+5 more

Side effects data

From 2022 Phase 2 trial • 10 Patients • NCT02430077

40%

Itching

100%

80%

60%

40%

20%

Study treatment Arm

Placebo for Obeticholic Acid

Active Capsule of Obeticholic Acid

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Arm I (OCA)Experimental Treatment5 Interventions

Patients receive OCA PO QD for 6 months in the absence of unacceptable toxicity. Patients also undergo GI endoscopy with biopsy and collection of blood samples at screening and on study.

Group II: Arm II (placebo)Placebo Group5 Interventions

Patients receive placebo PO QD for 6 months in the absence of unacceptable toxicity. Patients also undergo GI endoscopy with biopsy and collection of blood samples at screening and on study.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Biopsy

2014

Completed Phase 4

~1150

Biospecimen Collection

2004

Completed Phase 3

~2030