Attenuated Familial Adenomatous PolyposisPhase 2NCT05223036
Testing Obeticholic Acid for Familial Adenomatous Polyposis
A Phase 2 study testing Biopsy Procedure, Biospecimen Collection and Gastrointestinal Endoscopy in people with Attenuated Familial Adenomatous Polyposis and Colorectal Carcinoma, run by National Cancer Institute (NCI) at 7 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Attenuated Familial Adenomatous Polyposis, Colorectal Carcinoma, Duodenal Carcinoma
- What is being tested?
- Biopsy Procedure, Biospecimen Collection and Gastrointestinal Endoscopy
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 15 participants
- Where
- 7 sitesin 2 countries
- Study dates
- 19 Jan 2023to 17 Apr 2026
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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)
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- 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 glutamic-oxaloacetic transaminase [SGOT])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) =< 1.5 x the institutional upper limit of normal (ULN)
- Total bilirubin =< 1.0 x ULN
- Alkaline phosphatase =< 1.5 x ULN
- Gamma-glutamyl transferase (GGT) =< 1.5 x ULN
- Presence of Spigelman stage II or III duodenal polyposis at screening
- Presence of intact rectum or ileo-rectal anastomosis (IRA) or ileal pouch-anal anastomosis (IPAA) or end ileostomy
- Participants 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
- Participants must have negative test for hepatitis C virus (HCV) and B virus (HBV)
- Willing and able to adhere to the prohibitions and restrictions specified in the final approved protocol
- The effects of OCA on the developing human fetus at the recommended therapeutic dose are unknown. For this reason, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, throughout the duration of study participation, and for at least 6 months after receiving the last dose of study drug. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her study physician immediately
- Willingness to moderate alcohol intake (consuming no more than 1 or 2 alcoholic drinks per day for women and men, respectively)
- Ability to understand and the willingness to sign a written informed consent document
Who cannot join
- Prior use of study drug
- Duodenal or rectal/pouch polyp burden that is not quantifiable
- Histologically-confirmed high-grade dysplasia or cancer on biopsy at screening
- Individuals with active, known or suspected chronic liver disease including cirrhosis, nonalcoholic steatohepatitis (NASH) with liver fibrosis, NASH with cirrhosis, primary sclerosing cholangitis, biliary atresia
- Individuals with acute cholecystitis (defined by a syndrome of right upper quadrant pain, fever, and leukocytosis associated with gallbladder inflammation)
- Individuals with a history of pancreatitis or presence of pancreatic abnormalities suggestive of increased risk of pancreatitis
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- Individuals with hepatic steatosis and velocity > 1.7 meters/second (m/s) as determined by liver ultrasound elastography
- Individuals with hyperlipidemia not well controlled with the use of pharmacotherapy and/or dietary modifications
- History of severe, progressive, or uncontrolled renal, genitourinary, hepatic, hematologic, endocrine, cardiac, vascular, pulmonary, rheumatologic, neurologic, psychiatric, or metabolic disturbances, or signs and symptoms thereof
- Pregnant, breast-feeding, or women of childbearing potential unwilling to use a reliable contraceptive method. Pregnant women are excluded from this study because OCA is an agent with unknown effects on the developing human fetus. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with OCA, breastfeeding should be discontinued if the mother is treated with OCA
- Known hypersensitivity, allergies, or intolerance to the study drug or compounds of similar chemical or biologic composition
- 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
- Participants may not be receiving any other investigational agents
- Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
- Individuals with active and untreated hepatitis C virus (HCV) and/or or hepatitis B virus (HBV) infection
- Individuals with HIV infection are eligible for participation if:
- CD4+ count >= 300/uL
- Viral load is undetectable
- Receiving highly active antiretroviral therapy (HAART) without known or suspected drug interactions with OCA
- Consultation with the participant's infectious disease specialist may be obtained
- Individuals taking the drugs listed below may not be randomized unless they are willing to stop the medications (and possibly change to alternative non-excluded medications to treat the same conditions) no less than 5 half-lives days prior to starting OCA or placebo on this study. Consultation with the participant's primary care provider may be obtained but is not required. The use of the following drugs or drug classes is prohibited during OCA/placebo treatment:
- Investigational agents
- Bile acid sequestrants (bile acid binding resins): cholestyramine, colestipol, or colesevelam
- Bile salt efflux pump (BSEP) inhibitors
- Clozapine
- Theophylline derivatives
- Tizanidine
- Warfarin
- Hepatotoxic drugs such as amiodarone, sodium valproate, certain herbal/dietary supplements, and long-term doxycycline or tetracycline
Trial locations and contacts
7 sites in 2 countries.
7 sites in 2 countries
United States6 sites
University of Michigan Rogel Cancer Center
Ann Arbor, Michigan, 48109
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
Cleveland Clinic Foundation
Cleveland, Ohio, 44195
M D Anderson Cancer Center
Houston, Texas, 77030
University of Kansas Cancer Center
Kansas City, Kansas, 66160
Mayo Clinic in Arizona
Scottsdale, Arizona, 85259
About this study
Official title: A Phase IIa, Placebo-Controlled, Randomized Study of Daily Obeticholic Acid (OCA) to Reduce Intestinal Polyp Burden in Familial Adenomatous Polyposis (FAP)
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.
Read the detailed description
PRIMARY OBJECTIVE:
- To evaluate the effect of treatment with OCA versus treatment with placebo on duodenal polyp burden (sum of polyp diameters) in participants with FAP.
SECONDARY OBJECTIVES:
- To assess the safety profile of treatment with OCA versus placebo in participants with FAP.
II. To evaluate the effect of treatment with OCA versus placebo on rectal and pouch polyp burden (sum of polyp diameters) in participants with FAP.
III. To assess the effect of treatment with OCA versus placebo on polyp burden (absolute number) in the duodenum of participants with FAP.
IV. To assess the effect of treatment with OCA versus placebo on polyp burden (absolute number) in the rectum and rectal pouch of participants with FAP.
- To evaluate the effect of treatment with OCA versus placebo on serum levels of fibroblast growth factor-19 (FGF19) and 7 alpha-hydroxy-4-cholesten-3-one (7AC4, also known as C4) in participants with FAP.
VI. To determine the effects of treatment with OCA versus placebo on gene expression in duodenal, rectal pouch, and rectal adenomas and uninvolved mucosa in participants with FAP:
VIa. Identify differentially expressed genes between duodenal and colorectal adenomas and uninvolved tissue at baseline and post-intervention for participants who received OCA or placebo; VIb. Quantify the effect of OCA on the expression of downstream targets of FXR in adenomas and uninvolved tissue; VIc. Quantify the effect of OCA on the expression of cancer stem cell markers (e.g. LGR5, ASCL2, LRIG, BMI) and intestinal stem cell markers (e.g. Villin, KRT20, MUC, LYZ) in adenomas and uninvolved tissue.
VII. To evaluate the cell-type specific effects of treatment with OCA versus placebo on gene expression and abundance in duodenal, rectal pouch, and rectal adenomas and uninvolved mucosa in participants with FAP via single-cell transcriptomics.
VIII. To evaluate the effect of treatment with OCA versus placebo on microbiome diversity in duodenal, rectal pouch, and rectal adenomas and uninvolved tissue in participants with FAP.
VIIIa. Compare alpha- and beta-diversity analysis and identify differential abundance in adenomas and uninvolved tissue.
OUTLINE: Patients are randomized to 1 of 2 arms.
ARM I: Patients receive OCA 25 mg orally (PO) once daily (QD) for 6 months in the absence of unacceptable toxicity. Patients also undergo gastrointestinal (GI) endoscopy with biopsy and collection of blood samples at screening and on study.
ARM II: Patients receive matching placebo PO QD for 6 months in the absence of unacceptable toxicity. Patients also undergo GI endoscopy biopsy and collection of blood samples at screening and on study.
After completion of the study treatment, patients are followed within 14-21 days.
- Conditions
- Attenuated Familial Adenomatous Polyposis, Colorectal Carcinoma, Duodenal Carcinoma, Familial Adenomatous Polyposis
- Being tested
- Biopsy Procedure, Biospecimen Collection, Gastrointestinal Endoscopy, Obeticholic Acid, Placebo Administration
- Medical subject headings
- Attenuated familial adenomatous polyposis, Colorectal Neoplasms, Duodenal Neoplasms, Adenomatous Polyposis Coli
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Biopsy ProcedureProcedure or surgery
Also called Biopsy, BIOPSY_TYPE, Bx
Undergo biopsy
Biospecimen CollectionProcedure or surgery
Also called Biological Sample Collection, Biospecimen Collected, Sample Collection, Specimen Collection
Undergo collection of blood samples
Gastrointestinal EndoscopyProcedure or surgery
Also called Enteroscopy
Undergo GI endoscopy
Obeticholic AcidBiological
Also called INT-747, Ocaliva
Given PO
Placebo AdministrationDrug
Given PO
Questionnaire AdministrationOther
Ancillary studies
| Study group | Role | What they receive |
|---|---|---|
| Arm I (OCA) | Experimental | 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. Receives: Procedure: Biopsy Procedure; Procedure: Biospecimen Collection; Procedure: Gastrointestinal Endoscopy; Biological: Obeticholic Acid; Other: Questionnaire Administration |
| Arm II (placebo) | Placebo comparator | 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. Receives: Procedure: Biopsy Procedure; Procedure: Biospecimen Collection; Procedure: Gastrointestinal Endoscopy; Drug: Placebo Administration; Other: Questionnaire Administration |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage change in duodenal polyp burden
Change from baseline will be summarized by treatment arm. The difference in change from baseline between active treatment arms and the placebo arm will also be summarized.
Measured: Baseline to 6 months
Secondary outcomes (8)
Incidence of adverse events
Will summarize adverse events for each group by grade and study drug attribution using descriptive statistics.
Measured: Up to 21 days after completion of treatment
Absolute differences in the levels of serum FGF19 and C4
Differences in the median serum levels of FGF19 and C4 between treatment arms will be compared.
Measured: Baseline to 6 months
Percentage change in the number of duodenal polyps
Will use parametric models to evaluate the absolute and/or relative changes from baseline of these measures between obeticholic acid (OCA) and placebo groups.
Measured: Baseline to 6 months
Percentage change in the number of colorectal, rectal/pouch polyps
Will use parametric models to evaluate the absolute and/or relative changes from baseline of these measures between OCA and placebo groups.
Measured: Baseline to 6 months
Percentage change in colorectal, rectal/pouch polyp burden
Will use parametric models to evaluate the absolute and/or relative changes from baseline of these measures between OCA and placebo groups.
Measured: Baseline to 6 months
Changes in gene expression (messenger ribonucleic acid) profiles of adenomas and normal mucosa
Will apply methods such as the beta-uniform mixture (BUM) model, the Wilcoxon rank-sum test with empirical Bayes, and the significance analysis of microarrays (SAM) to control the first degree relative (FDR).
Measured: Baseline to 6 months
Changes in the microbiome of adenomas and normal mucosa
Will apply methods such as the BUM model, the Wilcoxon rank-sum test with empirical Bayes, and the SAM to control the FDR.
Measured: Baseline to 6 months
Future candidate biomarkers measured by genomic and transcriptomic platforms in residual tissue biopsies of adenomas and normal mucosa
Will apply methods such as the BUM model, the Wilcoxon rank-sum test with empirical Bayes, and the SAM to control the FDR.
Measured: At 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Feb 2022
- Study start19 Jan 2023
- Primary completion17 Mar 2026
- Study completion17 Apr 2026
Why it stopped: Protocol has been completed prematurely due to poor accrual and study feasibility.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Attenuated Familial Adenomatous PolyposisStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Eduardo Vilar-Sanchez, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05223036
- Sponsor's protocol ID
- NCI-2022-00341
- Other IDs
- NCI-2022-00341, 2021-0286, 2021-0286, MDA20-01-01, P30CA016672, UG1CA242609
- Status last verified
- May 2026
- First submitted
- 1 Feb 2022
- Last updated
- 14 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: NCI is committed to sharing data in accordance with NIH policy. For more details on how clinical trial data is shared, access the link to the NIH data sharing policy page.
Study at a glance
From the registry record, last updated by the sponsor on 14 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase IIa, Placebo-Controlled, Randomized Study of Daily Obeticholic Acid (OCA) to Reduce Intestinal Polyp Burden in Familial Adenomatous Polyposis (FAP)
- ClinicalTrials.gov ID
- NCT05223036
- Acronym / sponsor ID
- NCI-2022-00341
- Conditions
- Attenuated Familial Adenomatous Polyposis, Colorectal Carcinoma, Duodenal Carcinoma, Familial Adenomatous Polyposis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Protocol has been completed prematurely due to poor accrual and study feasibility.
- Enrolment
- 15 participants (actual)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- 19 Jan 2023 (actual)
- Primary completion
- 17 Mar 2026 (actual)
- Study completion
- 17 Apr 2026 (actual)
- First posted
- 3 Feb 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
PRIMARY OBJECTIVE:
- To evaluate the effect of treatment with OCA versus treatment with placebo on duodenal polyp burden (sum of polyp diameters) in participants with FAP.
SECONDARY OBJECTIVES:
- To assess the safety profile of treatment with OCA versus placebo in participants with FAP.
II. To evaluate the effect of treatment with OCA versus placebo on rectal and pouch polyp burden (sum of polyp diameters) in participants with FAP.
III. To assess the effect of treatment with OCA versus placebo on polyp burden (absolute number) in the duodenum of participants with FAP.
IV. To assess the effect of treatment with OCA versus placebo on polyp burden (absolute number) in the rectum and rectal pouch of participants with FAP.
- To evaluate the effect of treatment with OCA versus placebo on serum levels of fibroblast growth factor-19 (FGF19) and 7 alpha-hydroxy-4-cholesten-3-one (7AC4, also known as C4) in participants with FAP.
VI. To determine the effects of treatment with OCA versus placebo on gene expression in duodenal, rectal pouch, and rectal adenomas and uninvolved mucosa in participants with FAP:
VIa. Identify differentially expressed genes between duodenal and colorectal adenomas and uninvolved tissue at baseline and post-intervention for participants who received OCA or placebo; VIb. Quantify the effect of OCA on the expression of downstream targets of FXR in adenomas and uninvolved tissue; VIc. Quantify the effect of OCA on the expression of cancer stem cell markers (e.g. LGR5, ASCL2, LRIG, BMI) and intestinal stem cell markers (e.g. Villin, KRT20, MUC, LYZ) in adenomas and uninvolved tissue.
VII. To evaluate the cell-type specific effects of treatment with OCA versus placebo on gene expression and abundance in duodenal, rectal pouch, and rectal adenomas and uninvolved mucosa in participants with FAP via single-cell transcriptomics.
VIII. To evaluate the effect of treatment with OCA versus placebo on microbiome diversity in duodenal, rectal pouch, and rectal adenomas and uninvolved tissue in participants with FAP.
VIIIa. Compare alpha- and beta-diversity analysis and identify differential abundance in adenomas and uninvolved tissue.
OUTLINE: Patients are randomized to 1 of 2 arms.
ARM I: Patients receive OCA 25 mg orally (PO) once daily (QD) for 6 months in the absence of unacceptable toxicity. Patients also undergo gastrointestinal (GI) endoscopy with biopsy and collection of blood samples at screening and on study.
ARM II: Patients receive matching placebo PO QD for 6 months in the absence of unacceptable toxicity. Patients also undergo GI endoscopy 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 design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm I (OCA) | Experimental | Procedure: Biopsy Procedure, Procedure: Biospecimen Collection, Procedure: Gastrointestinal Endoscopy, Biological: Obeticholic Acid, Other: Questionnaire AdministrationPatients 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. |
| Arm II (placebo) | Placebo comparator | Procedure: Biopsy Procedure, Procedure: Biospecimen Collection, Procedure: Gastrointestinal Endoscopy, Drug: Placebo Administration, Other: Questionnaire AdministrationPatients 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (25 listed)
- Participants must have a diagnosis of phenotypic FAP with disease involvement of the duodenum and rectum as defined by:
- 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%
Key exclusion criteria (29 listed)
- Prior use of study drug
- Duodenal or rectal/pouch polyp burden that is not quantifiable
- Histologically-confirmed high-grade dysplasia or cancer on biopsy at screening
- Individuals with active, known or suspected chronic liver disease including cirrhosis, nonalcoholic steatohepatitis (NASH) with liver fibrosis, NASH with cirrhosis, primary sclerosing cholangitis, biliary atresia
- Individuals with acute cholecystitis (defined by a syndrome of right upper quadrant pain, fever, and leukocytosis associated with gallbladder inflammation)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Biopsy ProcedureAlso: Biopsy, BIOPSY_TYPE, BxUndergo biopsy | Procedure or surgery | Arm I (OCA), Arm II (placebo) |
| Biospecimen CollectionAlso: Biological Sample Collection, Biospecimen Collected, Sample Collection, Specimen CollectionUndergo collection of blood samples | Procedure or surgery | Arm I (OCA), Arm II (placebo) |
| Gastrointestinal EndoscopyAlso: EnteroscopyUndergo GI endoscopy | Procedure or surgery | Arm I (OCA), Arm II (placebo) |
| Obeticholic AcidAlso: INT-747, OcalivaGiven PO | Biological | Arm I (OCA) |
| Placebo AdministrationGiven PO | Drug | Arm II (placebo) |
| Questionnaire AdministrationAncillary studies | Other | Arm I (OCA), Arm II (placebo) |
Primary outcome
Percentage change in duodenal polyp burden
Time frame: Baseline to 6 months
Change from baseline will be summarized by treatment arm. The difference in change from baseline between active treatment arms and the placebo arm will also be summarized.
8 secondary outcomes
Incidence of adverse events
Time frame: Up to 21 days after completion of treatment
Absolute differences in the levels of serum FGF19 and C4
Time frame: Baseline to 6 months
Percentage change in the number of duodenal polyps
Time frame: Baseline to 6 months
Percentage change in the number of colorectal, rectal/pouch polyps
Time frame: Baseline to 6 months
Percentage change in colorectal, rectal/pouch polyp burden
Time frame: Baseline to 6 months
Changes in gene expression (messenger ribonucleic acid) profiles of adenomas and normal mucosa
Time frame: Baseline to 6 months
Changes in the microbiome of adenomas and normal mucosa
Time frame: Baseline to 6 months
Future candidate biomarkers measured by genomic and transcriptomic platforms in residual tissue biopsies of adenomas and normal mucosa
Time frame: At 6 months
Results and safety
The study was stopped early (reason given: Protocol has been completed prematurely due to poor accrual and study feasibility.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 May 2026.
- Individual participant dataSharing planned: Yes. NCI is committed to sharing data in accordance with NIH policy. For more details on how clinical trial data is shared, access the link to the NIH data sharing policy page.
Related trials for Attenuated Familial Adenomatous Polyposis
Active, not recruitingObservational study
Familial Adenomatous Poliposys Italian Network (Rete Italiana Poliposi Adenomatosa Familiare)
Familial Adenomatous Polyposis (FAP), Attenuated Familial Adenomatous Polyposis (AFAP)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Eduardo Vilar-Sanchez principal investigator
M.D. Anderson Cancer Center
7 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Biopsy Procedure, Biospecimen Collection and Gastrointestinal Endoscopy in people with Attenuated Familial Adenomatous Polyposis and Colorectal Carcinoma, run by National Cancer Institute (NCI) at 7 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 25 inclusion and 29 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 14 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States, Puerto Rico. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Protocol has been completed prematurely due to poor accrual and study feasibility.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage change in duodenal polyp burden (time frame: Baseline to 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 May 2026.