Using obeticholic acid to prevent progression in Barrett's esophagus
Official title: Obeticholic Acid for Prevention in Barrett's Esophagus
Run by National Cancer Institute (NCI) · NCT04939051 · Data updated 2026-11-12
This study is testing if the medication obeticholic acid can help prevent Barrett's esophagus from getting worse in people at risk of developing esophageal cancer.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Ages | 18 Years and up |
| Sex | All |
| Enrollment | 30 (estimated) |
| Sponsor | National Cancer Institute (NCI) NIH |
| Drugs / interventions | chemotherapy, immunotherapy, radiation |
| Locations | 8 sites (Kansas City, Kansas and 7 other locations) |
| Status | Recruiting |
| Started | January 3, 2024 |
| Main results expected | September 1, 2027 |
| Study ends | September 1, 2027 |
| Registry record updated | August 11, 2026 |
| Trial ID | NCT04939051 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates include adults aged 18 and older with histologically-confirmed Barrett's esophagus and specific dysplasia classifications.
✕Probably not a fit
Patients with high-grade dysplasia or esophageal adenocarcinoma may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This phase II trial investigates the effects of obeticholic acid on patients with Barrett's esophagus, a condition that can lead to esophageal adenocarcinoma. The study aims to assess changes in specific cell populations in esophageal tissue after treatment with obeticholic acid compared to a placebo. It will also explore the drug's impact on bile acid composition, biomarkers of cancer progression, and histological changes in Barrett's tissue. Participants will receive daily doses of obeticholic acid for 180 days while undergoing various assessments.
Why it matters
Potential benefit: If successful, this treatment could significantly reduce the risk of progression to esophageal cancer in patients with Barrett's esophagus.
How similar studies have gone: While this approach is novel in the context of Barrett's esophagus, similar studies have shown promise in targeting bile acid pathways for cancer prevention.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria:
* Known diagnosis of histologically-confirmed BE with either no dysplasia, indefinite for dysplasia, or low-grade dysplasia as defined by the presence of specialized columnar epithelium on histology and \>= 2 cm of involvement on endoscopy
* Adequate Barrett's mucosa, which is defined as at least one sample with \>= 50% intestinal metaplasia in biopsies required to satisfy the endpoints of the study
* Participants are on proton pump inhibitors (PPI) therapy for \>= 28 days duration
* Age \>= of 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 \>= 10g/dL or hematocrit \>= 30 %
* Leukocyte count \>= 3,500/microliter
* Platelet count \>= 100,000/microliter
* Creatinine clearance (calculated if measured is not available) \>= 30mL/min/1.73m\^2
* Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase \[SGOT\])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase \[SGPT\]) =\< 1.5 X 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
* The effects of OCA on the developing human fetus are unknown. For this reason, all men and women of child-bearing potential 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
* Ability to understand the study procedures, benefits and risks, and sign a written informed consent document. Non-English speaking participants are allowed to enroll even if they skip answering quality-of-life (QOL) questionnaires. Special efforts will be made through community advisory boards at participating sites to reach Spanish speaking participants
* Willing to undergo testing for human immunodeficiency virus (HIV) testing if not tested within the past 6 months
* Willing to undergo hepatitis B and C screening if not tested within the past 6 months
* Willing and able to adhere to the prohibitions and restrictions specified in the approved protocol
* Willingness to moderate alcohol intake (consuming no more than 1 or 2 alcoholic drinks per day for women and men, respectively)
* 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)
Exclusion Criteria:
* History of prior ablative therapy such as radiofrequency ablation, cryotherapy or argon plasma coagulation (APC) in BE segment
* Prior use of OCA
* Prior history or presence of high-grade disease (HGD) or cancer on pre-intervention endoscopy
* Cutaneous diseases manifesting with severe pruritus
* Individuals with active, known or suspected chronic liver disease including cirrhosis, nonalcoholic steatohepatitis (NASH) with fibrosis or 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 pancreatic abnormalities
* Individuals with hepatic steatosis and velocity \> 1.7 m/sec as determined by liver ultrasound elastography. Results of a right upper quadrant ultrasound with elastography performed within 6 months of starting study treatment may be used to assess this criteria
* Individuals with hyperlipidemia that is 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
* Individuals with 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
* 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
* 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
* Participants may not be receiving any other investigational agents
Where it's running
-
University of Kansas Cancer Center
Kansas City, Kansas, United States Recruiting -
University of Michigan Rogel Cancer Center
Ann Arbor, Michigan, United States Recruiting -
Washington University School of Medicine
St Louis, Missouri, United States Recruiting -
UNC Lineberger Comprehensive Cancer Center
Chapel Hill, North Carolina, United States Recruiting -
Case Western Reserve University
Cleveland, Ohio, United States Recruiting -
Cleveland Clinic Foundation
Cleveland, Ohio, United States Recruiting
Show 2 more locations
-
Seidman Cancer Center at University Hospitals Case Medical Center, Case Comprehensive Cancer Center
Cleveland, Ohio, United States Not yet recruiting -
University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States Not yet recruiting
Study contacts
- Principal investigator: Prashanthi Thota — University of Michigan Rogel Cancer Center
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
-
Itraconazole with endoscopic ablation to prevent esophageal cancer in high‑risk Barrett's esophagus
This study tests if adding itraconazole to the standard endoscopic ablation can better prevent esophageal cancer in people with high-risk Barrett's esophagus.
-
Detecting Barrett's Esophagus in Patients Without GERD Symptoms
This study is testing a new way to find Barrett's esophagus in people who don’t have GERD symptoms by using a simple capsule test instead of the usual endoscopy.
-
Assessing Barrett's Esophagus in Patients with Anorexia Nervosa and Purging Behaviors
This study is trying to see if people with anorexia nervosa who purge have Barrett's esophagus, using non-invasive tests to check for this condition in patients aged 18-65.
-
Using a swallowable sponge to collect esophageal samples for Barrett's esophagus detection
This study is testing a swallowable sponge to see if it can safely collect samples from the esophagus to help detect Barrett's esophagus and esophageal cancer in patients who may have these conditions.
-
Registry for patients undergoing esophageal cancer screening
This study is collecting information from patients getting screened for Barrett Esophagus and Esophageal Adenocarcinoma using a new test to see how well it works and how patients feel about the process.
-
Comparing surveillance and therapy for Barrett's esophagus with low-grade dysplasia
This study is testing whether regular check-ups or a treatment to remove precancerous cells is better for people with Barrett's esophagus and low-grade dysplasia.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.