Statin Therapy in Primary Sclerosing Cholangitis (PSC): a Multi-omics Study

February 23, 2026 updated by: Sidhartha Ranjit Sinha, Stanford University

The Effect of Statin Therapy on Bile Acid Physiology and the Microbiome in Primary Sclerosing Cholangitis (PSC): a Multi-omics Study

PSC is a liver disease that has no medical cure. Patients with PSC are at a greatly increased risk of cancer and infection. Additionally, many patients require a liver transplant. Progress towards a cure has been severely limited by an incomplete understanding of why patients develop PSC. The investigators aim to close this gap by conducting a pilot human study in patients with PSC, using statin therapy as a model

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

Database studies have suggested that use of statins is associated with lower mortality in patients with PSC. Statins are also safe, widely used medications for the treatment of high cholesterol. This track record of safety makes repurposing statins for use in PSC an attractive option.

This study will evaluate the impact of bile acid profile and the microbiome. Rosuvastatin induced changes in cell signaling pathways in the body, as well as its impact of bacterial gene expression in the microbiome will be evaluated. The investigators anticipate that this study will provide key insights into the biologic basis of PSC, which may aid in the development of drugs for the treatment of PSC.

This research study will enroll patients with PSC. The study will be conducted in 3 phases: baseline measurements, study period (treatment with rosuvastatin), and follow-up (follow-up after completing statin treatment). All patients will receive the study drug, and no patients will receive placebo treatment. Rosuvastatin is FDA approved for treatment of high cholesterol, but its use in this trial is off label.

Study Type

Interventional

Enrollment (Estimated)

15

Phase

  • Early Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

Yes

Description

Inclusion Criteria:

  • Males and females, greater than or equal to 18 years of age
  • Established diagnosis of PSC, defined by either appropriate cholangiographic findings or supportive liver biopsy plus an established diagnosis of inflammatory bowel disease (IBD - Crohn's disease or ulcerative colitis) per American College of Gastroenterology (ACG) guidelines for the PSC-IBD arm
  • Hypercholesterolemia with BMI < 25.0 for the comparison arm

Exclusion Criteria:

  • Diagnosis of PSC-autoimmune hepatitis overlap syndrome
  • Woman who are pregnant, nursing, or expect to be pregnant
  • The presence of any comorbidity known to cause secondary sclerosing cholangitis, including: immunoglobulin G-4 (IgG4), associated cholangitis, recurrent bacterial cholangitis, recurrent pyogenic cholangitis, ischemic cholangiopathy, surgical biliary trauma, cholangiocarcinoma, and portal hypertensive biliopathy
  • Diagnosis of a serious medical condition (unless approved in writing by a physician)
  • Patients taking statin therapy prior to study initiation
  • Patients with known clinically allergy to statin therapy
  • aspartate aminotransferase (AST) or alanine aminotransferase (ALT) greater than 5 times the upper limit of normal
  • Bilirubin greater than 3.0 mg/dL
  • Recent use of antibiotics (within the last 90 days)
  • Concurrent use of any immunosuppressive medications (such as any calcineurin inhibitor, steroids at a dose greater than 10 mg of prednisone-equivalents per day)
  • Actively using a fibrate drug
  • Actively using a ritonavir containing drug
  • Familial hypercholesterolemia or other inherited disorder of lipid metabolism
  • Recent myocardial infarction or cerebrovascular accident
  • Body mass index > 25.0 for the comparison arm
  • Chronic kidney disease stage 5 or end-stage renal disease

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Basic Science
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Rosuvastatin therapy
Participants will receive rosuvastatin for 12 weeks followed by a 2 week washout period prior to the final follow-up visit. All patients will receive the study drug, and will serve as their own control. No participants will receive placebo. Rosuvastatin is FDA approved for treatment of high cholesterol, but its use in this trial is off label.
Drug: Rosuvastatin
Rosuvastatin 20 mg tablet once daily by mouth

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in bile acid (BA) profile: total bile acid
Time Frame: Baseline and week 12
BA profile of biliary/intestinal aspirate and/or feces in response to statin therapy.
Baseline and week 12
Change in bile acid (BA) profile: secondary bile acids:primary bile acids ratio
Time Frame: Baseline and week 12
BA profile of biliary/intestinal aspirate and/or feces in response to statin therapy.
Baseline and week 12
Change in bile acid (BA) profile: conjugated:unconjugated BAs ratio
Time Frame: Baseline and week 12
BA profile of biliary/intestinal aspirate and/or feces in response to statin therapy.
Baseline and week 12
Change in pathogen density in the small intestine
Time Frame: Baseline and week 12
Measure impact of statin therapy upon pathogen density (ratio of good bacteria to pathogenic bacteria) within the microbial community of the duodenum.
Baseline and week 12
Change in bacterial gene expression profile in the small intestine
Time Frame: Baseline and week 12
This outcome aims to develop an understanding of the profile of microbial metabolic pathways in the duodenum and changes to the profile in response to statin therapy; gene sequencing with be done using shotgun metagenomics followed by pathway analysis.
Baseline and week 12

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in bile acid (BA) profile: total bile acid
Time Frame: Baseline, week 4, week 14
BA profile of biliary/intestinal aspirate and/or feces in response to statin therapy.
Baseline, week 4, week 14
Change in bile acid (BA) profile: secondary bile acids:primary bile acids ratio
Time Frame: Baseline, week 4, week 14
BA profile of biliary/intestinal aspirate and/or feces in response to statin therapy.
Baseline, week 4, week 14
Change in bile acid (BA) profile: conjugated:unconjugated BAs ratio
Time Frame: Baseline, week 4, week 14
BA profile of biliary/intestinal aspirate and/or feces in response to statin therapy.
Baseline, week 4, week 14
Change in pathogen density in the small intestine
Time Frame: Baseline, week 4, week 14
Measure impact of statin therapy upon pathogen density (ratio of good bacteria to pathogenic bacteria) within the microbial community of the duodenum.
Baseline, week 4, week 14
Change in bacterial gene expression profile in the small intestine
Time Frame: Baseline, week 4, week 14
This outcome aims to develop an understanding of the profile of microbial metabolic pathways in the duodenum and changes to the profile in response to statin therapy; gene sequencing with be done using shotgun metagenomics followed by pathway analysis.
Baseline, week 4, week 14

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Stanford University

Investigators

  • Principal Investigator: Sidhartha Sinha, MD, Stanford University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 31, 2023

Primary Completion (Estimated)

May 31, 2027

Study Completion (Estimated)

December 31, 2027

Study Registration Dates

First Submitted

June 1, 2023

First Submitted That Met QC Criteria

June 12, 2023

First Posted (Actual)

June 22, 2023

Study Record Updates

Last Update Posted (Actual)

February 27, 2026

Last Update Submitted That Met QC Criteria

February 23, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 67292

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

Individual participant data will not be shared

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Subscribe