Using statins to understand and treat Primary Sclerosing Cholangitis

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

Early Phase 1 Interventional Stanford University · NCT05912387

This study is testing if the cholesterol-lowering drug rosuvastatin can help people with Primary Sclerosing Cholangitis by looking at how it affects their bile acids and gut bacteria.

Quick facts

PhaseEarly Phase 1
Study typeInterventional
Enrollment15 (estimated)
Ages18 Years to 80 Years
SexAll
SponsorStanford University Academic / other
Drugs / interventionsprednisone
Locations1 site (Stanford, California)
Trial IDNCT05912387 on ClinicalTrials.gov

What this trial studies

This study investigates the effects of rosuvastatin, a commonly used cholesterol-lowering medication, on patients with Primary Sclerosing Cholangitis (PSC). The research aims to explore how statin therapy influences bile acid profiles and the microbiome, potentially revealing insights into the biological mechanisms underlying PSC. The study will consist of three phases: baseline measurements, treatment with rosuvastatin, and follow-up assessments after treatment. All participants will receive the active medication, as there is no placebo group in this pilot study.

Who should consider this trial

Good fit: Ideal candidates include adults aged 18 and older with a confirmed diagnosis of PSC and inflammatory bowel disease.

Not a fit: Patients with PSC-autoimmune hepatitis overlap syndrome or other specific comorbidities may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to new therapeutic strategies for managing PSC and improving patient outcomes.

How similar studies have performed: Previous studies have suggested that statins may lower mortality in PSC patients, indicating potential for success in this novel application.

Eligibility criteria

Show full inclusion / exclusion criteria
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

Where this trial is running

Stanford, California

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Primary Sclerosing CholangitisInflammatory Bowel DiseasesStatin
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.