Primary Sclerosing CholangitisEarly Phase 1NCT05912387
Statin Therapy in Primary Sclerosing Cholangitis (PSC): a Multi-omics Study
A Early Phase 1 study testing Rosuvastatin in people with Primary Sclerosing Cholangitis and Inflammatory Bowel Diseases, run by Stanford University at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 80 yearsHealthy volunteers can take part
- Conditions the study is for
- Primary Sclerosing Cholangitis, Inflammatory Bowel Diseases
- What is being tested?
- Rosuvastatin
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- 31 May 2023to 31 Dec 2027
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
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 to 80 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 16Show fewer
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Stanford University
Stanford, California, 94305
Touran Fardeen, tfardeen@stanford.edu
About this study
Official title: 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
Read the 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.
- Being tested
- Rosuvastatin
- Keywords
- Statin
- Medical subject headings
- Cholangitis, Sclerosing, Inflammatory Bowel Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RosuvastatinDrug
Rosuvastatin 20 mg tablet once daily by mouth
| Study group | Role | What they receive |
|---|---|---|
| Rosuvastatin therapy | Experimental | 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. Receives: Drug: Rosuvastatin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in bile acid (BA) profile: total bile acid
BA profile of biliary/intestinal aspirate and/or feces in response to statin therapy.
Measured: Baseline and week 12
Change in bile acid (BA) profile: secondary bile acids:primary bile acids ratio
BA profile of biliary/intestinal aspirate and/or feces in response to statin therapy.
Measured: Baseline and week 12
Change in bile acid (BA) profile: conjugated:unconjugated BAs ratio
BA profile of biliary/intestinal aspirate and/or feces in response to statin therapy.
Measured: Baseline and week 12
Change in pathogen density in the small intestine
Measure impact of statin therapy upon pathogen density (ratio of good bacteria to pathogenic bacteria) within the microbial community of the duodenum.
Measured: Baseline and week 12
Change in bacterial gene expression profile in the small intestine
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.
Measured: Baseline and week 12
Secondary outcomes (5)
Change in bile acid (BA) profile: total bile acid
BA profile of biliary/intestinal aspirate and/or feces in response to statin therapy.
Measured: Baseline, week 4, week 14
Change in bile acid (BA) profile: secondary bile acids:primary bile acids ratio
BA profile of biliary/intestinal aspirate and/or feces in response to statin therapy.
Measured: Baseline, week 4, week 14
Change in bile acid (BA) profile: conjugated:unconjugated BAs ratio
BA profile of biliary/intestinal aspirate and/or feces in response to statin therapy.
Measured: Baseline, week 4, week 14
Change in pathogen density in the small intestine
Measure impact of statin therapy upon pathogen density (ratio of good bacteria to pathogenic bacteria) within the microbial community of the duodenum.
Measured: Baseline, week 4, week 14
Change in bacterial gene expression profile in the small intestine
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.
Measured: Baseline, week 4, week 14
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jun 2023
- Study start31 May 2023
- Primary completion31 May 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Sclerosing CholangitisStudies of the same condition, open and under way.
- More trials run by Stanford UniversityThe 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
- Early Phase 1
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Stanford University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Sidhartha Ranjit Sinha, Stanford University
- Investigators
- Sidhartha Sinha, MD, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05912387
- Sponsor's protocol ID
- 67292
- Status last verified
- Feb 2026
- First submitted
- 1 Jun 2023
- Last updated
- 27 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: Individual participant data will not be shared
Study at a glance
From the registry record, last updated by the sponsor on 27 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Effect of Statin Therapy on Bile Acid Physiology and the Microbiome in Primary Sclerosing Cholangitis (PSC): a Multi-omics Study
- ClinicalTrials.gov ID
- NCT05912387
- Acronym / sponsor ID
- 67292
- Keywords
- Statin
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Stanford UniversityUniversity, hospital or other
- Study start
- 31 May 2023 (actual)
- Primary completion
- 31 May 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 22 Jun 2023 (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.
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
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 design
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Rosuvastatin therapy | Experimental | Drug: RosuvastatinParticipants 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. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (16 listed)
- 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
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 |
|---|---|---|
| RosuvastatinRosuvastatin 20 mg tablet once daily by mouth | Drug | Rosuvastatin therapy |
Primary outcome
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.
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.
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.
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.
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.
5 secondary outcomes
Change in bile acid (BA) profile: total bile acid
Time frame: 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
Change in bile acid (BA) profile: conjugated:unconjugated BAs ratio
Time frame: Baseline, week 4, week 14
Change in pathogen density in the small intestine
Time frame: Baseline, week 4, week 14
Change in bacterial gene expression profile in the small intestine
Time frame: Baseline, week 4, week 14
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Feb 2026.
- Individual participant dataSharing planned: No. Individual participant data will not be shared
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PSC
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.
Sidhartha Sinha, MD principal investigator
Stanford University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Rosuvastatin in people with Primary Sclerosing Cholangitis and Inflammatory Bowel Diseases, run by Stanford University at 1 site in United States.
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 3 inclusion and 16 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: looking for participants now, as of the sponsor's last update on 27 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Touran Fardeen ((650) 725-5890, tfardeen@stanford.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in bile acid (BA) profile: total bile acid (time frame: Baseline and week 12). The study lists 5 primary outcomes in all. 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 27 Feb 2026.