What side effects are associated with this type of intervention?

Common treatments for Primary Sclerosing Cholangitis (PSC) include statins, ursodeoxycholic acid (UDCA), and immunosuppressive medications. Statins, such as lovastatin, simvastatin, atorvastatin, and rosuvastatin, can cause muscle pain, liver enzyme abnormalities, and, in rare cases, rhabdomyolysis. UDCA is generally well-tolerated but may cause diarrhea and, at higher doses, could be detrimental. Immunosuppressive medications, used to manage associated autoimmune conditions, can increase the risk of infections and may have other side effects depending on the specific drug used.

What prior FDA approvals exist?

As of now, there are no FDA-approved treatments specifically for Primary Sclerosing Cholangitis (PSC). However, research is ongoing into therapies with anti-inflammatory and immunomodulatory effects, such as statins, which are being studied for their potential benefits in PSC. Statins, commonly used for hypercholesterolemia, have shown promise due to their anti-inflammatory properties. Other biologic agents like vedolizumab and ustekinumab, which target specific molecular pathways involved in inflammation, are also being explored for their potential use in PSC, although they are primarily approved for inflammatory bowel diseases.

What other types of research exist?

Promising novel alternatives to statin therapy for PSC patients include ursodeoxycholic acid (UDCA) at a standard dose (approximately 15 mg/kg/day) and vancomycin, which has shown positive outcomes in small observational studies. Additionally, immunosuppressive medications used for autoimmune liver diseases may offer benefits, although they do not alter the course of PSC. These alternatives provide anti-inflammatory and immunomodulatory effects that could be beneficial for PSC patients.

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HMG-CoA Reductase Inhibitor

Statin Therapy for Primary Sclerosing Cholangitis

Q: What is the mechanism of action of this treatment?

The most common treatments for Primary Sclerosing Cholangitis (PSC) include statins, which are being studied for their anti-inflammatory and immunomodulatory effects. These mechanisms are crucial for PSC patients as the disease involves chronic inflammation and immune-mediated damage to the bile ducts, leading to fibrosis and cirrhosis. Statins may help reduce this inflammation and modulate the immune response, potentially slowing disease progression. Ursodeoxycholic acid (UDCA) is another treatment that improves bile flow and reduces liver enzyme levels, although its impact on disease progression is limited. Immunosuppressive medications are also used to manage associated autoimmune conditions but do not significantly alter the course of PSC.

Phase < 1

Recruiting

Led By Chiraag Kulkarni, MD

Research Sponsored by Stanford University

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, week 4, week 14

Awards & highlights

No Placebo-Only Group

Summary

This trial uses Rosuvastatin, a cholesterol-lowering drug, to treat patients with PSC, a severe liver disease. The study aims to see if the drug can change cell communication and gut bacteria behavior to help understand and treat PSC. Rosuvastatin is effective in lowering LDL cholesterol and is well tolerated in various dosages.

Who is the study for?

This trial is for adults over 18 with Primary Sclerosing Cholangitis (PSC) and Inflammatory Bowel Disease. Participants must not be pregnant, have PSC-autoimmune hepatitis overlap, secondary sclerosing cholangitis conditions, serious medical issues without physician approval, or a history of statin use or allergy to it. They should also meet certain blood test criteria and not be on specific medications.

What is being tested?

The study tests the effects of Rosuvastatin, a cholesterol-lowering drug (statin), in patients with PSC. It aims to understand how this treatment might help manage PSC by monitoring changes through various health markers collected from participants.

What are the potential side effects?

While the side effects are not explicitly listed here, common side effects of statins like Rosuvastatin include muscle pain or weakness, headache, difficulty sleeping, flushing of the skin, nausea or dizziness. More serious but rare side effects can involve liver damage and increased blood sugar levels.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, week 4, week 14

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, week 4, week 14

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, week 4, week 14 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Change in bacterial gene expression profile in the small intestine

Change in bile acid (BA) profile: conjugated:unconjugated BAs ratio

Change in bile acid (BA) profile: secondary bile acids:primary bile acids ratio

+2 more

Side effects data

From 2018 Phase 4 trial • 12 Patients • NCT03074630

genital fungal infection

100%

80%

60%

40%

20%

Study treatment Arm

Adverse Events

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Rosuvastatin therapyExperimental Treatment1 Intervention

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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Rosuvastatin

2019

Completed Phase 4

~3150

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Primary Sclerosing Cholangitis (PSC) include statins, which are being studied for their anti-inflammatory and immunomodulatory effects. These mechanisms are crucial for PSC patients as the disease involves chronic inflammation and immune-mediated damage to the bile ducts, leading to fibrosis and cirrhosis. Statins may help reduce this inflammation and modulate the immune response, potentially slowing disease progression. Ursodeoxycholic acid (UDCA) is another treatment that improves bile flow and reduces liver enzyme levels, although its impact on disease progression is limited. Immunosuppressive medications are also used to manage associated autoimmune conditions but do not significantly alter the course of PSC.

Gemfibrozil. A reappraisal of its pharmacological properties and place in the management of dyslipidaemia.Beneficial Effects of Statins on the Rates of Hepatic Fibrosis, Hepatic Decompensation, and Mortality in Chronic Liver Disease: A Systematic Review and Meta-Analysis.Statins in heart failure--With preserved and reduced ejection fraction. An update.