Fenofibrate for Primary Sclerosing Cholangitis

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Mayo Clinic

Disqualifiers: Ischemic cholangiopathy, Primary biliary cholangitis, Autoimmune hepatitis, GFR <30 ml/min, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 3

Duration: 36 months

80 Participants Needed

Trial Summary

Do I need to stop my current medications to join the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team to understand any specific requirements.

What data supports the effectiveness of the drug fenofibrate for primary sclerosing cholangitis?

The study showed that fenofibrate significantly reduced liver enzyme levels (ALP and ALT) in patients with primary sclerosing cholangitis, with 66.7% of patients achieving a significant reduction in ALP levels compared to 20% in the placebo group. This suggests that fenofibrate may improve liver function in these patients. ^{1 2 3 4 5}

Is fenofibrate safe for use in humans?

Fenofibrate has been studied for safety in patients with liver conditions like primary biliary cholangitis and primary sclerosing cholangitis. While its long-term effects are not fully clear, short-term studies have shown it can be used safely in these patients, with some improvements in liver function tests. ^{1 2 4 5 6}

How does the drug fenofibrate differ from other treatments for primary sclerosing cholangitis?

Fenofibrate is unique for primary sclerosing cholangitis (PSC) because it targets liver biochemistry, specifically reducing alkaline phosphatase (ALP) levels, which may improve disease prognosis. Unlike other treatments, it has shown significant reductions in ALP and alanine aminotransferase (ALT) levels in PSC patients, suggesting a potential benefit where no standard treatment exists to improve survival or delay liver transplantation. ^{1 2 3 4 5}

What is the purpose of this trial?

This study aims to determine the efficacy of 36 months once-daily fenofibrate in preventing clinically-detectable recurrence of primary sclerosing cholangitis after liver transplantation, compared with a historical control cohort that was not treated with

Research Team

Channa Jayasekera

Principal Investigator

Mayo Clinic

Eligibility Criteria

This trial is for individuals who have had a liver transplant due to primary sclerosing cholangitis (PSC) and are at risk of recurrence. Specific eligibility criteria were not provided, but typically participants would need to meet certain health standards and may be required to have no history of allergic reactions to the interventions being tested.

Inclusion Criteria

I had a liver transplant for cancer, have bowel disease, or had certain infections or rejections after my transplant.

I had a liver transplant for PSC or related cancer between 1 and 7 years ago.

I can travel to Mayo Clinic Arizona every 4 months at my own expense.

Exclusion Criteria

I am pregnant or planning to become pregnant.

I have a condition that looks like rPSC affecting my bile ducts.

I had a liver transplant due to specific liver conditions and have unresolved issues with the liver's blood supply.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive fenofibrate 160mg oral daily for 36 months to prevent clinically detectable rPSC after liver transplantation

36 months

Serum assessments every 3 months, MRI at baseline, 12 months, and 36 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 months

Treatment Details

Interventions

• Fenofibrate

Trial Overview The study tests if taking fenofibrate daily for 36 months can prevent PSC from coming back after a liver transplant. It's compared with past data where patients didn't get this treatment. Participants will also undergo MRI scans using Gd-EOB-DPTA contrast agent and regular blood tests.

Participant Groups

Group I: Treatment Experimental Treatment 3 Interventions

Individuals who underwent liver transplantation for primary sclerosing cholangitis 1-7 years before study initiation, and meeting study criteria, will receive fenofibrate 160mg oral daily for 36 months Participants will undergo the following serum assessments as part of the study every 3 months during the study period: total bile acids, bile acid profile, fibroblast growth factor 19, and 7-alpha-C4 Participants will undergo gadoxate-enhanced magnetic resonance imaging at baseline, 12 months, and 36 months.

Group II: Historical control Active Control 1 Intervention

Historical control of individuals who underwent liver transplantation for primary sclerosing cholangitis who were not treated with any peroxisome proliferator activated receptor agonist treatment.

Who Is Running the Clinical Trial?

Mayo Clinic

Lead Sponsor

Trials

3,427

Recruited

3,221,000+

Findings from Research

In a study of 106 patients with primary biliary cholangitis (PBC) who did not respond well to ursodeoxycholic acid (UDCA), adding fenofibrate (FF) to their treatment significantly reduced alkaline phosphatase (ALP) levels and improved liver transplant-free survival after one year.

Patients receiving FF alongside UDCA showed better stabilization or improvement in liver fibrosis and bile duct loss compared to those on UDCA alone, indicating that FF may enhance the overall management of PBC.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fenofibrate improves GLOBE and UK-PBC scores and histological features in primary biliary cholangitis. Wang, L., Sun, K., Tian, A., et al.[2023]

References

1. United States pubmed.ncbi.nlm.nih.gov

Fenofibrate in primary sclerosing cholangitis; a randomized, double-blind, placebo-controlled trial. [2023]

2. Italy pubmed.ncbi.nlm.nih.gov

Fenofibrate improves GLOBE and UK-PBC scores and histological features in primary biliary cholangitis. [2023]

3. France pubmed.ncbi.nlm.nih.gov

Fenofibrate is effective adjunctive therapy in the treatment of primary biliary cirrhosis: A meta-analysis. [2018]

4. United States pubmed.ncbi.nlm.nih.gov

A randomized, controlled trial on fenofibrate in primary biliary cholangitis patients with incomplete response to ursodeoxycholic acid. [2022]

5. United States pubmed.ncbi.nlm.nih.gov

Long-Term Fenofibrate Treatment in Primary Biliary Cholangitis Improves Biochemistry but Not the UK-PBC Risk Score. [2022]

6. United States pubmed.ncbi.nlm.nih.gov

Effectiveness of Fenofibrate in Treatment-Naive Patients With Primary Biliary Cholangitis: A Randomized Clinical Trial. [2023]