Use of Fenofibrate for Primary Biliary Cirrhosis

Study Overview
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Brief Summary
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This is a pilot study to evaluate the safety and efficacy of fenofibrate on patients with primary biliary cirrhosis who have an incomplete response to ursodeoxycholic acid.

Detailed Description
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This is a multicenter open label pilot study to evaluate the efficacy of fenofibrate in 20 patients with PBC treated for 1 year. A randomized design would not be feasible at this stage of the research. Two sites are enrolling patients: University of Florida, Gainesville, and Mayo Clinic Rochester, with the first as the coordinating site. The total number of patients to start treatment will not exceed 20. Our endpoints are 1) surrogate markers of disease activity -serum alkaline phosphatase and immunoglobulin M; 2) a well validated prognostic index -Mayo risk score and 3) the NIDDK quality of life questionnaire.

Official Title
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Pilot Study of Fenofibrate for Primary Biliary Cirrhosis

Conditions
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Primary Biliary Cirrhosis

Intervention / Treatment
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* Drug: Fenofibrate IDD-P (Insoluble Drug Delivery-Micro Particle)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Established diagnosis of PBC by serological (positive antimitochondrial antibodies) and/or histological evidence (diagnostic liver biopsy) Previous treatment with UDCA 13-15 mg/kg/day for at least 1 year Persistent elevation of serum alkaline phosphatase ≥ 2 times the upper limit of normal on two separate measurements Female patients of childbearing age should have a pregnancy test done within -days of the beginning of this trial, and should agree to be on adequate contraception throughout the study period Signed informed consent after careful review of the information and study details by one of the investigators Exclusion Criteria Hypersensitivity to fenofibrate Prisoners and institutionalized subjects Pregnant or nursing women Anticipated need for liver transplantation in one year (estimated one year survival < 80%) as determined by the Mayo risk score. The Mayo risk score takes into account the patient's age, serum bilirubin, albumin and prothrombin time, as well as presence or absence of peripheral edema. Recipients of liver transplantation Recurrent variceal hemorrhage, uncontrolled encephalopathy or refractory ascites Co-existing liver diseases, such as primary sclerosing cholangitis, acute or chronic hepatitis, alcoholic liver disease, choledocholithiasis, autoimmune hepatitis, cholangiocarcinoma Acute or chronic renal failure Known history of cholecystitis with intact gallbladder Current use of statins, as the concomitant use of fibrates and statins would increase the risk of toxicity

Ages Eligible for Study
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Minimum Age: 21 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Patients treated with Fenofibrate<br>Fenofibrate IDD-P (Insoluble Drug Delivery-Micro Particle) 160 mg table per day for 1 year | Drug: Fenofibrate IDD-P (Insoluble Drug Delivery-Micro Particle)<br>* 160 mg per day for 1 year<br>* Other names: Triglide-Insoluble Drug Delivery-Micro Particle Fenofibrate (IDD-P);|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Serum Level of Alkaline Phosphatase | We analyzed whether there was a difference in median ALP at 1 year compared to baseline values. | 1 year | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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PBC, Primary Biliary Cirrhosis, Fenofibrate, Triglide