Use of Fenofibrate for Primary Biliary Cirrhosis

Phase 2

Completed

• Conditions: Primary Biliary Cirrhosis
• Interventions: Drug: Fenofibrate IDD-P (Insoluble Drug Delivery-Micro Particle)

• Registration Number: NCT00575042
• Lead Sponsor: University of Florida

Brief Summary

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

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.

Study Timeline

• Study Start: 2007-08
• Study First Submitted: 2007-12-13
• Study First Submitted QC: 2007-12-13
• Study First Posted: 2007-12-17
• Primary Completion: 2009-08
• Study Completion: 2010-08
• Results First Submitted: 2011-12-02
• Results First Submitted QC: 2011-12-02
• Results First Posted: 2012-01-09
• Status Verified: 2018-02
• Last Update Submitted: 2018-02-07
• Last Update Posted: 2018-03-08

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 20

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

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Patients treated with Fenofibrate	Fenofibrate IDD-P (Insoluble Drug Delivery-Micro Particle)	Fenofibrate IDD-P (Insoluble Drug Delivery-Micro Particle) 160 mg table per day for 1 year

Primary Outcome Measures

Name	Time	Method
Serum Level of Alkaline Phosphatase	1 year	We analyzed whether there was a difference in median ALP at 1 year compared to baseline values.

Trial Locations

Locations (2)

University of Florida; 🇺🇸 Gainesville, Florida, United States

Mayo Clinic; 🇺🇸 Rochester, Minnesota, United States