Clinical trial NCT00575042: Use of Fenofibrate for Primary Biliary Cirrhosis
Official title: Pilot Study of Fenofibrate for Primary Biliary Cirrhosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Primary Biliary Cirrhosis
Keywords: PBC
Primary Biliary Cirrhosis
Fenofibrate
Triglide
Lead sponsor: University of Florida
Sponsor class: OTHER
Collaborators: name: The PBCers Organization
name: Shionogi Inc.
First posted: 2007-12-17
Enrollment: 20
Sex: ALL
Minimum age (years): 21
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Serum Level of Alkaline Phosphatase
timeFrame: 1 year
Secondary outcomes:
Other outcomes:
Locations:
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
Study officials:
affiliation: University of Florida
name: Cynthia Levy, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Levy C, Peter J, Keach J, Petz J, Lindor KD, Cabrera R, et al. Fenofibrate improves liver biochemistries in primary biliary cirrhosis. Hepatology. 2009;50(4 (suppl)):995A.
citation: Levy C, Peter JA, Nelson DR, Keach J, Petz J, Cabrera R, Clark V, Firpi RJ, Morelli G, Soldevila-Pico C, Lindor K. Pilot study: fenofibrate for patients with primary biliary cirrhosis and an incomplete response to ursodeoxycholic acid. Aliment Pharmacol Ther. 2011 Jan;33(2):235-42. doi: 10.1111/j.1365-2036.2010.04512.x. Epub 2010 Nov 17.