Use of Fenofibrate for Primary Biliary Cirrhosis

Condition(s) targeted: Primary Biliary Cirrhosis

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

Phase: Phase 2

Status: Recruiting

Sponsored by: University of Florida

Official(s) and/or principal investigator(s):
Cynthia Levy, MD, Principal Investigator, Affiliation: University of Florida

Overall contact:
Cynthia Levy, MD, Phone: 352-376-1611, Ext: 7-5446, Email: cynthia.levy@medicine.ufl.edu

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.

Official title: Pilot Study of Fenofibrate for Primary Biliary Cirrhosis

Study design: Treatment, Open Label, Single Group Assignment, Safety/Efficacy Study

Primary outcome: Serum level of alkaline phosphatase, serum immunoglobulin M level and Mayo risk score

Secondary outcome: Quality of life according to NIDDK questionnaire

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.

Minimum age: 21 Years. Maximum age: 75 Years. Gender(s): Both.

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

Cynthia Levy, MD, Phone: 352-376-1611, Ext: 7-5446, Email: cynthia.levy@medicine.ufl.edu

University of Florida, Gainesville, Florida 32610, United States; Recruiting
Cynthia Levy, MD, Phone: 352-376-1611, Ext: 7-5446, Email: cynthia.levy@medicine.ufl.edu
Joy Peter, RN, BSN, CCRC, Phone: 352-392-7956, Email: joy.peter@medicine.ufl.edu
Cynthia Levy, MD, Principal Investigator

Mayo Clinic, Rochester, Minnesota 55905, United States; Recruiting
Roberta Jorgensen, Phone: 507-538-0678, Email: jorgensen.roberta@mayo.edu
Jill Keach, Phone: (507)538-0678, Email: keach.jill@mayo.edu
Keith Lindor, MD, Principal Investigator

Starting date: August 2007
Ending date: August 2010
Last updated: December 13, 2007