Cholic Acid for Hepatic Steatosis in Lipodystrophy

• Status: Completed
• Trial end date: 2011-04-01
• Target enrollment: 0
• Participant gender: All

Summary

To evaluate the efficacy and safety of cholic acid therapy in treating lipodystrophy patients with hepatic steatosis. This is a randomized, double-blind, placebo-controlled cross-over study.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of Texas Southwestern Medical Center
• Collaborator: FDA Office of Orphan Products Development
• Treatments: Cholic Acids

Criteria

Inclusion Criteria:

• Patients with lipodystrophies as diagnosed by clinical criteria.

• Hepatic steatosis (>5.6% hepatic triglyceride content) as demonstrated by 1H magnetic resonance spectroscopy.

• Age 6-70 years.

• Alcohol intake of less than 40 g per week.

Exclusion Criteria:

• Laboratory or other histologic findings highly suggestive of liver disease due to causes other than non-alcoholic steatohepatitis, such as chronic viral hepatitis, autoimmune hepatitis, primary biliary cirrhosis, biliary obstruction or genetic liver diseases such as Wilson's disease, hemochromatosis or alpha-1-antitrypsin deficiency.

• Treatment with drugs associated with steatohepatitis, e.g., corticosteroids, high dose estrogens, methotrexate, amiodarone, , sulfasalazine, or oxacillin in the 6 months prior to the study.

• Decompensated liver disease as evidenced by clinical features of hepatic failure (variceal bleeding, ascites, hepatic encephalopathy etc.) and laboratory investigations (prolonged prothrombin time, hypoalbuminemia, presence of esophageal varices etc.)

• Evidence of hepatocellular carcinoma: alpha-fetoprotein levels greater than 200 ng/ml and/or liver mass on imaging study suggestive of liver cancer.

• Use of drugs which can potentially decrease hepatic steatosis during previous 3 months; ursodeoxycholic acid, high-dose vitamin E, betaine, acetylcysteine and choline. Thiazolidinediones are allowed if dose has been stable for 3 months prior to screening.

• Significant systemic or major illnesses other than liver disease, such as congestive heart failure, cerebrovascular disease, respiratory failure, renal failure (serum creatinine >2 mg/dL), acute pancreatitis, organ transplantation, serious psychiatric disease, and malignancy, that could interfere with the trial and adequate follow up.

• Acute medical illnesses precluding participation in the studies.

• Known HIV-infected patient.

• Current substance abuse.

• Pregnant or lactating women.

• Hematocrit of less than 30%. - History of weight loss during past 3 months.

• Patients on bile acid binding resins, cholestyramine, colestipol, colesevelam.

• Hypersensitivity or intolerance to CA or any components of its formulation