Overview

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