Cholic Acid for Hepatic Steatosis in Lipodystrophy
Condition: Hepatic Steatosis
MeSH terms: Fatty Liver; Lipodystrophy
Intervention: Cholic Acid
Intervention description: Capsules of active Cholic Acid or matching placebo, total dose is 15 mg/kg per day, maximum dose of 1500 mg per day, taken PO, BID.
Intervention MeSH terms: Cholic Acids
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Enrollment: 18.0
Sex: All
Minimum age: 6 Years
Maximum age: 70 Years
Healthy volunteers: No
Keywords: lipodystrophy

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.
    

Eligibility 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