Cholic Acid for Hepatic Steatosis in Lipodystrophy

NCT00457639 | Completed Phase 2 Results DMC

• 2 other identifiers: STU 122010-032FD003085
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Hepatic Steatosis

Keywords

lipodystrophy

Outcome Measures

Primary Outcomes (1)

• Hepatic Triglyceride (%)

  Measured by proton magnetic resonance spectroscopy (MRS)

  6 months

Secondary Outcomes (1)

• Serum Triglycerides

  Months 6

Study Arms (2)

Cholic Acid active capsules

EXPERIMENTAL

Cholic Acid weight based dose for 6 months double-blind

Drug: Cholic Acid

Placebo for Cholic Acid

PLACEBO COMPARATOR

Placebo for Cholic Acid for 6 months double-blind

Drug: Cholic Acid

Interventions

Cholic Acid DRUG

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.

Cholic Acid active capsules; Placebo for Cholic Acid

Eligibility Criteria

• Age: 6 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• University of Texas Southwestern Medical Center: lead
• FDA Office of Orphan Products Development: collaborator

Study Sites (1)

University of Texas Southwestern Medical Center

Dallas, Texas, 75390-9052, United States

Location

Related Publications (1)

• Ahmad Z, Subramanyam L, Szczepaniak L, Simha V, Adams-Huet B, Garg A. Cholic acid for hepatic steatosis in patients with lipodystrophy: a randomized, controlled trial. Eur J Endocrinol. 2013 Apr 15;168(5):771-8. doi: 10.1530/EJE-12-0969. Print 2013 May.

  PMID: 23447519 RESULT

MeSH Terms

Conditions

Fatty Liver; Lipodystrophy

Interventions

Cholic Acid

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases; Skin Diseases, Metabolic; Skin Diseases; Skin and Connective Tissue Diseases; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Cholic Acids; Bile Acids and Salts; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Cholanes

Results Point of Contact

• Title: Zahid Ahmad, M.D.
• Organization: UT Southwestern Medical Center

zahid.ahmad@utsouthwestern.edu

214-648-2377

Study Officials

• Abhimanyu Garg, M.D.

  University of Texas Southwestern Medical Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: PI

Study Record Dates

• First Submitted: April 4, 2007
• First Posted: April 6, 2007
• Study Start: April 1, 2006
• Primary Completion: April 1, 2011
• Study Completion: April 1, 2011
• Last Updated: August 6, 2025
• Results First Posted: January 15, 2019

Record last verified: 2025-08

Locations

US (1)