Novel Therapies for Metabolic Complications of Lipodystrophies

NCT ID: NCT00457938

Last Updated: 2018-10-16

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 4 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2006-04-30
• Study Completion Date: 2014-11-30

Brief Summary

Lipodystrophies represent a therapeutic challenge with regards to the management of the diabetes, insulin resistance, hypertriglyceridemia and fatty liver which frequently present in conjunction with significant adipose tissue loss. The purpose of the study and it's four subprojects is to examine the safety and efficacy of various novel interventions designed to improve or resolve the fatty liver, hypertriglyceridemia, and insulin resistance or diabetes that is seen in these patients.

Detailed Description

We propose novel therapeutic approaches for the management of metabolic complications in patients with lipodystrophies. The four interventions to be tested are:

Hypothesis 1: An extremely low fat diet.

Conditions

Insulin Resistance; Hypertriglyceridemia; Diabetes; Hepatic Steatosis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

Low fat diet is the "drug"

Diet 10% fat versus 35% fat

Group Type: OTHER

low-fat diet ( Still recruiting )

Intervention Type: OTHER

This study will compare 10% fat versus 35% fat diets in terms if effect on liver fat, triglycerides adn other metabolic parameters.

Diet

Intervention Type: OTHER

10 % versus 35 % fat in diet

Interventions

low-fat diet ( Still recruiting )

This study will compare 10% fat versus 35% fat diets in terms if effect on liver fat, triglycerides adn other metabolic parameters.

Intervention Type: OTHER

Diet

10 % versus 35 % fat in diet

Intervention Type: OTHER

Other Intervention Names

Low fat versus extremely low fat Diet

Eligibility Criteria

Inclusion Criteria

• Patients with lipodystrophies as diagnosed by clinical criteria
• Any one of the following:

• Diabetes mellitus, or
• Fasting serum triglycerides greater than 200 mg/dL, or
• Fasting serum insulin greater than 30 U/mL, or
• Hepatic steatosis ( greater than 5.6% hepatic triglyceride content) as demonstrated by 1H MRS.

Exclusion Criteria

• Known liver disease due to causes other than non-alcoholic steatohepatitis:
• Current alcohol abuse (more than 7 drinks or 210 g per wk for women and more than 14 drinks or 420 g per wk for men).
• Positive serological markers of hepatitis B and C.
• Autoimmune hepatitis, autoimmune cholestatic liver disorders, Wilson disease and Alpha-1-antitrypsin deficiency as indicated by clinical and laboratory tests.
• Drug-induced liver disease
• Evidence of hepatocellular carcinoma: alpha-fetoprotein levels greater than 200 ng/ml and/or liver mass on imaging study suggestive of liver cancer.
• 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.)
• Use of the drugs which can potentially decrease hepatic steatosis during previous 3 months; high-dose vitamin E, betaine, acetylcysteine, choline and probucol.
• Significant systemic or major illnesses other than liver disease (congestive heart failure, unstable angina, cerebrovascular disease, respiratory failure, renal failure [serum creatinine more then 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 ( more than 10%) or use of weight loss drugs such as sibutramine or orlistat in the last 3 months.

• Minimum Eligible Age: 14 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Institutes of Health (NIH)

NIH

Sponsor Role: collaborator

Takeda

INDUSTRY

Sponsor Role: collaborator

Amylin Pharmaceuticals, LLC.

INDUSTRY

Sponsor Role: collaborator

University of Texas Southwestern Medical Center

OTHER

Sponsor Role: lead

Responsible Party

Abhimanyu Garg

Chairman, Division Nutrition and Metabolic Diseases, Professor Internal Medicine

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Abhimanyu Garg, MD

Role: PRINCIPAL_INVESTIGATOR

UT Southwestern Medical Center

Locations

UT Southwestern Medical Center

Dallas, Texas, United States

Countries

United States

References

Simha V, Subramanyam L, Szczepaniak L, Quittner C, Adams-Huet B, Snell P, Garg A. Comparison of efficacy and safety of leptin replacement therapy in moderately and severely hypoleptinemic patients with familial partial lipodystrophy of the Dunnigan variety. J Clin Endocrinol Metab. 2012 Mar;97(3):785-92. doi: 10.1210/jc.2011-2229. Epub 2011 Dec 14.

Reference Type: DERIVED

PMID: 22170723 (View on PubMed)

Other Identifiers

DK074959

Identifier Type: -

Identifier Source: secondary_id

RO1-074959

Identifier Type: -

Identifier Source: org_study_id