Trial of Leptin Replacement Therapy in Patients With Lipodystrophy

NCT ID: NCT00896298

Last Updated: 2024-03-27

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 25 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 insulin resistance, hypertriglyceridemia and fatty liver which often is coupled with significant adipose tissue loss. The purpose of the study is to examine the safety and efficacy of Leptin on subjects with lipodystrophy.

Detailed Description

The mechanism by which leptin improves glucose and lipid control is not clear. We will examine the possible mechanisms of leptin action by studying the effects of leptin administration on food intake, insulin resistance, insulin secretory response, hepatic and intramuscular triglyceride stores in a large sample of patients with lipodystrophy.

Hypothesis: Leptin replacement in patients with generalized and partial lipodystrophy and hypoleptinemia will be safe and efficacious in improving the metabolic abnormalities associated with insulin resistance.

Conditions

Hypoleptinemia; Generalized Lipodystrophy; Partial Lipodystrophy; Insulin Resistance

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

1 Leptin

Active Comparator for 4 months, then for 8 months.

Group Type: ACTIVE_COMPARATOR

Leptin

Intervention Type: DRUG

Leptin injected subcutaneously twice a day, to be administered at 100% of the estimated replacement dose during the first month, which is a dosage of 0.03mg/kg for female children, 0.04mg/kg for adult females, and0.02 mg/kg for all males, and then at 200% subsequently, or placebo.

2 Sugar pill

Placebo for 4 months, then active comparator for 8 months.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Sugar pill

Interventions

Leptin

Leptin injected subcutaneously twice a day, to be administered at 100% of the estimated replacement dose during the first month, which is a dosage of 0.03mg/kg for female children, 0.04mg/kg for adult females, and0.02 mg/kg for all males, and then at 200% subsequently, or placebo.

Intervention Type: DRUG

Placebo

Sugar pill

Intervention Type: DRUG

Other Intervention Names

Recombinant-methionyl Human Leptin.; r-metHuLeptin

Eligibility Criteria

Inclusion Criteria

• age > 6 years
• Partial and generalized lipodystrophy
• Serum leptin levels less than the 7th percentile of normal values reported by the 3rd National Health and Nutrition Examination survey (less than 7.0 ng/mL in females and less than 3/0 ng/mL in males)
• Presence of at least one of the following metabolic abnormalities:

1. Type 2 Diabetes Mellitus

2. Fasting serum insulin >20 uU/mL

3. Fasting serum triglycerides > 300 mg/dL

4. Previous participation in leptin trial (Amgen 991265, GCRC 660) - for Part B of the Study.

Exclusion Criteria

• Known liver disease due to causes other than non-alcoholic steatohepatitis.
• Hematocrit of less than 30%.
• Current alcohol or substance abuse.
• Use of anorexigenic drugs, anabolic steroids, GH and thiazolidinediones
• Active tuberculosis
• Psychiatric disorder impeding competence or compliance
• Malignancies
• HIV infection
• Subjects who have a known hypersensitivity to E. Coli derived proteins
• Other condition, which in the opinion of the clinical investigators would impede completion of the study.

• Minimum Eligible Age: 6 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

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

Other Identifiers

0502-294

Identifier Type: -

Identifier Source: org_study_id