This trial is active, not recruiting.

Conditions hypoleptinemia, generalized lipodystrophy, partial lipodystrophy, insulin resistance
Treatments leptin, placebo
Phase phase 2/phase 3
Sponsor University of Texas Southwestern Medical Center
Start date April 2006
End date July 2011
Trial size 24 participants
Trial identifier NCT00896298, 0502-294


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.

United States No locations recruiting
Other countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model crossover assignment
Masking double blind (subject, caregiver, investigator)
Primary purpose treatment
(Active Comparator)
Active Comparator for 4 months, then for 8 months.
leptin Recombinant-methionyl Human 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.
(Placebo Comparator)
Placebo for 4 months, then active comparator for 8 months.
Sugar pill

Primary Outcomes

Project Specific: to determine if r-metHuLeptin can be safely replaced in hypoleptinemic patients with generalized and partial lipodystrophies.
time frame: 12 months

Secondary Outcomes

Project Specific: to determine if r-metHuLeptin administration is effective in improving glucose and lipid abnormalities in hypoleptinemic patients with generalized and partial lipodystrophies.
time frame: 12 months

Eligibility Criteria

Male or female participants from 6 years up to 70 years old.

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.

Additional Information

Official title Double-Blind, Placebo-Controlled Trial of Leptin Replacement Therapy in Patients With Lipodystrophy
Principal investigator Abhimanyu Garg, MD
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.
Trial information was received from ClinicalTrials.gov and was last updated in June 2013.
Information provided to ClinicalTrials.gov by University of Texas Southwestern Medical Center.