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Completed

NCT Number: NCT00896298

Trial of Leptin Replacement Therapy in Patients With Lipodystrophy

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.

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Key information

Conditions

Hypoleptinemia Congenital, Hereditary, and Neonatal Diseases and Abnormalities Generalized Lipodystrophy Genetic Diseases, Inborn Glucose Metabolism Disorders Hyperinsulinism Insulin Resistance Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipodystrophy Lipodystrophy, Congenital Generalized Lipodystrophy, Partial, Acquired Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases Partial Lipodystrophy Skin Diseases Skin Diseases, Metabolic Skin and Connective Tissue Diseases

Age range

6 year–70 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

UT Southwestern Medical Center

Dallas, Texas, 75390, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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:
  • Type 2 Diabetes Mellitus
  • Fasting serum insulin >20 uU/mL
  • Fasting serum triglycerides > 300 mg/dL
  • 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.

Treatment and study plan

Leptin

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.

Other names: Recombinant-methionyl Human Leptin., r-metHuLeptin

Placebo

Drug

Sugar pill

Primary outcomes

  1. Fasting Serum Triglycerides

    Time frame: 4 months

  2. HbA1c

    Time frame: 4 months

Secondary outcomes

  1. Fasting Serum Glucose

    Time frame: 4 months

  2. Body Weight

    Time frame: 4 months

Sponsors and collaborators

Lead sponsor

University of Texas Southwestern Medical Center

Other

Registry information

Official study title

Double-Blind, Placebo-Controlled Trial of Leptin Replacement Therapy in Patients With Lipodystrophy

Important dates

Study start
2006
Primary completion
2014
Study completion
2014
First posted
May 11, 2009
Registry last updated
Mar 27, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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