Clinical Trial: Leptin to Treat Lipodystrophy

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional



Official Title: Long-Term Efficacy of Leptin Replacement in Treatment of Lipodystrophy

Brief Summary:

This study will evaluate the safety and effectiveness of leptin replacement therapy in patients with lipodystrophy (also called lipoatrophy). Patients have a total or partial loss of fat cells. They also lack the hormone leptin, which is produced by fat cells. The leptin deficiency usually causes high blood lipid (fat) levels and insulin resistance that may lead to diabetes. Patients may have hormone imbalances, fertility problems, large appetite, and liver disease due to fat accumulation.

Patients age greater than or equal to 6 months with significant lipodystrophy may be eligible for this study. Participants will be admitted to the NIH Clinical Center for 10 days for the following studies before beginning 12 months of leptin therapy:

  • Insulin tolerance test
  • Ultrasound of the liver and, if abnormalities are found, possibly liver biopsies.
  • Fasting blood tests
  • Resting metabolic rate
  • Magnetic resonance imaging of the liver and other organs, and of muscle and fat.
  • Pelvic ultrasound in women to detect ovarian cysts.
  • Estimation of body fat
  • Oral glucose tolerance test
  • Intravenous glucose tolerance test
  • Appetite level and food intake
  • Hormone function tests
  • Questionnaires to assess activity and mood
  • 24-hour urine collections

Additional studies may include blood tests for genetic studies of lipodystrophy, a muscle biopsy to study mus