Trial of Leptin Replacement Therapy in Patients With Lipodystrophy

The University of Texas System (UT)

Status and phase: Completed; Phase 3; Phase 2

Conditions: Generalized Lipodystrophy; Hypoleptinemia; Insulin Resistance; Partial Lipodystrophy

Treatments: Drug: Placebo; Drug: Leptin

Study type: Interventional

Funder types: Other

Identifiers

NCT00896298

0502-294

Details and patient eligibility

About

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.

Full 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.

Enrollment: 25 patients

Sex: All

Ages: 6 to 70 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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25 participants in 2 patient groups, including a placebo group

1 Leptin

Active Comparator group

Description:

Active Comparator for 4 months, then for 8 months.

Treatment:

Drug: Leptin

2 Sugar pill

Placebo Comparator group

Description:

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

Treatment:

Drug: Placebo