Trial of Leptin Replacement Therapy in Patients With Lipodystrophy

Phase 2

Completed

• Conditions: Hypoleptinemia; Generalized Lipodystrophy; Partial Lipodystrophy; Insulin Resistance
• Interventions: Drug: Leptin; Drug: Placebo

• Registration Number: NCT00896298
• Lead Sponsor: University of Texas Southwestern Medical Center

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.

Study Timeline

• Study Start: 2006-04
• Study First Submitted: 2009-05-08
• Study First Submitted QC: 2009-05-08
• Study First Posted: 2009-05-11
• Primary Completion: 2014-11
• Study Completion: 2014-11
• Results First Submitted: 2019-01-15
• Results First Submitted QC: 2019-02-28
• Results First Posted: 2019-03-05
• Status Verified: 2024-03
• Last Update Submitted: 2024-03-25
• Last Update Posted: 2024-03-27

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 25

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
1 Leptin	Leptin	Active Comparator for 4 months, then for 8 months.
2 Sugar pill	Placebo	Placebo for 4 months, then active comparator for 8 months.

Primary Outcome Measures

Name	Time	Method
Fasting Serum Triglycerides	4 months
HbA1c	4 months

Secondary Outcome Measures

Name	Time	Method
Fasting Serum Glucose	4 months
Body Weight	4 months

Trial Locations

Locations (1)

UT Southwestern Medical Center; 🇺🇸 Dallas, Texas, United States