Clinical trial NCT00896298: Trial of Leptin Replacement Therapy in Patients With Lipodystrophy
Official title: Double-Blind, Placebo-Controlled Trial of Leptin Replacement Therapy in Patients With Lipodystrophy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Hypoleptinemia
Generalized Lipodystrophy
Partial Lipodystrophy
Insulin Resistance
Keywords: Lipodystrophy
Insulin secretory response
Lead sponsor: University of Texas Southwestern Medical Center
Sponsor class: OTHER
First posted: 2009-05-11
Enrollment: 25
Sex: ALL
Minimum age (years): 6
Maximum age (years): 70
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Fasting Serum Triglycerides
timeFrame: 4 months
measure: HbA1c
timeFrame: 4 months
Secondary outcomes:
measure: Fasting Serum Glucose
timeFrame: 4 months
measure: Body Weight
timeFrame: 4 months
Other outcomes:
Locations:
city: Dallas
country: United States
facility: UT Southwestern Medical Center
state: Texas
Study officials:
affiliation: UT Southwestern Medical Center
name: Abhimanyu Garg, MD
role: PRINCIPAL_INVESTIGATOR
References: