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Trial of Leptin Replacement Therapy in Patients With Lipodystrophy

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by:
University of Texas Southwestern Medical Center
ClinicalTrials.gov Identifier:
NCT00896298
First received: May 8, 2009
Last updated: August 2, 2011
Last verified: August 2011
History of Changes
  Purpose

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.


Condition Intervention Phase
Hypoleptinemia
Generalized Lipodystrophy
Partial Lipodystrophy
Insulin Resistance
 Drug: Leptin
Drug: Placebo
 Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, Investigator)
Primary Purpose: Treatment
Official Title: Double-Blind, Placebo-Controlled Trial of Leptin Replacement Therapy in Patients With Lipodystrophy

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by University of Texas Southwestern Medical Center:

Primary Outcome Measures:
  • Project Specific: to determine if r-metHuLeptin can be safely replaced in hypoleptinemic patients with generalized and partial lipodystrophies. [ Time Frame: 12 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Project Specific: to determine if r-metHuLeptin administration is effective in improving glucose and lipid abnormalities in hypoleptinemic patients with generalized and partial lipodystrophies. [ Time Frame: 12 months ] [ Designated as safety issue: No ]

Estimated Enrollment: 24
Study Start Date: April 2006
Estimated Study Completion Date: July 2012
Primary Completion Date: July 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: 1 Leptin
Active Comparator for 4 months, then for 8 months.
 Drug: Leptin
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 Comparator: 2 Sugar pill
Placebo for 4 months, then active comparator for 8 months.
 Drug: Placebo
Sugar pill

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

Ages Eligible for Study:   6 Years to 70 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00896298

Locations
United States, Texas
UT Southwestern Medical Center
Dallas, Texas, United States, 75390
Sponsors and Collaborators
University of Texas Southwestern Medical Center
Investigators
Principal Investigator: Abhimanyu Garg, MD UT Southwestern Medical Center
  More Information

No publications provided

Responsible Party: Dr. Abhimanyu Garg, UT Southwestern Medical Center
ClinicalTrials.gov Identifier: NCT00896298     History of Changes
Other Study ID Numbers: 0502-294
Study First Received: May 8, 2009
Last Updated: August 2, 2011
Health Authority: United States: Food and Drug Administration

Keywords provided by University of Texas Southwestern Medical Center:
Lipodystrophy
Insulin secretory response

Additional relevant MeSH terms:
Insulin Resistance
Lipodystrophy
Lipodystrophy, Congenital Generalized
Hyperinsulinism
Glucose Metabolism Disorders
 Metabolic Diseases
Skin Diseases, Metabolic
Skin Diseases
Lipid Metabolism Disorders

ClinicalTrials.gov processed this record on May 23, 2013