Trial of Leptin Replacement Therapy in Patients With Lipodystrophy

Study Description

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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.

Condition or disease	Intervention/treatment	Phase
Hypoleptinemia; Generalized Lipodystrophy; Partial Lipodystrophy; Insulin Resistance	Drug: Leptin; Drug: Placebo	Phase 2; Phase 3

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 Design

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Study Type :	Interventional (Clinical Trial)
Estimated Enrollment :	24 participants
Allocation:	Randomized
Intervention Model:	Crossover Assignment
Masking:	Triple (Participant, Care Provider, Investigator)
Primary Purpose:	Treatment
Official Title:	Double-Blind, Placebo-Controlled Trial of Leptin Replacement Therapy in Patients With Lipodystrophy
Study Start Date :	April 2006
Actual Primary Completion Date :	November 2014
Actual Study Completion Date :	November 2014

Arms and Interventions

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Arm	Intervention/treatment
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

Outcome Measures

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Primary Outcome Measures :

1. Project Specific: to determine if r-metHuLeptin can be safely replaced in hypoleptinemic patients with generalized and partial lipodystrophies. [ Time Frame: 12 months ]

Secondary Outcome Measures :

1. 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 ]

Eligibility Criteria

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Ages Eligible for Study:	6 Years to 70 Years (Child, Adult, Senior)
Sexes Eligible for Study:	All
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

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Responsible Party:	Abhimanyu Garg, Chairman, Division Nutrition and Metabolic Diseases, Professor Internal Medicine, University of Texas Southwestern Medical Center
ClinicalTrials.gov Identifier:	NCT00896298 History of Changes
Other Study ID Numbers:	0502-294
First Posted:	May 11, 2009
Last Update Posted:	December 4, 2015
Last Verified:	December 2015

Keywords provided by Abhimanyu Garg, 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; Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn