Trial of Leptin Replacement Therapy in Patients With Lipodystrophy
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ClinicalTrials.gov Identifier: NCT00896298 |
Recruitment Status :
Completed
First Posted : May 11, 2009
Results First Posted : March 5, 2019
Last Update Posted : April 2, 2019
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Condition or disease | Intervention/treatment | Phase |
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Hypoleptinemia Generalized Lipodystrophy Partial Lipodystrophy Insulin Resistance | Drug: Leptin Drug: Placebo | Phase 2 Phase 3 |
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 Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 25 participants |
Allocation: | Randomized |
Intervention Model: | Parallel 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 |

Arm | Intervention/treatment |
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Active Comparator: 1 Leptin
Active Comparator for 4 months, then for 8 months.
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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:
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Placebo Comparator: 2 Sugar pill
Placebo for 4 months, then active comparator for 8 months.
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Drug: Placebo
Sugar pill |
- Fasting Serum Triglycerides [ Time Frame: 4 months ]
- HbA1c [ Time Frame: 4 months ]
- Fasting Serum Glucose [ Time Frame: 4 months ]
- Body Weight [ Time Frame: 4 months ]

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Ages Eligible for Study: | 6 Years to 70 Years (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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)
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Presence of at least one of the following metabolic abnormalities:
- Type 2 Diabetes Mellitus
- Fasting serum insulin >20 uU/mL
- Fasting serum triglycerides > 300 mg/dL
- 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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00896298
United States, Texas | |
UT Southwestern Medical Center | |
Dallas, Texas, United States, 75390 |
Principal Investigator: | Abhimanyu Garg, MD | UT Southwestern Medical Center |
Responsible Party: | Abhimanyu Garg, Chairman, Division Nutrition and Metabolic Diseases, Professor Internal Medicine, University of Texas Southwestern Medical Center |
ClinicalTrials.gov Identifier: | NCT00896298 |
Other Study ID Numbers: |
0502-294 |
First Posted: | May 11, 2009 Key Record Dates |
Results First Posted: | March 5, 2019 |
Last Update Posted: | April 2, 2019 |
Last Verified: | March 2019 |
Lipodystrophy Insulin secretory response |
Lipodystrophy, Congenital Generalized Lipodystrophy Insulin Resistance Hyperinsulinism Glucose Metabolism Disorders Metabolic Diseases |
Skin Diseases, Metabolic Skin Diseases Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Metabolism, Inborn Errors Genetic Diseases, Inborn |