Double-Blind, Placebo-Controlled Trial of Leptin Replacement Therapy in Patients With Lipodystrophy
| Registry | NCT00896298 |
|---|---|
| Status | Completed |
| Phase | Phase 2 / Phase 3 |
| Study type | Interventional; randomized, parallel, masking: triple, treatment |
| Enrollment | 25 (Actual) |
| Sponsor | University of Texas Southwestern Medical Center (Other) |
| Conditions | Hypoleptinemia, Generalized Lipodystrophy, Partial Lipodystrophy, Insulin Resistance |
| Interventions | Leptin, Placebo (Drug) |
| Dates | start 2006-04-01, primary completion 2014-11-01, completion 2014-11-01, first posted 2009-05-11, last updated 2024-03-27 |
| Locations | 1 site in United States |
Related Papers Related Patents Related Grants Related Experts
All trials for Hypoleptinemia All trials for Generalized Lipodystrophy All trials of Leptin All trials of Placebo
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.
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.
| Arm | Type | Interventions | Description |
|---|---|---|---|
| 1 Leptin | Active comparator | Drug: Leptin | Active Comparator for 4 months, then for 8 months. |
| 2 Sugar pill | Placebo comparator | Drug: Placebo | Placebo for 4 months, then active comparator for 8 months. |
Sex: All, ages: 6 Years to 70 Years
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.
| Milestone | Leptin Therapy | Placebo |
|---|---|---|
| Started | 13 | 12 |
| Completed | 10 | 9 |
| Not completed | 3 | 3 |
Time frame: 4 month
| Arm | Deaths | Serious adverse events | Other adverse events |
|---|---|---|---|
| Leptin Therapy | 0/13 (0.0%) | 0/13 (0.0%) | 2/13 (15.4%) |
| Placebo | 0/12 (0.0%) | 0/12 (0.0%) | 4/12 (33.3%) |
| Term | Organ system | Leptin Therapy | Placebo |
|---|---|---|---|
| viral illness, sinusitis, backache | Infections and infestations | 1/13 | 0/12 |
| Headaches | General disorders | 1/13 | 0/12 |
| Pneumonia | Blood and lymphatic system disorders | 0/13 | 1/12 |
| Tonsillectomy | Surgical and medical procedures | 0/13 | 1/12 |
| Right Ankle Pain | Surgical and medical procedures | 0/13 | 1/12 |
| Blurry Vision and Epigastric pain | Eye disorders | 0/13 | 1/12 |
| Facility | City | Country | Status |
|---|---|---|---|
| UT Southwestern Medical Center | Dallas, Texas | United States |