Double-Blind, Placebo-Controlled Trial of Leptin Replacement Therapy in Patients With Lipodystrophy
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 25
- 试验地点
- 1
- 主要终点
- Fasting Serum Triglycerides
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 6 Years 至 70 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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:
- •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.
排除标准
- •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.
研究组 & 干预措施
1 Leptin
Active Comparator for 4 months, then for 8 months.
干预措施: Leptin (Drug)
2 Sugar pill
Placebo for 4 months, then active comparator for 8 months.
干预措施: Placebo (Drug)
结局指标
主要结局
Fasting Serum Triglycerides
时间窗: 4 months
HbA1c
时间窗: 4 months
次要结局
- Fasting Serum Glucose(4 months)
- Body Weight(4 months)
研究者
Abhimanyu Garg
Chairman, Division Nutrition and Metabolic Diseases, Professor Internal Medicine
University of Texas Southwestern Medical Center