Growth Hormone Use in Adults With Prader-Willi Syndrome
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 11
- 试验地点
- 1
- 主要终点
- Body Composition
研究概览
简要总结
The main research question this protocol aims to answer is whether treatment with growth hormone will impact body composition, quality of life, and energy balance in PWS adults, and if there is a loss of effects after cessation of treatment for at least 12 months.
详细描述
The main research question this protocol aims to answer is whether treatment with growth hormone will impact body composition, quality of life, and energy balance in PWS adults, and if there is a loss of effects after cessation of treatment for at least 12 months. Specific outcomes to be evaluated are as follows:
• Increased IGF-1 as a function of human growth hormone dosage compared with baseline.
- Improvement of indicators or risk factors for co-morbid diseases [diabetes (by measuring insulin and glucose levels), cardiovascular disease (by measuring lipids and fatty acids), and pulmonary function] in participants.
- Improvement in quality of life measures as indicated by ratings on established behavior checklists in participants.
- Changes in body composition (decreased fat, increased lean body mass and bone density) as determined by DEXA in participants.
- Increased energy expenditure as determined by whole-room calorimeter measures (8 hour energy expenditure, RMR, TEF, mechanical work); diet records, physical activity monitors and strength measures.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Diagnostic
- 盲法
- None
入排标准
- 年龄范围
- 16 Years 至 60 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •16 to 60 years old
- •Male or female with diagnosed Prader-Willi syndrome confirmed by genetic testing (e.g., mPCR)
- •Low IGF-1 level (e.g.,≤25%) at baseline
- •Ability to provide informed consent or availability of a suitable legally authorized representative
排除标准
- •Pregnancy
- •Previous treatment with growth hormone
- •Uncontrolled endocrine disease, (i.e. diabetes or thyroid)
- •History of severe scoliosis
- •Heart disease
- •Uncontrolled high blood pressure or history of stroke
- •Morbid obesity (using PWS growth charts)
- •Severe sleep apnea or known breathing difficulties/obstruction (as per history or diagnostic testing results
研究组 & 干预措施
Primary Cohort
Nutropin AQ
干预措施: Nutropin AQ (Drug)
结局指标
主要结局
Body Composition
时间窗: 12 and 24 months
Anthropometric Measures and Body Composition
Blood Chemistry
时间窗: 12 and 24 months
Insulin-like growth factor I- IGF-1
Physical Activity
时间窗: 12 and 24 months
Duration of daily physical activity
次要结局
未报告次要终点