Growth Hormone Study in Adults With Prader-Willi Syndroom
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 25
- 试验地点
- 1
- 主要终点
- Change in lean body mass
研究概览
简要总结
The overall objective of this study is to measure the effect of growth hormone (GH) treatment on physical and psychosocial health in adults with Prader-Willi syndrome. Adults with PWS who have not been treated with GH during the past three years and who will start with GH treatment as part of regular patient care will be asked for informed consent to participate in this open-label prospective cohort study. We hypothesize that growth hormone treatment will improve the physical and psychosocial health.
详细描述
OBJECTIVES:
To measure the effect of GHt on physical and psychosocial health in adults with PWS.
The primary endpoint is change in lean body mass (LBM (kg)) as assessed by Dual Energy X-ray Absorptiometry (DEXA) scan. Secondary endpoints are total fat mass, bone density, physical health and psychosocial health. Also the occurrence of side-effects will be assessed. Only data that are collected as part of regular patient care will be used.
STUDY DESIGN:
Open-label prospective cohort study.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The patient is diagnosed with Prader-Willi syndrome (genetically confirmed)
排除标准
- •Non cooperative behaviour
- •Pregnancy
- •Known malignancies
- •Poorly controlled diabetes (HbA1c > 64 mmol/mol (8%))
- •Untreated obstructive sleep apnea (apnea-hypopnea index > 5)
- •Body mass index above 40 kg/m2
- •Upper-airway obstruction of any cause
结局指标
主要结局
Change in lean body mass
时间窗: 36 months
Change in lean body mass (in kg) as measured by Dual Energy X-ray Absorptiometry scan
次要结局
- Change in fat mass(36 months)
- Change in bone density(36 months)
- Change in physical strength(36 months)
- Change in laboratory measurements(36 months)
- Change in psychosocial functioning(36 months)
- Change in caregiver burden(36 months)
研究者
dr. Laura C. G. de Graaff-Herder
MD, PhD
Erasmus Medical Center