Nordic Study on the Effects of Growth Hormone (Norditropin SimpleXx) Treatment in Adults With Prader-Willi Syndrome
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 46
- 试验地点
- 3
- 主要终点
- Changes in body composition (lean body mass and fat mass) measured by dual energy X-ray absorptiometry (DXA)
研究概览
简要总结
The aim of this study is to study the effects of GH on body composition, lipid and glucose metabolism, physical performance and safety aspects in adults with PWS.The patients are randomized to either GH or placebo the first year of the study, subsequently followed by two years of GH treatment. the study is performed in Norway, Sweden and Denmark.
详细描述
OBJECTIVE(S):
Prader Willi syndrome (PWS) is a multi-symptomatic genetic disorder associated with abnormalities in the growth hormone (GH)-insulin-like-growth factor (IGF)-I axis and in the body composition. GH treatment is a registered indication in children with PWS, and improves growth rate and body composition. One pilot study in adult patients with clinical PWS has shown beneficial effects on body composition without simultaneous significant side effects. The aim of the present study is to evaluate the effects of GH treatment on body composition, muscle function and quality of life in PWS adults.
TRIAL DESIGN:
The study will be an investigator initiated and investigator sponsored multinational and multi-centre trial, including centres in Norway, Sweden and Denmark. Within each centre patients will be randomised (double blind) to one year treatment with daily injections of GH or placebo (efficacy), followed by a two year observation period on GH treatment (safety).
TRIAL POPULATION:
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
- 18 Years 至 50 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Genetically verified PWS diagnosis (by methylation and FISH test.)
- •Between 18 and 50 years old
- •Informed consent obtained before any trial-related activities. (Trial-related activities are any procedure that would not have been performed during normal management of the treatment of the subject.)
排除标准
- •Known or suspected allergy to GH preparation.
- •Previous participation in this trial.
- •GH treatment within the last 1 years
- •Malignancy or other serious diseases (ex severe cardiovascular diseases, severe infections)
- •Sexhormone treatment initiated within the last year
- •Pregnancy
- •Untreated respiratory impairment, untreated sleep apnoea or untreated respiratory infection.
研究组 & 干预措施
Norditropin SimpleXx
0.3 mg/day or 0.4 mg/day if bodyweight was below or above 100 kg,for 4 weeks, 0.6 mg/day or 0.8 mg/day, for 11 months.
干预措施: Norditropin SimpleXx (Drug)
Placebo
Placebo for 12 months
干预措施: Norditropin SimpleXx (Drug)
结局指标
主要结局
Changes in body composition (lean body mass and fat mass) measured by dual energy X-ray absorptiometry (DXA)
时间窗: 36 months
次要结局
- Standard photography appearance according to visual analogue scale (VAS)(36 months)
- Effects on free and total IGF-I, IGF-binding protein (BP)-1 and 3(36 months)
- Effects on lipids (fasting triglycerides(TG), total, HDL and LDL cholesterol)(36 months)
- Effects on body composition measured with bioimpedance(36 months)
- Activity of daily living measured a.m. Guralnik(36 months)
- Bone mineral density measured by DXA(36 months)
- Effects on forced expiratory volume (Peakflow)(36 months)
- Effects on haemoglobin (Hb), leucocyte and thrombocyte counts, FSH, LH, estradiol, Testosterone, inhibin B, TSH and Thyroxine(36 months)
- Muscle and fat mass measured by abdominal and mid-femoral computerized tomography(36 months)
- Quality of life estimated by questionnaires(36 months)
研究者
Charlotte Hoeybye
MD, PhD
Karolinska University Hospital