Can Treatment With Human Growth Hormone Increase Strength in Spinal Muscular Atrophy Type II and III?
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 20
- 试验地点
- 3
- 主要终点
- Primary: sum of strength (hand held myometry)
研究概览
简要总结
The purpose of this study is to determine whether Growth hormon can increase strength in spinal muscular atrophy type II and III.
详细描述
This pilot study is planned as a placebo-controlled cross-over trial in a limited number of patients. It is the aim to investigate the effect of GH on the short-term changes of strength and to investigate the tolerability of the treatment. If the results of the study are positive, further studies of longer duration addressing the development of motor function and quality of life could follow
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 6 Years 至 35 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •genetically confirmed diagnosis of Spinal Muscular Disease
- •Spinal muscular atrophy type II or III
- •age between 6 years and 35 years
- •ability to perform the tests for measurement of muscle strength (handheld myometry)
- •informed consent of the patient and/or parents
排除标准
- •pregnancy or lactation
- •woman with child bearing potential without contraception
- •overweight or BMI over 30 kg/m²
- •Treatment with other drugs, that can influence strength 8 weeks before participation in the study or during participation
- •medical history or evidence of a malignant or cerebral tumor
- •cardiovascular, intestinal, endocrinologically or airway disease
- •Hypertension
- •growth hormone deficiency
- •hypersensitivity to one component part of the study medication
- •participation on a clinical trial during the study or 3 month before
- •abuse to drugs or alcohol
- •patient incapable of contracting or not able to understand the character, meaning and consequences of the clinical trial
研究组 & 干预措施
Somatotropin
subcutaneous application of somatotropin over 12 weeks followed by 8 weeks wash out period followed by 12 weeks subcutaneous placebo application
干预措施: somatotropin (Drug)
Placebo
12 weeks placebo subcutaneous application followed by 8 weeks wash out and 12 weeks subcutaneous application of somatotropin
干预措施: Placebo (Drug)
结局指标
主要结局
Primary: sum of strength (hand held myometry)
时间窗: 20 weeks
次要结局
- Functional (time) tests, lung function, quality of life,(20 weeks)
研究者
Janbernd Kirschner
MD
University Hospital Freiburg