Effect of Creatine Monohydrate on Functional Muscle Strength and Muscle Mass in Children With FSHD: a Multi-centre, Randomised, Double-blind Placebo-controlled Crossover Trial
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 11
- 试验地点
- 1
- 主要终点
- Motor Function Measure for Neuromuscular disease
研究概览
简要总结
This multi-centre, randomised, double-blind, placebo-controlled crossover trial will compare changes in strength-related motor function following treatment with creatine monohydrate to treatment with placebo, as measured by the Motor Function Measure, from baseline to 12 weeks. Eligible subjects will undergo baseline assessments then will be randomised to either creatine monohydrate therapy or placebo for three months, followed by a six week wash-out period, then crossover to a further three months of therapy with either placebo or creatine. Subjects will undergo clinical assessments and study safety assessments at the beginning and end of each treatment period. The study will begin recruitment in early 2017.
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
- 5 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Is between the ages of 5 and 18 years inclusive at the time of randomisation;
- •Has a confirmed genetic diagnosis of Facioscapulohumeral Muscular Dystrophy (FSHD) types 1 or 2;
- •Has a legally acceptable representative capable of understanding the informed consent document and providing consent on the participant's behalf.
排除标准
- •Has clinically significant elevation in plasma creatinine level or unexplained hypertension at screening;
- •Has a prior diagnosis of chronic renal failure;
- •Has a known hypersensitivity to creatine monohydrate of maltodextrin placebo;
- •Patients already taking any medications to increase muscle bulk or strength or concomitant use of regular sodium valproate, corticosteroids of alpha agonists such as salbutamol.
研究组 & 干预措施
Group A - Active/Placebo
Participants will receive 3 months of creatine monohydrate followed by a 6 week washout period followed by 3 months of placebo.
干预措施: Placebo (Dietary Supplement)
Group B - Placebo/Active
Participants will receive 3 months of placebo followed by a 6 week washout period followed by 3 months of creatine monohydrate.
干预措施: Placebo (Dietary Supplement)
Group A - Active/Placebo
Participants will receive 3 months of creatine monohydrate followed by a 6 week washout period followed by 3 months of placebo.
干预措施: Creatine Monohydrate (Dietary Supplement)
Group B - Placebo/Active
Participants will receive 3 months of placebo followed by a 6 week washout period followed by 3 months of creatine monohydrate.
干预措施: Creatine Monohydrate (Dietary Supplement)
结局指标
主要结局
Motor Function Measure for Neuromuscular disease
时间窗: 3 months
Composite functional outcome measure
次要结局
- Muscle Ultrasound Scan(3 months)
- PedsQL Neuromuscular(3 months)
- Quantitative muscle strength testing(3 months)
- GPX3 Level(3 months)
- FSH-COM(3 months)
- FSH-Health Index (Pediatric Version)(3 months)
- FSHD Severity Score(3 months)
- Laboratory safety monitoring(3 months)
- Six Minute Walk Test(3 months)
- Step Counter(3 months)
- Muscle Magnetic Resonance Imaging(3 months)
- Performance of the Upper Limb Measure(3 months)
- ACTIVILIM(3 Months)