Determining Biomechanical and Morphological Factors That Affect Children With Duchenne Muscular Dystrophy (DMD) Who Loss of the Ability to Walk
试验速览
- 阶段
- 不适用
- 入组人数
- 50
- 试验地点
- 15
- 主要终点
- Determine biomechanical and morphological predictive factors of the loss of the walk ability of the children with DMD
研究概览
简要总结
The loss of ability to walk in many children with DMD (Duchenne muscular Dystrophy) is a pejorative event. Biomechanical and morphological unknowledge about the loss of the walk ability in children with DMD is an obstacle in reeducative, pharmacological or surgical therapeutic targets.
详细描述
The loss of ability to walk in many children with DMD (Duchenne muscular Dystrophy) is a pejorative event. Biomechanical and morphological unknowledge about the loss of the walk ability in children with DMD is an obstacle in reeducative, pharmacological or surgical therapeutic targets. We suppose that there are muscular characteristics and predictive parameters of the loss of walk ability. The identification of these potential therapeutic targets would improve the surveillance and the clinical care but would also guide future clinical and fundamental trials too.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- 5 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Young man ou woman (5 to 17 years old) with Duchenne Muscular Dystrophy (confirmed by immunohistochimy on the muscular biopsy and/or mutation in the dystrophin confirmed by molecular biology)
- •Time more than 7 secondes to test of 10 m and/or distance less than 330 m to walk test of 6 minutes. These values are recent markers to include children with a strong risk of loss of walking ability in 2 years.
- •Parental inform sign consent and / or child inform consent
排除标准
- •Recent orthopaedic surgery of lower limbs (6 months)
- •Other chronic disease associated, which have an impact on the walking
- •Cognitive Deficiency or behavior disorders limiting the understanding of the study
- •Children who can benefit ATU (translarna ® or other) during the study
- •All MRI contradications : pacemaker or neurosensory stimulator or implantable defibrillator, neurosurgical valves, cochlear implant or ferromagnetic implants near nervous structures, brace, metallic prostheses, not cooperative or agitated patients, patient claustrophobic, pregnant woman.
研究组 & 干预措施
Duchenne muscular dystrophy children
No drug and no placebo were used in this study. For 2h30 (time)
- Measurement of leg strength using a dynamometer
- Measurement of Motor Function
- Walk test 6 minutes
- Walk test 10 meters
- Walk analysis: 3D recording of walking
- Muscle MRI
干预措施: No drug and no placebo were used in this study (Other)
Healthy children
No drug and no placebo were used in this study. For 2h30 (time)
- Measurement of leg strength using a dynamometer
- Measurement of Motor Function
- Walk test 6 minutes
- Walk test 10 meters
- Walk analysis: 3D recording of walking
- Muscle MRI
干预措施: No drug and no placebo were used in this study (Other)
结局指标
主要结局
Determine biomechanical and morphological predictive factors of the loss of the walk ability of the children with DMD
时间窗: 24 years
Determine the biomechanical factors (muscular atrophy, muscular strength, muscular shrinkage) and morphological (greasy infiltration, contractile portion, muscular geometry) predictive of the loss of the walk ability of the children with DMD
次要结局
- Identify the muscles wasting and their implication in the loss of strength and the walking ability(24 years)
- Biomechanical evolutionary data collected during the last 2 years of walking(24 years)
- Establish the relationship between the parameters of walking and the scrawny body morphological anomalies(24 years)