NCT01385917Unknown不适用
Study of Clinical and Radiological Changes in Patients With Duchenne Muscular Dystrophy Theoretically Treatable With Exon 53 Skipping
适应症
试验速览
- 阶段
- 不适用
- 发起方
- Genethon
- 入组人数
- 45
- 试验地点
- 2
- 主要终点
- PreU7-53 is a natural history study
研究概览
简要总结
PreU7-53 is a natural history study. The objective is to monitor the clinical and radiological course of upper limb muscle impairment in patients with Duchenne Muscular Dystrophy (DMD), potentially treatable with AAV-mediated exon 53 skipping.
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
- 12 Years 至 20 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of Duchenne muscular dystrophy confirmed by at least genetic testing, theoretically treatable by exon 53 skipping.
- •Age between ≥ 12 and <20 years old.
- •Non ambulant patients (i;e; inability to walk more than 10 meters without any of assistance).
- •Patients covered by a national health insurance scheme.
- •Signed informed consent.
排除标准
- •Patient incapable of sitting upright in a wheelchair for at least one hour.
- •Patients with severe intellectual impairment preventing them from fully understanding the exercises to be performed.
- •Recent (less than 6 months ago) upper limb surgery or trauma This criteria is however no definitive. Patients who have undergone upper limb surgery or trauma may nonetheless be enrolled once the 6 month period is over.
- •Known immune deficiency.
- •Contraindications to NMR exams
结局指标
主要结局
PreU7-53 is a natural history study
时间窗: Every year
The objective is to monitor the clinical and radiological course of upper limb muscle impairment in patients with DMD, potentially treatable with AAV-mediated exon 53 skipping.
次要结局
未报告次要终点
研究者
研究点 (2)
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