Clinical Outcomes Validation in Non Ambulatory and Young Boys/Men With Duchenne Muscular Dystrophy (DMD)
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 50
- 试验地点
- 1
- 主要终点
- Dystrophin gene mutations that predispose to early onset cardiomyopathy
研究概览
简要总结
Many therapeutic trials in DMD exclude non-ambulatory boys and men. Rate of progression in these non-ambulatory patients has been studied but consensus has not been reached for what measures are most reliable and reproducible. Furthermore, any treatment trial would be expected to demonstrate improved function and improvement in quality of life. Therefore, function, strength, and quality of life must be understood and standardized. While the goal of this proposal is to standardize clinical outcomes for therapeutic trials, careful understanding of the progression of DMD in non ambulatory boys may also lead to better medical treatment.
详细描述
For Non-ambulatory study, boys must be unable to walk without assistive devices for greater than or equal to one year.
Age at onset of study for non-ambulatory boys and men: greater than or equal to 7 years through age 22 years.
Genetic or biopsy confirmation of dystrophinopathy or clinical diagnosis of DMD with biopsy/genetic confirmation in a primary relative.
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
- 7 Years 至 22 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •For Non-ambulatory study, boys must be unable to walk without assistive devices for greater than or equal to one year.
- •Age at onset of study for non-ambulatory boys and men: greater than or equal to 7 years through age 22 years.
- •Genetic or biopsy confirmation of dystrophinopathy or clinical diagnosis of DMD with biopsy/genetic confirmation in a primary relative.
排除标准
- •For Non-ambulatory study, the inability to understand and cooperate with the testing would exclude a subject. -
结局指标
主要结局
Dystrophin gene mutations that predispose to early onset cardiomyopathy
时间窗: 3 years
次要结局
未报告次要终点
研究者
Anne Connolly
Professor of Neurology
Washington University School of Medicine