Duchenne Muscular Dystrophy Video Assessment Registry
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 150
- 试验地点
- 1
- 主要终点
- Duchenne Video Assessment (DVA)
研究概览
简要总结
ARISE is a prospective and longitudinal clinical study of individuals with Duchenne Muscular Dystrophy (DMD) aimed at creating a database of functional motor abilities in this population to support validation efforts of the Duchenne Video Assessment (DVA).
详细描述
ARISE is a longitudinal, observational study of individuals with Duchenne muscular dystrophy (DMD). The Duchenne Video Assessment (DVA) is a home-based clinical outcome assessment that measures ease of movement through identification of compensatory movement patterns. The DVA directs caregivers to video record patients doing specific movement activities at home using a secure mobile application. In this study, participants will remotely complete the DVA in addition to existing DMD functional assessments (North Star Ambulatory Assessment (NSAA) and Performance of Upper Limb (PUL 2.0)) to evaluate the longitudinal measurement properties of the DVA.
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
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •At least 2 years of age at time of consent
- •Written consent (English only) by adult participant or parent/legal guardian of minor participant
- •Written assent (English only) if minor participant is at least 7 years of age
- •Documentation provided for the participant's diagnosis of DMD (i.e., genetic report, clinic note)
排除标准
- •Participants will be excluded if they are unable to use a hand to hold a pen, pick up pennies or drive a powerchair.
结局指标
主要结局
Duchenne Video Assessment (DVA)
时间窗: 24 months
The DVA is a home-based clinical outcome assessment that measures ease of movement through identification of compensatory movement patterns. The DVA includes 18 standardized movement tasks that are assigned to participants based on functional subgroup. The DVA directs and trains caregivers to video record patients doing specific movement tasks at home using a secure mobile application. DVA videos are scored by DVA-certified physical therapists using validated scorecards with prespecified compensatory movement criteria.
次要结局
- North Star Ambulatory Assessment (NSAA)(24 months)
- Performance of Upper Limb 2.0 (PUL 2.0)(24 months)
- Caregiver Task-Specific Impression of Change(24 months)