Development of the Mitochondrial Video Assessment (MVA) as an Outcome Measure for the Evaluation of Patients With Mitochondrial Myopathies
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 28
- 试验地点
- 1
- 主要终点
- Source Material Videos
研究概览
简要总结
Source material collection of videos to develop the Mitochondrial Video Assessment (MVA) for patients with mitochondrial myopathies.
详细描述
This observational research is being conducted to facilitate the development of 2 related, disease-specific outcome measures assessing ease of movement in PMM and PolG clinical trials. The Mitochondrial Video Assessment (MVA) utilizes remote video capture to assess subtle shifts in compensatory movement patterns and measure change in function over time, and the Current State Questionnaire asks participants about external factors in their daily lives which may influence fluctuations in their symptoms. As part of the questionnaire, participants are asked to rate their own perception of their functional ability using a Patient Global Impression of Severity (PGI-S) scale.
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
- 16 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •16 years old and above
- •Confirmed diagnosis of PMM or PolG
- •Ability to understand and speak English
- •Ability and willing to consent
- •Able and willing to participate and perform tasks while being recorded
- •Designating a recording partner to assist with video captures is strongly advised, but not required
排除标准
- 未提供
结局指标
主要结局
Source Material Videos
时间窗: 2 weeks
Initiate feasibility testing and collect source material videos from patients with Primary Mitochondrial Myopathy (PMM, n=35) or a PolGamma (PolG) mutation (n=15) to facilitate development of the Mitochondrial Video Assessment (MVA).
次要结局
- PGI-S(2 weeks)