Longitudinal Study of Neurodegenerative Disorders
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 1,500
- 试验地点
- 1
- 主要终点
- Adaptive living skills
研究概览
简要总结
The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understanding of the progression of the rare neurodegenerative disorders and the effects of interventions.
详细描述
Patients would be evaluated by a multidisciplinary team at specific time points every 3 months the first year, every 6 months the second year and once a year thereafter.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Any patient with a genetic neurodegenerative disorder
排除标准
- 未提供
结局指标
主要结局
Adaptive living skills
时间窗: 15 years
Repeated standardized age equivalent scores.
Language development
时间窗: 15 years
Repeated standardized age equivalent scores.
Gross Motor development .
时间窗: 15 years
Repeated standardized age equivalent scores.
Cognitive development
时间窗: 15 years
Repeated standardized age equivalent scores.
Fine Motor development
时间窗: 15 years
Repeated standardized age equivalent scores.
次要结局
- Neurodegeneration of the brain as measured by MRI diffusion tensor imaging from birth to 5 years of age(5 years)
- Exploratory biomarkers(15 years)
研究者
Deepa Soundara Rajan
Associate Professor
University of Pittsburgh