Magnetic Resonance Imaging and Spectroscopy Biomarkers for Facioscapulohumeral Muscular Dystrophy
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 75
- 试验地点
- 2
- 主要终点
- Intramuscular fat infiltration on MRI
研究概览
简要总结
The purpose of this research study is to identify and study changes in muscle in people with facioscapulohumeral muscular dystrophy using magnetic resonance imaging and spectroscopy.
详细描述
This research study is being done to study changes in muscle imaging over time in people with facioscapulohumeral muscular dystrophy (FSHD). Whole-body magnetic resonance imaging (MRI) and magnetic resonance spectroscopy (MRS) will be used to evaluate skeletal muscle in study participants. This research is being done to assess how changes in muscle imaging correspond to muscle strength and function. Qualified participants will be asked to complete 5 study visits over 21 months. Each visit will include muscle strength and function testing in addition to the MRI/MRS scan. The investigators plan to use MRI and MRS in developing outcome measures that can be used in future clinical trials for FSHD.
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 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Ability to give written informed consent for study participation (a parent or guardian will be asked to provide informed consent for participants younger than 18 years old)
- •Confirmed diagnosis of FSHD through genetic testing (participants will be asked to provide copies of genetic testing results)
排除标准
- •Any contraindication to MRI scanning
- •Inability to complete a physical examination, including strength measurements
结局指标
主要结局
Intramuscular fat infiltration on MRI
时间窗: Baseline, 3 months, 9 months, 15 months, and 21 months
Intramuscular fat infiltration on MRI
次要结局
- Intramuscular edema on MRI(Baseline, 3 months, 9 months, 15 months, and 21 months)
- Timed function testing(Baseline, 3 months, 9 months, 15 months, and 21 months)
- Muscle strength testing(Baseline, 3 months, 9 months, 15 months, and 21 months)