Assessment of Cardiopulmonary Function in Duchenne Muscular Dystrophy
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 9
- 试验地点
- 1
- 主要终点
- Magnetic Resonance (MRI) T2 and Magnetic Resonance Spectroscopy (MRS)
研究概览
简要总结
This study seeks to develop and validate non-invasive assessments of cardiac and respiratory muscles with magnetic resonance imaging (MRI) to better predict the natural disease progression of Duchenne muscular dystrophy (DMD) in affected individuals over time, as well as determine whether peripheral skeletal muscle dysfunction can predict cardiopulmonary dysfunction. The central hypothesis is that non-invasive MRI measures of the heart, muscle, and peripheral skeletal muscles can sensitively predict future cardiopulmonary decline.
详细描述
Magnetic Resonance Imaging (MRI) of the heart and breathing muscles, special breathing tests (called pulmonary function testing), special exercise tests (using a stationary bike), and possibly an echocardiogram (ultrasound of the heart, commonly known as an "echo") will be completed up to 4 times per year for up to 4 years. Most participants will complete testing once or twice each year; however, some participants will be asked to repeat some of the tests twice during each visit.
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
- 5 Years 至 15 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •5-15 years old at the time of enrollment
- •Diagnosed with DMD (as defined by parent project)
- •Written parental informed consent (and assent where appropriate) before any study procedures take place
排除标准
- •Contraindication to an MRI examination
- •Presence of a secondary condition that impacts muscle function or metabolism, that leads to developmental delay or impaired motor control, or that is not stable
- •Participant is unable to comply with study requirements
- •Congenital structural abnormality of the heart, repaired or unrepaired
- •Clinically contraindicated participation
结局指标
主要结局
Magnetic Resonance (MRI) T2 and Magnetic Resonance Spectroscopy (MRS)
时间窗: up to 4 years
The MRI T2 and MRS will be used as a noninvasive marker of myocardial damage/inflammation of participants of this study as an early detection for DMD.
次要结局
- Pulmonary Function Testing (PFT)(up to 4 years)
- Metabolic Exercise Testing (exercise capacity and MVO2)(up to 4 years)
- Multiple-echo Dixon(up to 4 years)