Prospective Cohort Study of Patients With Mutations in the Dystrophin Gene (X Linked Dilated Cardiomyopathy and Becker Muscular Dystrophy)
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 100
- 试验地点
- 1
- 主要终点
- Left ventricular ejection fraction
研究概览
简要总结
The purpose of this study is to determine whether electrocardiogram, echocardiography, cardiac MRI, sera biomarkers can improve early detection of myocardial involvement and clinical outcome.
详细描述
A cohort of 100 patients with mutations in the dystrophin gene associated with Becker muscular dystrophy and/or dilated cardiomyopathy will be included (patients with Duchenne muscular dystrophy are excluded).
Patients with undergo at baseline the following workups: electrocardiogram, echocardiography, cardiac MRI, sera biomarkers measurement.
At 3 years and 5 years, patients will be investigated according to the same protocol and occurrence of cardiac adverse events in the meanwhile will be recorded.
Statistical analysis will assess correlations between cardiac phenotype and DMD mutations and prognostic value of cardiac investigations.
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
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •mutation in the DMD gene
- •Becker muscular dystrophy and/or dilated cardiomyopathy
- •age>18 years
- •affiliation to the French medical insurance
排除标准
- •Duchenne muscular dystrophy
- •Any other chronic disease that may be associated with heart disease
结局指标
主要结局
Left ventricular ejection fraction
时间窗: 3 years
次要结局
- Composite endpoint: hospitalisation for heart failure, death due to heart failure(3 years)
研究者
Karim WAHBI
MD, PhD
Institut de Myologie, France