NCT03443115Unknown不适用
Multicenter Cohort Study on Duchenne Muscular Dystrophy Cardiomyopathy
Hôpital Cochin1 个研究点 分布在 1 个国家目标入组 700 人开始时间: 2017年6月27日最近更新:
适应症
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 700
- 试验地点
- 1
- 主要终点
- All-cause mortality
研究概览
简要总结
Retrospective cohort study including patients with genetically proven Duchenne muscular dystrophy, diagnosed from January 1993 to March 2020.
Inclusion of the data relative to genetic diagnosis, clinical characteristics at baseline, cardiac and respiratory workup, medical treatments (ACE inhibitors, steroids), surgical procedures, and occurrence during follow-up of cardiac, respiratory and fatal events.
Objectives are to describe long-term natural history of the disease, vital prognosis, genotype-phenotype correlations, effect of treatments.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Genetically-proven Duchenne Muscular Dystrophy (DMD mutation)
- •Covering by social security
排除标准
- •Patient refusal to participate to the study
结局指标
主要结局
All-cause mortality
时间窗: Follow-up completed in March 2020
次要结局
- Hospitalization for acute respiratory failure(Follow-up completed in March 2020)
- Hospitalization for heart failure(Follow-up completed in March 2020)
- Dilated cardiomyopathy(Follow-up completed in March 2020)
研究者
Karim Wahbi
MD, PhD
Hôpital Cochin
研究点 (1)
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