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临床试验/NCT01136330
NCT01136330已完成不适用

Cardiac and Respiratory Prognostic Factors in Patients With Myotonic Dystrophy Type 1

Institut de Myologie, France1 个研究点 分布在 1 个国家目标入组 914 人开始时间: 2010年5月最近更新:
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适应症

试验速览

阶段
不适用
状态
已完成
发起方
入组人数
914
试验地点
1
研究概览研究设计入排标准研究者研究点记录与标识符相似试验

研究概览

简要总结

Myotonic dystrophy type 1 (DM1) is the most frequent neuromuscular disease in adults. DM1 patients have an impaired prognosis (mean age of death <60 years) due to cardiac and respiratory complications.

Our primary objective was to identify cardiac and respiratory prognostic factors in DM1.

详细描述

  1. Patients with genetically proven DM1 who were admitted in Pitié Salpêtrière Hospital from 2000 and 2010 will be identified. These patients systematically underwent neurological, cardiac and respiratory investigations.
  2. Baseline medical and genetic information will be entered in a dedicated database, including cardiac and respiratory investigations.
  3. The occurence of severe cardiac and respiratory adverse events will also be collected.
  4. Statistical analysis will be performed to look for correlations between baseline patient characteristics and cardiac or respiratory adverse events during follow up.

研究设计

研究类型
Observational
观察模型
Cohort
时间视角
Retrospective

入排标准

年龄范围
18 Years 至 —（Adult, Older Adult）
性别
All
接受健康志愿者
否

入选标准

  • •DM1 mutation (>50 CTG repeats)
  • •Age > 18 years

排除标准

  • •Patient refusal

研究者

发起方
Institut de Myologie, France
申办方类型
Other
责任方
Principal Investigator
主要研究者

Karim WAHBI

Karim WAHBI MD,PHD.

Institut de Myologie, France

研究点 (1)

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