Beijing Children's Hospital
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 200
- 试验地点
- 1
- 主要终点
- Change from baseline in lung function on the spirometry
研究概览
简要总结
This study is a multicenter, prospective cohort study of patients diagnosed with respiratory monogenic diseases, the clinical information of recruited patients, including clinical manifestations, lung function, chest imaging, quality of life and other indicators, will be followed for 5 years.
详细描述
All new cases of respiratory monogenic diseases was confirmed or diagnosed at each center from the beginning of the study are made the investigation of the clinical manifestations and specific tests by the standard diagnostic process.Then all the patients' following clinical data will be followed for 5 years: clinical manifestations, lung function, chest imaging (once per year), gene test, quality of life and other indicators.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 年龄范围
- 1 Month 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age: 0-18 years, male and female.
- •Any organ system symptoms consistent with the diagnostic points of respiratory monogenic diseases.
- •Agree to retain specimens related to disease research and agree to store the specimens in the sample bank.
- •willing and able to cooperate with long-term follow-up.
- •The guardian of the child has a full understanding of the purpose of the study, a basic understanding of the clinical research program, and voluntarily allows the child to participate in the study and sign an informed consent form.
排除标准
- •It is unable to provide complete medical records or the current condition can not accept the diagnosis process
- •She or he cannot agree to participate in the study.
结局指标
主要结局
Change from baseline in lung function on the spirometry
时间窗: 1 year later
forced expiratory volume at one second (FEV1) in Liter
次要结局
未报告次要终点
研究者
Kunling Shen
Beijing Children's Hospital
Beijing Children's Hospital