International Prospective Primary Ciliary Dyskinesia (PCD) Registry for Systematic Data Collection on Incidence, Clinical Presentation, Treatment and Course of the Disease
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 2,000
- 试验地点
- 2
- 主要终点
- descriptive analysis
研究概览
简要总结
The purpose of the international prospective PCD Patient Registry is to systematically measure, survey and compare different aspects of PCD manifestation, course and treatment, to provide data for epidemiological research and to identify special patient groups suitable for multi-center trials.
This International PCD Registry is also part of the European Reference Network ERN-LUNG. We follow the recommendations of the EU Expert Committee on Rare Diseases (EUCERD), which recommend an international interoperability of registries and databases to pool and exchange knowledge and data on rare diseases.
详细描述
PCD is a rare disorder of mucociliary clearance caused by defective hair like organelles (cilia). The purpose of the PCD Registry is to measure, survey and compare different aspects of PCD manifestation, course and treatment, to provide data for epidemiological research and to identify special patient groups suitable for multi-centre trials.
The PCD Registry is part of the European Reference Network for Rare Lung Diseases ERN-LUNG (https://ern-lung.eu/). We follow the recommendations of the EU Expert Committee on Rare Diseases (EUCERD), which recommend an international interoperability of registries and databases to pool and exchange knowledge and data on rare diseases.
How can the data be entered to the registry? The data can either be entered into the PCD Registry directly after login, or imported via a specific *CSV file that we provide in advance (download option after Log In or e-mail request to PCDregistry.eu@ukmuenster.de, Simone.Helms@ukmuenster.de or Petra.Pennekamp@ukmuenster.de).
Under special circumstances (e.g. import of existing datasets from other registries/cohorts) we provide support to enter your data. The PCD Registry is accessible from the web. Each authorised operator is able to enter the data for their country/centre and export own center specific data to different formats (CSV/Microsoft Excel, PDF, SAS, Stata, R, ODM or SPSS).
A highly protected system will ensure data protection through the encryption of data. Identifying patient data are not collected in the PCD Registry.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
结局指标
主要结局
descriptive analysis
时间窗: 10 years
Descriptive statistical methods, i.e. frequency tables, location and scale statistics and graphical presentations will be applied to analyse: age at diagnosis, family history (consanguinity), associated malformations/diseases, laterality defects, clinical manifestations (otitis media, rhinosinusitis, pneumonia, bronchiectasis, neonatal respiratory distress syndrome), microbiological results, diagnostic findings (video microscopy, electron microscopy, nasal NO, immunofluorescence analysis), lung function, radiological findings and therapeutic measures (inhalation therapy, antibiotics, oxygen, ventilation, upper airways surgery, lung surgery). Furthermore, quality of life will be assessed using QOL-PCD, a disease specific questionnaire.
次要结局
未报告次要终点
研究者
Petra Pennekamp
Dr
University Hospital Muenster