Clinical Trial: International Prospective Patient Registry for Primary Ciliary Dyskinesia (PCD)

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational [Patient Registry]

Official Title: Prospective Primary Ciliary Dyskinesia (PCD) Registry for Systematic Data Collection on Incidence, Clinical Presentation, Treatment and Course of the Disease

Brief Summary: 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.

Detailed Summary: Main objective is to establish an international prospective registry for Primary Ciliary Dyskinesia (PCD) for systematic data collection on incidence, clinical presentation, treatment and course of the disease.This will allow to monitor trends in diagnosis, management and outcomes and will show how efforts to improve diagnosis and treatments will change clinical practice and long-term outcomes.
Sponsor: University Hospital Muenster

Current Primary Outcome: descriptive analysis [ Time Frame: 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.


Original Primary Outcome: Same as current

Current Secondary Outcome:

Original Secondary Outcome:

Information By: University Hospital Muenster

Dates:
Date Received: February 3, 2014
Date Started: January 2014
Date Completion: December 2030
Last Updated: April 13, 2015
Last Verified: April 2015