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

Status: Recruiting
Location: See all (2) locations...
Study Type: Observational
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. 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.

Eligibility
Participation Requirements
Sex: All
Healthy Volunteers: f
View:

• Patients of any age who fulfil the diagnostic criteria below are eligible:

• Clinical presentation consistent with PCD and consistent findings specific for PCD in at least two of the following methods:

• high frequency video microscopic finding transmission electron microscopy finding immunofluorescence finding low nasal NO concentration/production demonstration of biallelic disease-causing mutations by genotyping

• Given the complexity of diagnosing PCD, it is anticipated that not all patients will meet these definite diagnostic criteria. Therefore, individuals with typical clinical symptoms and only one abnormal diagnostic test are also eligible to enter the registry. These cases usually are considered to have a possible PCD diagnosis with exceptions made on an individual basis.

Locations
Other Locations
Germany
University Hospital Muenster, Department of General Pediatrics
RECRUITING
Münster
University Hospital Münster
RECRUITING
Münster
Contact Information
Primary
Petra Pennekamp, PhD
petra.pennekamp@ukmuenster.de
+4925183
Backup
Simone Helms
Simone.Helms@ukmuenster.de
+4925183
Time Frame
Start Date: 2014-01
Estimated Completion Date: 2030-12
Participants
Target number of participants: 2000
Treatments
PCD
Individuals with Primary Ciliary Dyskinesia will be assessed in a pure observational design without intervention
Related Therapeutic Areas
Sponsors
Collaborators: University of Nicosia, Federico II University, Royal Brompton & Harefield NHS Foundation Trust, Amsterdam UMC, location VUmc, Hospital Vall d'Hebron, University of Pisa, Hannover Medical School, Ruhr University of Bochum, University Hospital, Martin, Attikon Hospital, University of North Carolina, University of Giessen, Medical University of Vienna, Assistance Publique - Hôpitaux de Paris, Marmara University, University of Alberta, European Commission, University of Bern, University of Southampton, KU Leuven, Rigshospitalet, Denmark
Leads: University Hospital Muenster

This content was sourced from clinicaltrials.gov