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International Primary Ciliary Dyskinesia (PCD) Registry (PCDregistry)

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ClinicalTrials.gov Identifier: NCT02419365
Recruitment Status : Recruiting
First Posted : April 17, 2015
Last Update Posted : July 9, 2019
Sponsor:
Collaborators:
European Commission
University of Nicosia
Rigshospitalet, Denmark
KU Leuven
Hannover Medical School
Attikon Hospital
VU University Medical Center
University of Bern
University of Southampton
Royal Brompton & Harefield NHS Foundation Trust
University of North Carolina
Ruhr University of Bochum
Federico II University
Hospital Vall d'Hebron
Medical University of Vienna
Marmara University
University Hospital, Martin
University of Pisa
Assistance Publique - Hôpitaux de Paris
University of Alberta
University of Giessen
Information provided by (Responsible Party):
Petra Pennekamp, University Hospital Muenster

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.

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.


Condition or disease
Primary Ciliary Dyskinesia (PCD)

  Show Detailed Description

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Study Type : Observational [Patient Registry]
Estimated Enrollment : 2000 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 20 Years
Official Title: International Prospective Primary Ciliary Dyskinesia (PCD) Registry for Systematic Data Collection on Incidence, Clinical Presentation, Treatment and Course of the Disease
Study Start Date : January 2014
Estimated Primary Completion Date : December 2030
Estimated Study Completion Date : December 2030


Group/Cohort
PCD
Individuals with Primary Ciliary Dyskinesia will be assessed in a pure observational design without intervention



Primary Outcome Measures :
  1. 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.



Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Individuals with a diagnosis of Primary Ciliary Dyskinesia.
Criteria

Inclusion Criteria

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.

Exclusion Criteria Failure or unwillingness to give written informed consent. Missing qualification to perform legal acts or insufficient cognitive ability to give informed consent. A second disease is no exclusion criteria for including data in the PCD-Registry, especially since it is one of the fundamental goals, to learn about co-morbidities.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02419365


Contacts
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Contact: Petra Pennekamp, PhD +4925183 ext 41097 petra.pennekamp@ukmuenster.de
Contact: Simone Helms +4925183 ext 48358 Simone.Helms@ukmuenster.de

Locations
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Germany
University Hospital Münster Recruiting
Münster, NRW, Germany, 48149
Contact: Heymut Omran, Prof. Dr. MD    004925183 ext 41097    PCDregistry.eu@ukmuenster.de   
Contact: Simone Helms    +4925183 ext 48358    Simone.Helms@ukmuenster.de   
Principal Investigator: Heymut Omran, Prof. Dr. MD         
Sub-Investigator: Johanna Raidt, MD, Dr. med.         
University Hospital Muenster, Department of General Pediatrics Recruiting
Muenster, Germany, 48149
Contact: Petra Pennekamp, PhD    +4925183 ext 41097    petra.pennekamp@ukmuenster.de   
Contact: Simone Helms    +4925183 ext 48358    Simone.Helms@ukmuenster.de   
Principal Investigator: Heymut Omran, Prof. Dr.         
Sub-Investigator: Johanna Raidt, Dr. med.         
Sponsors and Collaborators
University Hospital Muenster
European Commission
University of Nicosia
Rigshospitalet, Denmark
KU Leuven
Hannover Medical School
Attikon Hospital
VU University Medical Center
University of Bern
University of Southampton
Royal Brompton & Harefield NHS Foundation Trust
University of North Carolina
Ruhr University of Bochum
Federico II University
Hospital Vall d'Hebron
Medical University of Vienna
Marmara University
University Hospital, Martin
University of Pisa
Assistance Publique - Hôpitaux de Paris
University of Alberta
University of Giessen

Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Petra Pennekamp, Dr, University Hospital Muenster
ClinicalTrials.gov Identifier: NCT02419365     History of Changes
Other Study ID Numbers: PCD Registry
First Posted: April 17, 2015    Key Record Dates
Last Update Posted: July 9, 2019
Last Verified: July 2019
Keywords provided by Petra Pennekamp, University Hospital Muenster:
Primary Ciliary Dyskinesia (PCD)
Additional relevant MeSH terms:
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Ciliary Motility Disorders
Kartagener Syndrome
Dyskinesias
Movement Disorders
Central Nervous System Diseases
Nervous System Diseases
Neurologic Manifestations
Signs and Symptoms
Respiratory Tract Diseases
Otorhinolaryngologic Diseases
Ciliopathies
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn
Bronchiectasis
Bronchial Diseases
Respiratory System Abnormalities
Dextrocardia
Heart Defects, Congenital
Cardiovascular Abnormalities
Cardiovascular Diseases
Heart Diseases
Situs Inversus