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International Primary Ciliary Dyskinesia Cohort (iPCD)

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ClinicalTrials.gov Identifier: NCT03517865
Recruitment Status : Recruiting
First Posted : May 8, 2018
Last Update Posted : May 8, 2018
Sponsor:
Collaborators:
European Commission
Swiss National Science Foundation
University of Southampton
Pierre and Marie Curie University
Bar-Ilan University, Israel
University of Padova
University Hospital, Gasthuisberg
Oslo University Hospital
VU University Medical Center
Royal Brompton & Harefield NHS Foundation Trust
Marmara University
Ruhr University of Bochum
Genetic Disorders of Mucociliary Clearance Consortium
Institute of Tuberculosis and Lung Disorders, Rabka Poland
University of Sydney
Copenhagen University Hospital, Denmark
University Hospital Muenster
Hannover Medical School
Hospital de Niños R. Gutierrez de Buenos Aires
University of Cyprus
Medical Centre Dr Dragisa Misovic
Hacettepe University
University Hospital, Motol
Clinica de neumologia pediatrica Compensar
Attikon Hospital
University of Leicester
Information provided by (Responsible Party):
University of Bern

Brief Summary:
The iPCD Cohort is a retrospective international cohort that assembles available datasets with clinical and diagnostic data from patients suffering from primary ciliary dyskinesia (PCD) worldwide, to answer pertinent questions on clinical phenotype, disease severity, prognosis and effect of treatments in patients with this rare multiorgan disease.

Condition or disease
Primary Ciliary Dyskinesia Kartagener Syndrome

  Show Detailed Description

Study Type : Observational
Estimated Enrollment : 4000 participants
Observational Model: Cohort
Time Perspective: Other
Official Title: International Primary Ciliary Dyskinesia Cohort
Actual Study Start Date : January 2013
Estimated Primary Completion Date : December 2028
Estimated Study Completion Date : December 2030





Primary Outcome Measures :
  1. Height [ Time Frame: every 3 months up to 10 years ]
    Height z-scores calculated based on available national and international references

  2. BMI [ Time Frame: every 3 months up to 10 years ]
    Body Mass Index (BMI) z-scores calculated based on available national and international references

  3. Lung function measurements [ Time Frame: every 3 months up to 10 years ]
    Spirometric indices, particularly Forced expiratory volume in 1 sec (FEV1) and Forced vital capacity (FVC) z-scores calculated based on Global Lung Function Initiative (GLI) reference values

  4. Diagnostic test results [ Time Frame: at diagnosis/ study entry ]
    Results of performed PCD diagnostic tests including measurement of nasal nitric oxide, electron microscopy findings, beat frequency and pattern.

  5. Clinical symptoms and signs [ Time Frame: every 3 months up to 10 years ]
    Prevalence of reported clinical symptoms at different age groups, including rhinitis, cough, otitis, sinusitis, pneumonia, laterality defects, congenital heart disease and fertility problems.

  6. Microbiology results [ Time Frame: every 3 months up to 10 years ]
    Results of microbiology cultures of respiratory samples (sputum, cough swabs, throat swabs, ear swabs, bronchoalveolar lavage) and information on antibiotic resistance (in positive cultures)

  7. Imaging results [ Time Frame: every 3 months up to 10 years ]
    Radiological findings from sinus and lung imaging tests including x-rays, computed tomography and magnetic resonance imaging



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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
Patients of all ages diagnosed with PCD, followed in a PCD centre or other specialised clinic or registered in a national registry
Criteria

Inclusion Criteria:

Patients diagnosed with primary ciliary dyskinesia

Exclusion Criteria:


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): NCT03517865


Contacts
Contact: Claudia E Kuehni, Prof 0041 316313507 claudia.kuehni@ispm.unibe.ch
Contact: Myrofora Goutaki, MD-PhD 0041 316315973 myrofora.goutaki@ispm.unibe.ch

Locations
Switzerland
University of Bern Recruiting
Bern, Switzerland, 3012
Contact: Claudia E Kuehni, Prof       claudia.kuehni@ispm.unibe.ch   
Contact: Myrofora Goutaki, MD-PhD       myrofora.goutaki@ispm.unibe.ch   
Sponsors and Collaborators
University of Bern
European Commission
Swiss National Science Foundation
University of Southampton
Pierre and Marie Curie University
Bar-Ilan University, Israel
University of Padova
University Hospital, Gasthuisberg
Oslo University Hospital
VU University Medical Center
Royal Brompton & Harefield NHS Foundation Trust
Marmara University
Ruhr University of Bochum
Genetic Disorders of Mucociliary Clearance Consortium
Institute of Tuberculosis and Lung Disorders, Rabka Poland
University of Sydney
Copenhagen University Hospital, Denmark
University Hospital Muenster
Hannover Medical School
Hospital de Niños R. Gutierrez de Buenos Aires
University of Cyprus
Medical Centre Dr Dragisa Misovic
Hacettepe University
University Hospital, Motol
Clinica de neumologia pediatrica Compensar
Attikon Hospital
University of Leicester

Publications of Results:
Responsible Party: University of Bern
ClinicalTrials.gov Identifier: NCT03517865     History of Changes
Other Study ID Numbers: iPCD cohort
First Posted: May 8, 2018    Key Record Dates
Last Update Posted: May 8, 2018
Last Verified: April 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description:

Researchers wanting to use the iPCD Cohort dataset can propose a research topic and draft a concept sheet describing the planned analyses and publication. All concept sheets have to be approved by all participating centres contributing data to the proposed analysis under question. After the participating centres agree to contribute their data and sign a publication agreement, the iPCD cohort managing centre (University of Bern) will prepare a partial dataset for the proposed analysis and will work closely with the lead researchers offering methodological input and support. In case additional data is collected to complete the partial dataset for a specific project, this will be added to the iPCD Cohort to enrich it after each project.

For further details, contact: pcd@ispm.unibe.ch

URL: http://

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Dyskinesias
Ciliary Motility Disorders
Kartagener Syndrome
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