The Ear-Nose-Throat (ENT) Prospective International Cohort of PCD Patients (EPIC-PCD) (EPIC-PCD)

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ClinicalTrials.gov Identifier: NCT04611516

Recruitment Status : Recruiting

First Posted : November 2, 2020

Last Update Posted : May 18, 2022

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View this study on Beta.ClinicalTrials.gov

Sponsor:

Collaborators:

Bicetre Hospital

Centre Hospitalier Intercommunal Creteil

University Hospital Southampton NHS Foundation Trust

Universitaire Ziekenhuizen KU Leuven

Amsterdam UMC, location VUmc

University of Cyprus

Hacettepe University

Marmara University

Hospital Universitario La Fe

University Hospital Inselspital, Berne

Information provided by (Responsible Party):

University of Bern

Study Description

Brief Summary:

The Ear-Nose-Throat (ENT) Prospective International Cohort of patients with Primary Ciliary Dyskinesia (EPIC-PCD) is a prospective observational clinical cohort study, set up as a multinational multi-centre study. It is embedded into routine patient care of participating reference centres for PCD and patients keep being managed according to local procedures and guidelines.

Condition or disease
Primary Ciliary Dyskinesia Kartagener Syndrome

Show detailed description

Detailed Description:

The EPICD-PCD is hosted at the Institute of Social and Preventive Medicine (ISPM) at the University of Bern, Switzerland. Research is performed in close collaboration with all data contributors. This study aims to characterise ENT disease in PCD patients and its association with lower respiratory disease, and to identify determinants of its prognosis.

The investigators aim to:

Assess the prevalence and severity of sinonasal and otologic symptoms and the frequency and range of signs and physiological findings assessed during standardised ENT physical examination, and describe differences by age;
Study the association of sinonasal and otologic disease with lower respiratory disease in PCD patients;
Identify determinants of disease course and prognosis of sinonasal and otologic disease in PCD patients.

Study design:

The EPIC-PCD is a prospective observational clinical cohort study, set up as a multinational multi-centre study. It is embedded into routine patient care of participating reference centres for PCD and patients will keep being managed according to local procedures and guidelines.

Patients with PCD are followed regularly at each centre, at 3-month to 6-month intervals. Each patient undergoes a detailed ENT sinonasal and otologic examination by ENT specialists, at minimum once a year, during a programmed follow-up visit. Additional ENT examinations are performed if indicated during in-between follow-up visits. Patients will not be subjected to additional invasive measurements solely for the purposes of the study.

What information is collected:

The study collect clinical data from patients assessment at regular consultations at the outpatient clinics.

For the collection of clinical data, participating centres will use FOLLOW-PCD, a disease-specific form for standardised prospective data collection during routine clinical follow-up of PCD patients.

Study database:

The EPIC-PCD database is web-based, using the Research Electronic Data Capture (REDCap) platform developed at Vanderbilt University. REDCap is widely used in academic research and allows data entry and extraction in various formats.

How to participate:

Centres that wish to participate to the project and contribute data can contact the EPIC-PCD managing centre to sign a data delivery agreement. They then will receive a password to access the online software REDCap and they will be able to enter their data directly. They can also upload follow-up data or add additional patients at a later time point.

Funding:

The setting up of the EPIC-PCD (salaries, consumables and equipment) was funded by the Swiss National Science Foundation. Data collection and management at each site was funded according to local arrangements. Most participating researchers and data contributors participate in the European Respiratory Society Clinical Research collaboration "Better Evidence to Advance Therapeutic options for PCD" (BEAT-PCD) (https://beat-pcd.squarespace.com/). Infrastructure is provided for free by the University of Bern, where the data are pooled and stored.

Study Design

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Study Type :	Observational
Estimated Enrollment :	500 participants
Observational Model:	Cohort
Time Perspective:	Prospective
Official Title:	The Ear-Nose-Throat (ENT) Prospective International Cohort of PCD Patients (EPIC-PCD)
Actual Study Start Date :	March 2, 2020
Estimated Primary Completion Date :	December 2024
Estimated Study Completion Date :	December 2030

Resource links provided by the National Library of Medicine

MedlinePlus Genetics related topics: Primary ciliary dyskinesia

Genetic and Rare Diseases Information Center resources: Primary Ciliary Dyskinesia Kartagener Syndrome Dextrocardia Situs Inversus

U.S. FDA Resources

Groups and Cohorts

Outcome Measures

Primary Outcome Measures :

Sinonasal symptoms and signs [ Time Frame: at baseline ]
Prevalence of reported clinical symptoms at different age groups, including rhinitis, sinusitis, snoring
Course of sinonasal symptoms and signs (year 1) [ Time Frame: at 12 months from recruitment ]
Change from baseline prevalence of reported clinical symptoms at different age groups, including rhinitis, sinusitis, snoring, at 12 months
Course of sinonasal symptoms and signs (year 2) [ Time Frame: at 24 months from recruitment ]
Change from baseline prevalence of reported clinical symptoms at different age groups, including rhinitis, sinusitis, snoring, at 24 months
Otological symptoms and signs [ Time Frame: at baseline ]
Prevalence of reported clinical symptoms at different age groups, including ear pain, hearing problems, ear discharge
Course of otological symptoms and signs (year 1) [ Time Frame: at 12 months from recruitment ]
Change from baseline prevalence of reported clinical symptoms at different age groups, including ear pain, hearing problems, ear discharge, at 12 months
Course of otological symptoms and signs (year 2) [ Time Frame: at 24 months from recruitment ]
Change from baseline prevalence of reported clinical symptoms at different age groups, including ear pain, hearing problems, ear discharge, at 24 months
Nasal endoscopy (description of nasal mucosa) [ Time Frame: at baseline ]
Description of nasal mucosa as seen via nasal endoscopy examination
Nasal endoscopy (changes of nasal mucosa at year 1) [ Time Frame: at 12 months from recruitment ]
Changes in nasal mucosa from baseline as seen via nasal endoscopy examination at 12 months
Nasal endoscopy (changes of nasal mucosa at year 2) [ Time Frame: at 24 months from recruitment ]
Changes in nasal mucosa from baseline as seen via nasal endoscopy examination at 24 months
Nasal endoscopy (nasal polyps) [ Time Frame: at baseline ]
Prevalence of nasal polyps at baseline
Nasal endoscopy (nasal polyps at year 1) [ Time Frame: at 12 months from recruitment ]
Changes in prevalence of nasal polyps from baseline to 12 months
Nasal endoscopy (nasal polyps at year 2) [ Time Frame: at 24 months from recruitment ]
Changes in prevalence of nasal polyps from baseline to 24 months
Sinonasal examination (Lidholdt score) [ Time Frame: at baseline ]
Lidholdt score at baseline
Sinonasal examination (Lidholdt score at year 1) [ Time Frame: at 12 months from recruitment ]
Changes in Lidholdt score from baseline to 12 months
Sinonasal examination (Lidholdt score at year 2) [ Time Frame: at 24 months from recruitment ]
Changes in Lidholdt score from baseline to 24 months
Ear examination (ear discharge) [ Time Frame: at baseline ]
Prevalence of ear discharge at baseline
Ear examination (ear discharge at year 1) [ Time Frame: at 12 months from recruitment ]
Changes in prevalence of ear discharge from baseline to 12 months
Ear examination (ear discharge at year 2) [ Time Frame: at 24 months from recruitment ]
Changes in prevalence of ear discharge from baseline to 24 months
Otoscopy (description of the tympanic membrane) [ Time Frame: at baseline ]
Description of tympanic membrane as seen via otoscopy examination
Otoscopy (description of the tympanic membrane at year 1) [ Time Frame: at 12 months from recruitment ]
Changes in the tympanic membrane from baseline to 12 months as seen via otoscopy examination
Otoscopy (description of the tympanic membrane at year 2) [ Time Frame: at 24 months from recruitment ]
Changes in the tympanic membrane from baseline to 24 months as seen via otoscopy examination
Tympanometry [ Time Frame: at baseline ]
Results of tympanometry (tympanogram type for both ears) at baseline
Tympanometry (at year 1) [ Time Frame: at 12 months from recruitment ]
Changes in tympanogram (for both ears) from baseline to 12 months
Tympanometry (at year 2) [ Time Frame: at 24 months from recruitment ]
Changes in tympanogram (for both ears) from baseline to 24 months
Audiometry [ Time Frame: at baseline ]
Results of audiometry: type of audiometry and results based on WHO hearing loss grades (for both ears)
Audiometry (at year 1) [ Time Frame: at 12 months from recruitment ]
Changes in audiometry results from baseline to 12 months
Audiometry (at year 2) [ Time Frame: at 24 months from recruitment ]
Changes in audiometry results from baseline to 24 months

Eligibility Criteria

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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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 collaborating PCD centre

Criteria

Inclusion Criteria:

Diagnosis of PCD (clinical and test certified)
Patient must undergo an ENT examination minimum once a year as part of their clinical follow-up

Exclusion Criteria:

None

Contacts and Locations

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): NCT04611516

Contacts

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Contact: Myrofora Goutaki, MD-PhD	0041 316315973	myrofora.goutaki@ispm.unibe.ch
Contact: Yin Ting Lam, MD	0041 316315973	yin.lam@ispm.unibe.ch

Locations

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Switzerland
University of Bern	Recruiting
Bern, Switzerland
Contact: Yin Ting Lam, MD yin.lam@ispm.unibe.ch

Sponsors and Collaborators

University of Bern

Bicetre Hospital

Centre Hospitalier Intercommunal Creteil

University Hospital Southampton NHS Foundation Trust

Universitaire Ziekenhuizen KU Leuven

Amsterdam UMC, location VUmc

University of Cyprus

Hacettepe University

Marmara University

Hospital Universitario La Fe

University Hospital Inselspital, Berne

Investigators

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Principal Investigator:	Myrofora Goutaki, MD-PhD	University of Bern

More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Goutaki M, Lam YT, Alexandru M, Anagiotos A, Armengot M, Bequignon E, Boon M, Burgess A, Coste A, Emiralioglu N, Erdem E, Haarman EG, Harris A, Hool SL, Karadag B, Kim S, Latzin P, Lorent N, Ozcelik U, Reula A, Roehmel J, van Gogh C, Yiallouros P, Zappe SM; EPIC-PCD team; Papon JF. Study protocol: the ear-nose-throat (ENT) prospective international cohort of patients with primary ciliary dyskinesia (EPIC-PCD). BMJ Open. 2021 Oct 11;11(10):e051433. doi: 10.1136/bmjopen-2021-051433.

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Responsible Party:	University of Bern
ClinicalTrials.gov Identifier:	NCT04611516
Other Study ID Numbers:	EPIC-PCD
First Posted:	November 2, 2020 Key Record Dates
Last Update Posted:	May 18, 2022
Last Verified:	May 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD:	Undecided
Plan Description:	Not all participating centres have ethical approval to share IPD to external researchers. Researchers wanting to use the 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 EPIC-PCD 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 EPIC-PCD dataset to enrich it after each project.

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

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 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

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