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NCT Number: NCT04611516

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

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.

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Key information

Conditions

Primary Ciliary Dyskinesia Abnormalities, Multiple Bronchial Diseases Bronchiectasis Cardiovascular Abnormalities Cardiovascular Diseases Ciliary Motility Disorders Ciliopathies Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dextrocardia Genetic Diseases, Inborn Heart Defects, Congenital Heart Diseases Kartagener Syndrome Otorhinolaryngologic Diseases Respiratory System Abnormalities Respiratory Tract Diseases Situs Inversus

Sex eligibility

All sexes

Study type

Observational

Primary location

About this study

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 management practices and identify determinants that inform them.

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Primary outcomes

  1. Sinonasal symptoms and signs

    Time frame: at baseline

    Prevalence of reported clinical symptoms at different age groups, including rhinitis, sinusitis, snoring

  2. 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

  3. 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

  4. Otological symptoms and signs

    Time frame: at baseline

    Prevalence of reported clinical symptoms at different age groups, including ear pain, hearing problems, ear discharge

  5. 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

  6. 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

  7. Nasal endoscopy (description of nasal mucosa)

    Time frame: at baseline

    Description of nasal mucosa as seen via nasal endoscopy examination

  8. 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

  9. 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

  10. Nasal endoscopy (nasal polyps)

    Time frame: at baseline

    Prevalence of nasal polyps at baseline

  11. 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

  12. 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

  13. Sinonasal examination (Lidholdt score)

    Time frame: at baseline

    Lidholdt score at baseline

  14. Sinonasal examination (Lidholdt score at year 1)

    Time frame: at 12 months from recruitment

    Changes in Lidholdt score from baseline to 12 months

  15. Sinonasal examination (Lidholdt score at year 2)

    Time frame: at 24 months from recruitment

    Changes in Lidholdt score from baseline to 24 months

  16. Ear examination (ear discharge)

    Time frame: at baseline

    Prevalence of ear discharge at baseline

  17. 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

  18. 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

  19. Otoscopy (description of the tympanic membrane)

    Time frame: at baseline

    Description of tympanic membrane as seen via otoscopy examination

  20. 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

  21. 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

  22. Tympanometry

    Time frame: at baseline

    Results of tympanometry (tympanogram type for both ears) at baseline

  23. Tympanometry (at year 1)

    Time frame: at 12 months from recruitment

    Changes in tympanogram (for both ears) from baseline to 12 months

  24. Tympanometry (at year 2)

    Time frame: at 24 months from recruitment

    Changes in tympanogram (for both ears) from baseline to 24 months

  25. Audiometry

    Time frame: at baseline

    Results of audiometry: type of audiometry and results based on WHO hearing loss grades (for both ears)

  26. Audiometry (at year 1)

    Time frame: at 12 months from recruitment

    Changes in audiometry results from baseline to 12 months

  27. Audiometry (at year 2)

    Time frame: at 24 months from recruitment

    Changes in audiometry results from baseline to 24 months

Study contacts

Contact information is provided by the study sponsor or research team.

Myrofora Goutaki, MD-PhD

CONTACT

[email protected]

0041 316315973

Sponsors and collaborators

Lead sponsor

University of Bern

Other

Collaborators

  • Amsterdam UMC, location VUmc
  • Bicetre Hospital
  • Centre Hospitalier Intercommunal Creteil
  • Hacettepe University
  • Hospital Universitario La Fe
  • Insel Gruppe AG, University Hospital Bern
  • Marmara University
  • Universitaire Ziekenhuizen KU Leuven
  • University Hospital Southampton NHS Foundation Trust
  • University of Cyprus

Registry information

Acronym: EPIC-PCD

Important dates

Study start
2020
Primary completion
2030
Study completion
2040
First posted
Nov 2, 2020
Registry last updated
Dec 2, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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