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

NCT04611516 | Recruiting DMC

• 1 other identifier: EPIC-PCD
• Study Type: observational
• Target: 1,000
• Locations: 1 country
• Sites: 1

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.

Conditions

Primary Ciliary Dyskinesia; Kartagener Syndrome

Outcome Measures

Primary Outcomes (27)

• Sinonasal symptoms and signs

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

  at baseline

• Course of sinonasal symptoms and signs (year 1)

  Change from baseline prevalence of reported clinical symptoms at different age groups, including rhinitis, sinusitis, snoring, at 12 months

  at 12 months from recruitment

• Course of sinonasal symptoms and signs (year 2)

  Change from baseline prevalence of reported clinical symptoms at different age groups, including rhinitis, sinusitis, snoring, at 24 months

  at 24 months from recruitment

• Otological symptoms and signs

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

  at baseline

• Course of otological symptoms and signs (year 1)

  Change from baseline prevalence of reported clinical symptoms at different age groups, including ear pain, hearing problems, ear discharge, at 12 months

  at 12 months from recruitment

• Course of otological symptoms and signs (year 2)

  Change from baseline prevalence of reported clinical symptoms at different age groups, including ear pain, hearing problems, ear discharge, at 24 months

  at 24 months from recruitment

• Nasal endoscopy (description of nasal mucosa)

  Description of nasal mucosa as seen via nasal endoscopy examination

  at baseline

• Nasal endoscopy (changes of nasal mucosa at year 1)

  Changes in nasal mucosa from baseline as seen via nasal endoscopy examination at 12 months

  at 12 months from recruitment

• Nasal endoscopy (changes of nasal mucosa at year 2)

  Changes in nasal mucosa from baseline as seen via nasal endoscopy examination at 24 months

  at 24 months from recruitment

• Nasal endoscopy (nasal polyps)

  Prevalence of nasal polyps at baseline

  at baseline

• Nasal endoscopy (nasal polyps at year 1)

  Changes in prevalence of nasal polyps from baseline to 12 months

  at 12 months from recruitment

• Nasal endoscopy (nasal polyps at year 2)

  Changes in prevalence of nasal polyps from baseline to 24 months

  at 24 months from recruitment

• Sinonasal examination (Lidholdt score)

  Lidholdt score at baseline

  at baseline

• Sinonasal examination (Lidholdt score at year 1)

  Changes in Lidholdt score from baseline to 12 months

  at 12 months from recruitment

• Sinonasal examination (Lidholdt score at year 2)

  Changes in Lidholdt score from baseline to 24 months

  at 24 months from recruitment

• Ear examination (ear discharge)

  Prevalence of ear discharge at baseline

  at baseline

• Ear examination (ear discharge at year 1)

  Changes in prevalence of ear discharge from baseline to 12 months

  at 12 months from recruitment

• Ear examination (ear discharge at year 2)

  Changes in prevalence of ear discharge from baseline to 24 months

  at 24 months from recruitment

• Otoscopy (description of the tympanic membrane)

  Description of tympanic membrane as seen via otoscopy examination

  at baseline

• Otoscopy (description of the tympanic membrane at year 1)

  Changes in the tympanic membrane from baseline to 12 months as seen via otoscopy examination

  at 12 months from recruitment

• Otoscopy (description of the tympanic membrane at year 2)

  Changes in the tympanic membrane from baseline to 24 months as seen via otoscopy examination

  at 24 months from recruitment

• Tympanometry

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

  at baseline

• Tympanometry (at year 1)

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

  at 12 months from recruitment

• Tympanometry (at year 2)

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

  at 24 months from recruitment

• Audiometry

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

  at baseline

• Audiometry (at year 1)

  Changes in audiometry results from baseline to 12 months

  at 12 months from recruitment

• Audiometry (at year 2)

  Changes in audiometry results from baseline to 24 months

  at 24 months from recruitment

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients of all ages diagnosed with PCD, followed in a collaborating PCD centre

You may qualify if:

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

You may not qualify if:

• None

Sponsors & Collaborators

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

Study Sites (1)

University of Bern

Bern, Switzerland

RECRUITING

Related Publications (1)

• 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.

  PMID: 34635525 DERIVED

MeSH Terms

Conditions

Ciliary Motility Disorders; Kartagener Syndrome

Condition Hierarchy (Ancestors)

Respiratory Tract Diseases; Otorhinolaryngologic Diseases; Ciliopathies; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Bronchiectasis; Bronchial Diseases; Respiratory System Abnormalities; Dextrocardia; Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Situs Inversus

Study Officials

• Myrofora Goutaki, MD-PhD

  University of Bern

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Myrofora Goutaki, MD-PhD

CONTACT

0041 316315973; myrofora.goutaki@unibe.ch

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 8, 2020
• First Posted: November 2, 2020
• Study Start: March 2, 2020
• Primary Completion (Estimated): December 1, 2030
• Study Completion (Estimated): December 1, 2040
• Last Updated: December 2, 2025

Record last verified: 2025-11

Locations

CH (1)