Clinical trial NCT04611516: The Ear-Nose-Throat (ENT) Prospective International Cohort of PCD Patients (EPIC-PCD)
Official title: The Ear-Nose-Throat (ENT) Prospective International Cohort of PCD Patients (EPIC-PCD)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Primary Ciliary Dyskinesia
Kartagener Syndrome
Lead sponsor: University of Bern
Sponsor class: OTHER
Collaborators: name: Bicetre Hospital
name: Centre Hospitalier Intercommunal Creteil
name: University Hospital Southampton NHS Foundation Trust
name: Universitaire Ziekenhuizen KU Leuven
name: Amsterdam UMC, location VUmc
name: University of Cyprus
name: Hacettepe University
name: Marmara University
name: Hospital Universitario La Fe
name: Insel Gruppe AG, University Hospital Bern
First posted: 2020-11-02
Enrollment: 1000
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients of all ages diagnosed with PCD, followed in a collaborating PCD centre

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.

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:

1. 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;
2. Study the association of sinonasal and otologic disease with lower respiratory disease in PCD patients;
3. 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.

Eligibility 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

Primary outcomes:
measure: Sinonasal symptoms and signs
timeFrame: at baseline
measure: Course of sinonasal symptoms and signs (year 1)
timeFrame: at 12 months from recruitment
measure: Course of sinonasal symptoms and signs (year 2)
timeFrame: at 24 months from recruitment
measure: Otological symptoms and signs
timeFrame: at baseline
measure: Course of otological symptoms and signs (year 1)
timeFrame: at 12 months from recruitment
measure: Course of otological symptoms and signs (year 2)
timeFrame: at 24 months from recruitment
measure: Nasal endoscopy (description of nasal mucosa)
timeFrame: at baseline
measure: Nasal endoscopy (changes of nasal mucosa at year 1)
timeFrame: at 12 months from recruitment
measure: Nasal endoscopy (changes of nasal mucosa at year 2)
timeFrame: at 24 months from recruitment
measure: Nasal endoscopy (nasal polyps)
timeFrame: at baseline
measure: Nasal endoscopy (nasal polyps at year 1)
timeFrame: at 12 months from recruitment
measure: Nasal endoscopy (nasal polyps at year 2)
timeFrame: at 24 months from recruitment
measure: Sinonasal examination (Lidholdt score)
timeFrame: at baseline
measure: Sinonasal examination (Lidholdt score at year 1)
timeFrame: at 12 months from recruitment
measure: Sinonasal examination (Lidholdt score at year 2)
timeFrame: at 24 months from recruitment
measure: Ear examination (ear discharge)
timeFrame: at baseline
measure: Ear examination (ear discharge at year 1)
timeFrame: at 12 months from recruitment
measure: Ear examination (ear discharge at year 2)
timeFrame: at 24 months from recruitment
measure: Otoscopy (description of the tympanic membrane)
timeFrame: at baseline
measure: Otoscopy (description of the tympanic membrane at year 1)
timeFrame: at 12 months from recruitment
measure: Otoscopy (description of the tympanic membrane at year 2)
timeFrame: at 24 months from recruitment
measure: Tympanometry
timeFrame: at baseline
measure: Tympanometry (at year 1)
timeFrame: at 12 months from recruitment
measure: Tympanometry (at year 2)
timeFrame: at 24 months from recruitment
measure: Audiometry
timeFrame: at baseline
measure: Audiometry (at year 1)
timeFrame: at 12 months from recruitment
measure: Audiometry (at year 2)
timeFrame: at 24 months from recruitment
Secondary outcomes:
Other outcomes:
Locations:
city: Bern
country: Switzerland
facility: University of Bern
Study officials:
affiliation: University of Bern
name: Myrofora Goutaki, MD-PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.