Figures
- FIGURE 1
Map showing the participating centres of the clinical trial network for primary ciliary dyskinesia (PCD-CTN) (red dots). The PCD-CTN consists of 22 trial sites from 12 European countries including >3000 children and adults with PCD. The map was created with R [16] and RStudio [17], the packages “maps” [18], “ggplot2” [19] and “dplyr” [20] were used.
- FIGURE 2
Organisational diagram representing the structure of the clinical trial network for primary ciliary dyskinesia (PCD-CTN). The PCD-CTN was founded under the framework of the European Respiratory Network (ERN)-LUNG PCD Core in 2020. Core structures are the steering committee (SC), the executive committee (EC) and the coordinating centre. The coordinating centre provides a director and an academic secretary. The EC includes the PCD-CTN director and both deputies, the ERN-LUNG PCD Core coordinator and one additional ERN-LUNG PCD Core member representing three different European trial sites. The SC is composed of one principal investigator from each PCD-CTN member site, all members of the EC, the chair of the additional committees, and all appointed patient–parent representatives. The PCD-CTN includes several subcommittees: a committee in charge of data safety monitoring and committees for protocol review, training and standardisations of diagnostic procedures and important outcome measures. Important cornerstones of the PCD-CTN are a strong association with the ERN-LUNG International PCD Registry, the patient organisations and partnering networks, like the PCD Foundation in the USA.
Tables
- TABLE 1
Details on available information from all participating centres of the PCD-CTN
Information requested At application for membership At yearly overall feasibility survey Patients with PCD: overview Paediatric, adult or mixed centre x Number of patients currently under direct care at the centre (adults/children) x x Number of patients within different age groups (0–2 years/ 2–6 years/6–12 years/12–18 years/>18 years) x Number of patients with genetically confirmed diagnosis x Number of patients with specific bi-allelic mutations x Experience Industry-sponsored trials (PCD or CF-related; past 5 years) x x Investigator-initiated trials (PCD or CF-related; past 5 years) x x GCP certifications x x Inspection of a regulatory authority x x Publications (PCD, non-CF bronchiectasis and CF-related publications (past 5 years)) x Site information Ethic committee (local, central) x x Monitoring regulations x x Experience with the use of EDC systems x x Resources for clinical research (personnel, database, available outcome parameters, available clinical research facility) x Institutional support x Commitment to work within PCD-CTN (hours dedicated to work for the CTN/month) x Diagnostics and follow-up Frequency of outpatient visits per patient x x SOPs for diagnostic methods and monitoring tools for patient follow-up and for study outcome parameters on site x x Participation in a national programme to improve quality in PCD care x Participation and entering patients in a registry x Recruitment strategies typically used at your centre x Treatment practice Fraction of patients with distinct medication (e.g. hydrator therapy, mucolytics, chronic antibiotic therapy, bronchodilators, inhaled corticosteroids, physiotherapy) x Multidisciplinary team (PCD/CF dedicated) x CF: cystic fibrosis; CTN: clinical trial network; GCP: good clinical practice; EDC: electronic data capture; PCD: primary ciliary dyskinesia; SOP: standard operating procedure
