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The disease-specific clinical trial network for primary ciliary dyskinesia: PCD-CTN
J. Raidt, B. Maitre, P. Pennekamp, J. Altenburg, P. Anagnostopoulou, M. Armengot, LD. Bloemsma, M. Boon, M. Borrelli, F. Brinkmann, SB. Carr, MP. Carroll, S. Castillo-Corullón, A. Coste, R. Cutrera, E. Dehlink, DMS. Destouches, ME. Di Cicco, L....
Language English Country Great Britain
Document type Journal Article
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PubMed Central
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- Publication type
- Journal Article MeSH
Primary ciliary dyskinesia (PCD) is a rare genetic disorder characterised by impaired mucociliary clearance leading to irreversible lung damage. In contrast to other rare lung diseases like cystic fibrosis (CF), there are only few clinical trials and limited evidence-based treatments. Management is mainly based on expert opinions and treatment is challenging due to a wide range of clinical manifestations and disease severity. To improve clinical and translational research and facilitate development of new treatments, the clinical trial network for PCD (PCD-CTN) was founded in 2020 under the framework of the European Reference Network (ERN)-LUNG PCD Core. Applications from European PCD sites interested in participating in the PCD-CTN were requested. Inclusion criteria consisted of patient numbers, membership of ERN-LUNG PCD Core, use of associated standards of care, experience in PCD and/or CF clinical research, resources to run clinical trials, good clinical practice (GCP) certifications and institutional support. So far, applications from 22 trial sites in 18 European countries have been approved, including >1400 adult and >1600 paediatric individuals with PCD. The PCD-CTN is headed by a coordinating centre and consists of a steering and executive committee, a data safety monitoring board and committees for protocol review, training and standardisation. A strong association with patient organisations and industrial companies are further cornerstones. All participating trial sites agreed on a code of conduct. As CTNs from other diseases have demonstrated successfully, this newly formed PCD-CTN operates to establish evidence-based treatments for this orphan disease and to bring new personalised treatment approaches to patients.
Association des Patients Ayant une Dyskinésie Ciliaire Primitive Saint Etienne France
Dept of General Pediatrics University Hospital Muenster Muenster Germany
Dept of Pediatric Pulmonology Hacettepe University Faculty of Medicine Ankara Turkey
Dept of Pediatrics University Hospital Leuven Leuven Belgium
Dept of Pulmonology Amsterdam UMC Location AMC Amsterdam the Netherlands
Dept of Respiratory Medicine Amsterdam UMC University of Amsterdam Amsterdam the Netherlands
Dept of Respiratory Medicine Hannover Medical School and DZL Hannover Germany
Dept of Respiratory Medicine University Hospitals of Leicester Leicester UK
German Center for Lung Research Berlin Germany
Kartagener Syndrom and Primäre Ciliäre Dyskinesie e 5 Hannover Germany
Medical School University of Cyprus Nicosia Cyprus
Paediatric Pulmonology Unit Hospital Clínico Universitario Valencia Valencia Spain
PCD Diagnosis and Research Dept of Respiratory Sciences University of Leicester Leicester UK
Primary Ciliary Dyskinesia Management Centre Leeds Teaching Hospitals NHS Trust Leeds UK
Pulmonology and Allergology Section Unit of Pediatrics Pisa University Hospital Pisa Italy
References provided by Crossref.org
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