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The disease-specific clinical trial network for primary ciliary dyskinesia: PCD-CTN

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The disease-specific clinical trial network for primary ciliary dyskinesia: PCD-CTN

dc.contributor.author	Raidt, Johanna
dc.contributor.author	Maitre, Bernard
dc.contributor.author	Pennekamp, Petra
dc.contributor.author	Altenburg, Josje
dc.contributor.author	Anagnostopoulou, Pinelopi
dc.contributor.author	Armengot Carceller, Miguel
dc.contributor.author	Bloemsma, Lizan D.
dc.contributor.author	Boon, Mieke
dc.contributor.author	Borrelli, Melissa
dc.contributor.author	Brinkmann, Folke
dc.contributor.author	Carr, Siobhan B.
dc.contributor.author	Carroll, Mary P.
dc.contributor.author	Castillo-Corullón, Silvia
dc.contributor.author	Coste, André
dc.contributor.author	Cutrera, Renato
dc.contributor.author	Dehlink, Eleonora
dc.contributor.author	Destouches, Damien M.S.
dc.contributor.author	Di Cicco, Maria E.
dc.contributor.author	Dixon, Lucy
dc.contributor.author	Emiralioglu, Nagehan
dc.contributor.author	Erdem Eralp, Ela
dc.contributor.author	Haarman, Eric G.
dc.contributor.author	Hogg, Claire
dc.contributor.author	Karadag, Bulent
dc.contributor.author	Kobbernagel, Helene E.
dc.contributor.author	Lorent, Natalie
dc.contributor.author	Mall, Markus A.
dc.contributor.author	Marthin, June K.
dc.contributor.author	Martinu, Venula
dc.contributor.author	Narayanan, Manjith
dc.contributor.author	Ozcelik, Ugur
dc.contributor.author	Peckham, Daniel
dc.contributor.author	Pifferi, Massimo
dc.contributor.author	Pohunek, Petr
dc.contributor.author	Polverino, Eva
dc.contributor.author	Range, Simon
dc.contributor.author	Ringshausen, Felix C.
dc.contributor.author	Robson, Evie
dc.contributor.author
dc.contributor.author	Rovira-Amigo, Sandra
dc.contributor.author	et al.
dc.date.accessioned	2023-02-23T13:10:37Z
dc.date.available	2023-02-23T13:10:37Z
dc.date.issued	2022
dc.identifier.citation	Raidt, Johanna Maitre, Bernard Pennekamp, Petra Altenburg, Josje Anagnostopoulou, Pinelopi Armengot Carceller, Miguel Bloemsma, Lizan D. Boon, Mieke Borrelli, Melissa Brinkmann, Folke Carr, Siobhan B. Carroll, Mary P. Castillo-Corullón, Silvia Coste, André Cutrera, Renato Dehlink, Eleonora Destouches, Damien M.S. Di Cicco, Maria E. Dixon, Lucy Emiralioglu, Nagehan Erdem Eralp, Ela Haarman, Eric G. Hogg, Claire Karadag, Bulent Kobbernagel, Helene E. Lorent, Natalie Mall, Markus A. Marthin, June K. Martinu, Venula Narayanan, Manjith Ozcelik, Ugur Peckham, Daniel Pifferi, Massimo Pohunek, Petr Polverino, Eva Range, Simon Ringshausen, Felix C. Robson, Evie Rovira-Amigo, Sandra et al. 2022 The disease-specific clinical trial network for primary ciliary dyskinesia: PCD-CTN Erj Open Research 15;8(3):00139 15;8(3):00139
dc.identifier.uri	https://hdl.handle.net/10550/85525
dc.description.abstract	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
dc.language.iso	eng
dc.relation.ispartof	Erj Open Research, 2022, vol. 15;8(3):00139, num. 15;8(3):00139
dc.subject	Pulmons Malalties
dc.title	The disease-specific clinical trial network for primary ciliary dyskinesia: PCD-CTN
dc.type	journal article	es_ES
dc.date.updated	2023-02-23T13:10:37Z
dc.identifier.doi	10.1183/23120541.00139-2022
dc.identifier.idgrec	156980
dc.rights.accessRights	open access	es_ES