RT Journal Article
SR Electronic
T1 The disease-specific clinical trial network for primary ciliary dyskinesia: PCD-CTN
JF ERJ Open Research
JO erjor
FD European Respiratory Society
SP 00139-2022
DO 10.1183/23120541.00139-2022
VO 8
IS 3
A1 Johanna Raidt
A1 Bernard Maitre
A1 Petra Pennekamp
A1 Josje Altenburg
A1 Pinelopi Anagnostopoulou
A1 Miguel Armengot
A1 Lizan D. Bloemsma
A1 Mieke Boon
A1 Melissa Borrelli
A1 Folke Brinkmann
A1 Siobhan B. Carr
A1 Mary P. Carroll
A1 Silvia Castillo-Corullón
A1 André Coste
A1 Renato Cutrera
A1 Eleonora Dehlink
A1 Damien M.S. Destouches
A1 Maria E. Di Cicco
A1 Lucy Dixon
A1 Nagehan Emiralioglu
A1 Ela Erdem Eralp
A1 Eric G. Haarman
A1 Claire Hogg
A1 Bulent Karadag
A1 Helene E. Kobbernagel
A1 Natalie Lorent
A1 Marcus A. Mall
A1 June K. Marthin
A1 Vendula Martinu
A1 Manjith Narayanan
A1 Ugur Ozcelik
A1 Daniel Peckham
A1 Massimo Pifferi
A1 Petr Pohunek
A1 Eva Polverino
A1 Simon Range
A1 Felix C. Ringshausen
A1 Evie Robson
A1 Jobst Roehmel
A1 Sandra Rovira-Amigo
A1 Francesca Santamaria
A1 Anne Schlegtendal
A1 Zsolt Szépfalusi
A1 Petra Tempels
A1 Guillaume Thouvenin
A1 Nicola Ullmann
A1 Woolf T. Walker
A1 Martin Wetzke
A1 Panayiotis Yiallouros
A1 Heymut Omran
A1 Kim G. Nielsen
YR 2022
UL http://openres.ersjournals.com/content/8/3/00139-2022.abstract
AB 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.The disease-specific clinical trial network for primary ciliary dyskinesia (PCD-CTN) was built under the framework of the European Reference Network (ERN)-LUNG PCD Core, and operates to establish evidence-based and new personalised treatment for PCD https://bit.ly/3sLtC8o