The disease-specific clinical trial network for primary ciliary dyskinesia: PCD-CTN
The result's identifiers
Result code in IS VaVaI
<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00216208%3A11130%2F22%3A10446021" target="_blank" >RIV/00216208:11130/22:10446021 - isvavai.cz</a>
Alternative codes found
RIV/00064203:_____/22:10446021
Result on the web
<a href="https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=7gKbmmGi1C" target="_blank" >https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=7gKbmmGi1C</a>
DOI - Digital Object Identifier
<a href="http://dx.doi.org/10.1183/23120541.00139-2022" target="_blank" >10.1183/23120541.00139-2022</a>
Alternative languages
Result language
angličtina
Original language name
The disease-specific clinical trial network for primary ciliary dyskinesia: PCD-CTN
Original language description
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.
Czech name
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Czech description
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Classification
Type
J<sub>imp</sub> - Article in a specialist periodical, which is included in the Web of Science database
CEP classification
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OECD FORD branch
30209 - Paediatrics
Result continuities
Project
<a href="/en/project/NV19-07-00210" target="_blank" >NV19-07-00210: Primary ciliary dyskinesia: Genetic, structural and functional determinants of the disease course and prognosis.</a><br>
Continuities
P - Projekt vyzkumu a vyvoje financovany z verejnych zdroju (s odkazem do CEP)
Others
Publication year
2022
Confidentiality
S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů
Data specific for result type
Name of the periodical
ERJ Open Research [online]
ISSN
2312-0541
e-ISSN
2312-0541
Volume of the periodical
8
Issue of the periodical within the volume
3
Country of publishing house
GB - UNITED KINGDOM
Number of pages
11
Pages from-to
139
UT code for WoS article
000841851600002
EID of the result in the Scopus database
2-s2.0-85137195374