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Updated: Aug 31, 2025

High-speed Video Microscopy Analysis for First-line Diagnosis of Primary Ciliary Dyskinesia
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High-speed Video Microscopy Analysis for First-line Diagnosis of Primary Ciliary Dyskinesia

Published on: January 19, 2022

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

Johanna Raidt1, Bernard Maitre2, Petra Pennekamp1

  • 1Dept of General Pediatrics, University Hospital Muenster, Muenster, Germany.

ERJ Open Research
|August 19, 2022
PubMed

Insights

A new European network for Primary Ciliary Dyskinesia (PCD) has been established to accelerate research and develop evidence-based treatments for this rare lung disease. This initiative aims to improve clinical trials and bring personalized therapies to patients with PCD.

Area of Science:

  • Rare diseases
  • Pulmonology
  • Clinical research networks

Background:

  • Primary ciliary dyskinesia (PCD) is a rare genetic disorder causing impaired mucociliary clearance and progressive lung damage.
  • Limited evidence-based treatments and clinical trials exist for PCD, unlike other rare lung diseases such as cystic fibrosis (CF).
  • Management of PCD is challenging due to diverse clinical presentations and disease severity, often relying on expert opinion.

Purpose of the Study:

  • To establish a dedicated clinical trial network (PCD-CTN) for Primary Ciliary Dyskinesia.
  • To improve clinical and translational research for PCD.
  • To facilitate the development of novel, evidence-based, and personalized treatments for PCD patients.

Main Methods:

  • The Primary Ciliary Dyskinesia Clinical Trial Network (PCD-CTN) was founded in 2020 under the European Reference Network (ERN)-LUNG PCD Core.
  • Inclusion criteria for trial sites included patient numbers, ERN-LUNG PCD Core membership, adherence to standards of care, research experience, resources, GCP certification, and institutional support.
  • Applications from 22 trial sites across 18 European countries were approved, encompassing over 1400 adults and 1600 pediatric patients with PCD.

Main Results:

  • The PCD-CTN has successfully recruited 22 trial sites in 18 European countries.
  • The network includes over 1400 adult and 1600 pediatric patients diagnosed with PCD.
  • The PCD-CTN is structured with a coordinating center, steering and executive committees, a data safety monitoring board, and protocol review/training committees.

Conclusions:

  • The newly formed PCD-CTN is poised to advance evidence-based treatments for this orphan disease.
  • The network aims to foster collaboration among European PCD sites to expedite the development of new therapies.
  • The ultimate goal is to deliver personalized treatment approaches to patients suffering from Primary Ciliary Dyskinesia.

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