skip to main content

Update in Work Package 5

(Progress in Work Package 5)

The first randomized, controlled trial on pharmacotherapy in PCD has now started! Patient recruitment started in spring 2014 and the first patients were included in the trial in June 2014. The aim of Bestcilia Work Package 5 (WP5) is to investigate the efficacy and safety of the antibiotic azithromycin as maintenance therapy in PCD patients.

Azithromycin is a widely used authorized antibiotic, and maintenance therapy with azithromycin has been shown to reduce the number of exacerbations and/or improve pulmonary function in patients with cystic fibrosis and patients with non-cystic fibrosis bronchiectasis. However, the efficacy of this antibiotic as maintenance therapy in PCD patients has not been previously evaluated.

This multi-national trial will include PCD patients from five European countries: Denmark, Germany, the Netherlands, Switzerland and the United Kingdom. In the past year the partners have been preparing applications for obtaining the required approvals from health authorities and ethics committees in each of the participating countries. The conduction of the trial has been approved in Denmark and Germany and the final approvals in the remaining countries are expected to be in place within the next month.

Patient recruitment started in Denmark in spring 2014, and to date seven patients have been screened. Within the next month, about 10 more Danish patients are planned to be screened  for the trial. The trial centre in Germany is now starting recruitment and screening of German patients. The goal is to include 125 PCD patients in this very first randomized, controlled trial in PCD.

This trial will also explore two new outcome measures in PCD: The PCD-specific health related quality of life questionnaires (QOL-PCD), which has been developed in BESTCILIA Work Package 4, and Nitrogen Multiple Breath Washout from which the Lung Clearance Index (LCI) is derived.