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Update in WP 5

The first randomized, controlled trial on pharmacotherapy in PCD started in spring 2014. More than 25% of the planned participants have now been randomized and 10% have already completed the trial!

The aim of BESTCILIA Work Package 5 (WP5) is to take the first step in developing evidence-based therapeutic guidelines for PCD. The main focus of WP5 is investigating the efficacy and safety of maintenance therapy with the authorized antibiotic azithromycin in PCD patients. This will be achieved by conducting the first randomized, controlled trial for this rare disease. Azithromycin is already widely used in chronic pulmonary disorders; however, the efficacy of maintenance therapy with azithromycin in PCD has not been evaluated previously.

The BESTCILIA project has provided the opportunity to set up a multi-national trial, which will include PCD patients from Denmark, Germany, the Netherlands, Switzerland and the United Kingdom. After completing intense preparatory work on the design of the trial, implementing similar study equipment and procedures in all trial sites and obtaining approvals from both competent authorities and ethics committees in the five European countries, the conduction of the trial has started. Patient recruitment commenced in Denmark in spring 2014 and is currently ongoing in all six trial sites. The goal is to include 125 PCD patients between 7 and 50 years of age in the trial. To date, more than 25% of the planned number of participants has been randomized and 10% have already completed the trial. More PCD patients have been screened for the trial and will soon be ready for randomization.

The outcome measures in the trial, besides respiratory system exacerbations, lung function, sputum microbiology, inflammatory markers and hearing impairment, include two new outcome measures in PCD: The PCD-specific health-related quality of life questionnaires (QOL-PCD), which were developed in BESTCILIA Work Package 4, and Nitrogen Multiple Breath Washout from which the Lung Clearance Index (LCI) is derived.  

This trial will facilitate future randomized, controlled trials in PCD and promote the development of evidence-based therapeutic guidelines.