Primary ciliary dyskinesia (PCD), is a rare, incurable, progressive, serious disorder with a large unmet need in both the diagnosis & treatment. To date, all currently used therapies are unproven. Our parallel, multicentre, 2x2 factorial design, double-blinded, double-dummy, superiority randomised controlled trial (concealed 1:1:1:1 allocation) will test the benefits of a currently used (but unapproved for long term use in PCD) antibiotic (azithromycin) and a novel mucolytic (erdosteine). Our primary question is: among children/young adults with PCD, does azithromycin or erdosteine reduce acute respiratory exacerbations during 12 months of treatment?

https://hdl.handle.net/10137/12790

Primary ciliary dyskinesia

REPEAT Study: Improving outcomes of children and young adults with primary ciliary dyskinesia: a multi-centre, double blind, double-dummy, 2x2 partial factorial, randomised controlled trial

Interventional/Clinical trials research

Project