Primary Ciliary Dyskinesia (PCD) is a rare, progressive, inherited disease that affects the motile cilia of cells. It is incurable and frequently complicated by the development of bronchiectasis. Although earlier diagnosis and management lead to better outcomes, the diagnosis is often delayed.

Reducing exacerbations in children and adults with primary ciliary dyskinesia using erdosteine and/or azithromycin therapy (REPEAT) trial is a multicentre randomised controlled trial examining whether azithromycin and/or erdosteine over a 12 month period can reduce acute lung flare-ups in people with PCD. We will also determine if we can diagnose the disease earlier through in-depth genetic studies. REPEAT is being carried out across 5 sites (Northern Territory, Queensland, New South Wales, Victoria and Western Australia), in conjunction with consumer organisations, PCD Foundation Australia and the Lung Foundation of Australia. The study leaders are across these sites and the UK. 

The study is funded by a competitive grant from the Medical Research Future Fund (MRFF) and administered via Australia’s most prestigious funding body, the National Health and Medical Research Council (NHMRC). We plan to enrol 104 children and adults with PCD where approximately will receive the study medications and the other half will not.

Results from this study potentially lead to the prevention of recurrent chest infections. By doing this, it will likely reduce chronic lung disease and thus subsequently preserves lung health.