Nebuliser which would be used to treat cystic fibrosis

New trial seeks to reduce cystic fibrosis treatment burden

Researchers at the University of Liverpool and Alder Hey Children’s Hospital are set to begin a new clinical trial aimed at improving the lives of people with cystic fibrosis (CF).

NIHR has awarded funding to the study through the NIHR Health Technology Programme.

The study which will seek to address how the treatment burden for people with CF could be safely reduced - an ambition which has been identified by patients and their healthcare teams as a top research priority.

For many people with CF, their care can involve spending many hours each day on multiple treatments, including aerosolised therapies delivered by nebulisers, to keep their lungs stable and prevent chest infections.

Recently the UK approved and introduced a highly-effective triple therapy drug, suitable for many people with CF, which provides researchers with an ideal opportunity to more robustly explore potential reductions in treatment burden.

The CF STORM clinical trial will enrol people with the condition from across the UK who have been established on the new drug combination and take a daily nebulised muco-active therapy.

It will then look to evaluate whether their daily treatment regime can be rationalised without a significant reduction in their respiratory function.

Professor Kevin Southern from the University of Liverpool and Dr Gwyneth Davies from University College London will lead the clinical trial, which is set to be run by the Liverpool Clinical Trials Centre.

Professor Southern, from the Department of Women’s and Children’s Health, said: “As transformative therapies emerge for CF and other conditions it is important that we evaluate rigorously the impact on the patient journey”.

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