New trial aims to improve the lives of people with cystic fibrosis

The NIHR has awarded funding to the University of Liverpool and Alder Hey Children’s Hospital to conduct a trial to improve the lives of people with cystic fibrosis (CF).

The CF STORM trial aims to address how the treatment burden for people with CF could be safely reduced. Something that has been identified as a top research priority by patients and their healthcare teams.

People with CF spend many hours every day on multiple treatments, including aerosolised therapies delivered by nebulisers to keep their lungs stable and prevent chest infections. The recent UK introduction of the highly effective triple therapy drug Kaftrio, which is suitable for many people with CF, provides an ideal opportunity to robustly explore reduction in treatment burden in a clinical trial.

CF STORM will enrol people with CF across the UK who have been established on Kaftrio and take a daily nebulised muco-active therapy. It will evaluate whether their daily treatment regime can be rationalised without a significant reduction in their respiratory function.

The Chief Investigators are Professor Kevin Southern from the University of Liverpool and Dr Gwyneth Davies from University College London, and the trial will be run by the Liverpool Clinical Trials Centre.

The CF STORM team has worked closely with the Cystic Fibrosis Trust and other partners to involve people with CF and their families in the research design and process. The current challenges of the COVID-19 pandemic have also been taken into consideration and trial participants will require no extra hospital visits.

Professor Kevin 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”.

The project has been funded by the NIHR Health Technology Programme and more details about the CF STORM project can be found on our Funding and Awards website.