The UK’s National Institute for Health Research (NIHR) has granted funding to the University of Liverpool and Alder Hey Children’s Hospital for a trial aiming to improve the lives of people living with cystic fibrosis (CF).
The trial, called CF STORM, will seek to address how the treatment burden for CF patients can be safely reduced – identified as a top research priority by patients and their healthcare teams.
The CF STORM study will enrol people with CF from across the UK, who have been established on Vertex’s Kaftrio (ivacaftor/tezacaftor /elexacaftor) and take daily nebulised muco-active therapy.
Kaftrio, a triple therapy drug, was recently introduced in the UK and is suitable for the treatment of many people living with CF.
‘[The introduction of Kaftrio in the UK] provides an ideal opportunity to robustly explore reduction in treatment burden in a clinical trial,’ the NIHR said in a statement.
It will evaluate if the daily treatment regime for these patients can be rationalised without a significant reduction in respiratory function.
The CF STORM research team has worked with the Cystic Fibrosis Trust, as well as other partners, to involve people living with CF and their families in the research design and process.