A randomised open label trial to assess change in respiratory function for people with cystic fibrosis (pwCF) established on triple combination therapy (Kaftrio™) after rationalisation of nebulised muco-active therapies (the CF STORM trial)

CF STORM will be a non-inferiority randomised open-label trial, undertaken on a national patient registry, to evaluate whether people with CF (pwCF) can rationalise their daily treatment without a significant reduction in their respiratory function. Patients established on triple therapy, elexacaftor-tezacaftor-ivacaftor (E-T-I), for more than three months will be enrolled and randomly allocated to either stop or continue their nebulised muco-active treatment (dornase alfa, hypertonic saline or both). The primary outcome will be change in percent predicted Forced Expiratory Volume in One Second (ppFEV1) at 12 months. This and other secondary outcomes (including need for extra antibiotic treatment, quality of life (QoL), and weight) will be collected on the national UK Cystic Fibrosis (CF) Registry. In addition, eligibility will be assessed, eConsent recorded, and randomisation will be undertaken on the registry. This provides opportunity for pwCF to be recruited remotely, in line with delivery of care during the COVID-19 pandemic. The results from the CF STORM trial, together with data from the SIMPLIFY trial (a shorter non-pragmatic trial being undertaken in the US) will inform the knowledge transfer exercise that will be undertaken by the CF STORM team at the end of the trial, co-ordinated by the PPI leads.