The CF STORM Trial
Research type
Research Study
Full title
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 mucoactive therapies (the CF STORM trial)
IRAS ID
293186
Contact name
Kevin Southern
Contact email
Sponsor organisation
Alder Hey Children's NHS Foundation Trust
Eudract number
2020-005864-77
ISRCTN Number
ISRCTN14081521
Duration of Study in the UK
2 years, 11 months, 31 days
Research summary
For people with cystic fibrosis (pwCF), how to reduce treatment burden has been identified as the top priority research question. Nebulised treatments are identified by pwCF as burdensome. The CF STORM trial will recruit pwCF age 12 years and above that have been established on Kaftrio™ therapy. They will have an equal chance of being allocated to either stop or continue their daily nebulised mucoactive therapies (dornase alfa, hypertonic saline or both). The trial is designed to provide confidence that stopping these nebulisers does not result in a significant decline in lung function over 12 months. This will be measured using spirometry (Forced Expiratory Volume in one second), a lung function test that pwCF are familiar with as part of their routine clinical care. In addition to collecting information about hospital admissions and additional antibiotic treatments, patients will be asked to complete short quality of life measures. All clinical outcomes will be collected on the national CF Registry, and the trial will not involve extra visits outside of normal clinical care. The trial has been designed to be conducted remotely if necessary. This includes the informed consent process for taking part in the trial, and opportunity for remote monitoring of lung function. For patients with nebuliser devices that enable electronic data capture, we will request these data to monitor how participants are managing their nebuliser regimens during the trial. This is in addition to other initiatives that will monitor and encourage participants to comply with the trial group they were allocated to (stopping or continuing mucoactive nebulisers). The CF STORM trial results will be evaluated together with data from other research groups. This will ensure that clear messages relating to the role of nebulised mucoactive therapies once pwCF are on Kaftrio™ are promptly available for pwCF and their CF team.
REC name
London - London Bridge Research Ethics Committee
REC reference
21/LO/0080
Date of REC Opinion
2 Feb 2021
REC opinion
Favourable Opinion