The drug, which was made available on the NHS following approval by European regulators last year, had previously been tested within clinical trials across the world, including at MFT. At our Trust, the research involved adult participants at the NIHR Manchester Clinical Research Facility (CRF) at Wythenshawe Hospital, as well as paediatric participants at Manchester CRF at Royal Manchester Children’s Hospital (RMCH).
Cystic fibrosis (CF) is a rare genetic disease that affects around 10,000 people in the UK and is caused by mutations in the CFTR gene.
Kaftrio is a breakthrough therapy, known as a CFTR modulator, because it targets the core defect rather than the effects of the disease, and it could be suitable for 90 per cent of patients with CF.
In the last few years MFT has successfully delivered many clinical trials of this class of medications, both at Wythenshawe Hospital and at RMCH. Researchers from MACFC have served as national and global leads on a number of pivotal studies, while RMCH was the first UK centre to recruit patients in the Kaftrio trial.
Dr Anirban Maitra