CF Lung Repair V 1.0
Research type
Research Study
Full title
Investigating Lung Repair Following Highly Effective CFTR Modulator Therapy
IRAS ID
286836
Contact name
Robert Gray
Contact email
Sponsor organisation
University of Edinburgh
Duration of Study in the UK
1 years, 9 months, 28 days
Research summary
Cystic fibrosis is a common genetic disorder with a prevalence of 1 in 2500 live births. CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. CF is a multi-organ disease but the majority of patients die from lung disease. The advent of highly effective CFTR modulator therapies may lead to the progression of CF being slowed or even halted. CFTR modulators turn the CF protein which is faulty back on and leads to increases in lung function, improved symptoms and less need for hospital admissions. We now wish to assess whether spontaneous repair occurs in the lungs following CFTR modulator therapy and whether the damage that has already occurred can be reversed.
We will sample nasal cells peripheral blood and sputum from 150 CF patients (aged 12+) before and after commencing highly effective CFTR modulator therapy at 0 months, 1 month, 3 months, 6 months, 12 months and 18 months. A sub-group of adult patients who have never had a CFTR modulator before (20) will be asked to attend for CT scans at 0 months, 1 month, and 12 months.
REC name
North of Scotland Research Ethics Committee 2
REC reference
20/NS/0106
Date of REC Opinion
11 Sep 2020
REC opinion
Favourable Opinion