Clinical Programme
We started our clinical programme in July 2006
Run-in Study
So-called because it is envisaged that this study will run into our
future multi-dose gene therapy trial, this study began in January 2008.
We planned to recruit up to 200 CF patients aged 10 and above with a
wide range of disease severities.
The purpose of the study is to assess the stability and repeatability of a wide range of tests which may be useful outcome measures in our gene therapy trials and to decide which patients might be most suitable to take forward into the multi-dose trial. Patients are seen at regular intervals when they undergo various measurements reflecting lung function, bacterial infection, inflammation and other measures of clinical severity including quality of life questionnaires. In addition, on a single occasion, we are asking all patients to travel to Southampton for a specialised scan measuring mucociliary clearance (this is the main way that the lungs defend themselves from infection and does not work properly in patients with CF). We realise that this has been a great deal of effort for patients and their families and are grateful that participants have been prepared to take this on.
Recruitment closed in August 2008 with 192 patients enrolled between London and Edinburgh, although for various reasons, several patients had to drop out of the study.