Together against CF we have successfully:
In preparation for the multi-dose trial, we designed a study of almost 200 people with cystic fibrosis. The purpose of this study was to help design the multi-dose trial and to choose the patients most likely to be able to demonstrate benefit.
We recruited ~130 people with CF (aged 12 years and above) into the trial. The trial was a blinded, placebo-controlled study, meaning that each participant had a 50:50 chance of receiving gene therapy or 'dummy' treatment, with neither participants nor investigators knowing which.