Phase 1

The clinical trials that led to the approval of the drug Kaftrio were conducted without taking into account its effect in people with advanced cystic fibrosis (CF). The aim of this study is to evaluate the effects of Kaftrio in the medium-long term period in people with advanced CF lung disease in terms of efficacy and safety. This is therefore a post-marketing study involving about 200 people with advanced CF, 12 years of age or older carrying an F508del mutation on one allele and a “minimal function” mutation on the second allele. The study will be conducted in collaboration with the Italian FC Society (SIFC) and with all the Italian regional centers that have at least one person with CF under treatment with Kaftrio.