A Phase 3 Open-label Study Evaluating the Long?term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 2 Years and Older
Vertex 445-112 OLE 2-5 YR
What is the goal of the study?
This multicenter, open-label extension study will provide data on the long-term safety, efficacy, and durability of a CFTR modulator (elexacaftor/tezacaftor/ivacaftor, Trikafta) in children with cystic fibrosis (CF) who are 2 years of age and older with 1 or 2 copies of the F508del CF mutation.Trikafta is FDA approved for use in people with CF who are 6 years of age and older. Participants in this study will have completed the VX20-445-111 study and will be enrolled for up to 96 weeks. Study visits to assess growth and lung function will be conducted with ECGs, eye exams and blood, urine, stool and sweat samples collected to evaluate safety and efficacy. Adverse events will be assessed throughout the study period. Participants will have the option to early terminate and transition to clinically prescribed Trikafta if FDA approved for children 2 and older during the study.
Who can participate in the study?
Please contact the study team listed below to learn more.