Lancet Respir Med 2021 May 6. Epub 2021 May 6.
Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL, USA; Stanley Manne Children's Research Institute, Chicago, IL, USA; Ann & Robert H Lurie Children's Hospital of Chicago, Chicago, IL, USA.
Background: A previous phase 3 study showed that lumacaftor-ivacaftor was generally safe and well tolerated over 24 weeks of treatment in children aged 2-5 years with cystic fibrosis homozygous for the F508del-CFTR mutation. In this study, we aimed to assess the long-term safety of lumacaftor-ivacaftor in a rollover study of children who participated in this previous phase 3 study.
Methods: In this multicentre, phase 3, open-label, extension study (study 116; VX16-809-116), we assessed safety of lumacaftor-ivacaftor in children included in a previous multicentre, phase 3, open-label study (study 115; VX15-809-115). Read More