The ECFS-CTN cannot guarantee that the information provided here is comprehensive. Please go to the link for detailed and up-to-date information on study background, eligibility criteria, study design, outcome measures, study status, participating sites, sponsor contact information and timelines.

You can also contact the CTN coordinating centre for additional information:

Study Name on (link is external)
A Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation
Study Drug Lumacaftor/Ivacaftor
Type of Study Drug CFTR modulator
Study Title An Exploratory Phase 2, 2-part, Randomized, Double blind, Placebo controlled Study With a Long term, Open label Period to Explore the Impact of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del
Study Phase 2a
Study Sponsor Vertex

Link on (link is external)

Participating ECFS-CTN sites Please contact for most recent sitelist
Age 2 through 5  years of age