The ECFS-CTN cannot guarantee that the information provided here is comprehensive. Please go to the ClinicalTrials.gov 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: ECFS-CTN@uzleuven.be (link sends e-mail)

Study Name on
ClinicalTrials.gov (link is external)
Extension Study of Ataluren for Nonsense Mutation Cystic Fibrosis
Study DrugAtaluren (PTC124®)
Type of Study DrugCFTR Modulation
Study TitleA Phase 3 Extension Study of Ataluren (PTC124) in Subjects With Nonsense-Mutation-Mediated Cystic Fibrosis
Study Phase3
Study SponsorPTC Therapeutics

Link on
clinicaltrials.gov (link is external)

https://clinicaltrials.gov/ct2/show/NCT02456103 (link is external)

Participating ECFS-CTN sitesBelgium: Leuven
France: Lyon, Montpellier, Roscoff, West-Paris (Necker)
Germany : Berlin, Cologne, Munich
Italy: Florence, Milan, Rome, Verona
The Netherlands: Rotterdam
Spain: Barcelona
Age6 Years and older