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: (link sends e-mail)

To be included in this study, subjects must have completed the placebo controlled phase 3 study of PTC (PTC124-GD-009)
Study Name on (link is external)
Extension Study of Ataluren TM (PTC124) in cystic Fibrosis
Study DrugAtaluren TM (PTC124)
Type of Study DrugCFTR Modulation
Study TitleA Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis
Study Phase3
Study SponsorPTC Therapeutics

Link on (link is external) (link is external)

Participating ECFS-CTN sites

University Hospital Leuven
Leuven, Belgium

Hôpital Cochin
Paris, France

Hôpital Necker – Enfants Malades
Paris, France

Klinikum der Universität Köln
Cologne, Germany

Azienda Ospedaliera di Verona
Verona, Italy

Universitair Medisch Centrum Utrecht
Utrecht, Netherlands

Karolinska University Hospital, Huddinge
Stockholm, Sweden

Age6 years and older