A Ph3 Extension Study of Ataluren (PTC124) in Patients with nm-CF


Trial status
Closed - follow up complete Participating Centres
Phase III

Full title

A Phase 3 Extension Study of Ataluren (PTC124®) in Patients With Nonsense Mutation Cystic Fibrosis

Ataluren is a novel investigational drug which works by helping the body to ignore the nonsense mutation
and make a complete and functional CFTR protein. So far the results from clinical trials in ataluren have
shown that the drug appears to be safe and well tolerated.
There is currently an ongoing clinical trial (PTC124-GD-021-CF) which assesses the safety and
effectiveness of ataluren in 208 patients worldwide with nmCF who are 6 years or older. After 48 weeks of
treatment with ataluren or placebo, patients who successfully complete this study will have the chance to go
onto this extension study (PTC124-GD-021e-CF).
There will be 9 study visits. Study procedures will include spirometry, blood tests, physical examination,
vital signs, sputum, urine sample collection, ECG, questionnaires and renal ultrasound.
The study is sponsored by PTC Therapeutics Inc.
Trial Reference Number
Recruitment target
Last edited date

Who can take part?

Your donation will make a difference:

Select amount