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

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.