Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial

Kerem, E.; Konstan, M. W.; De Boeck, K.; Accurso, F. J.; Sermet-Gaudelus, I.; Wilschanski, M.; Elborn, J. S.; Melotti, P.; Bronsveld, I.; Fajac, I.; Malfroot, A.; Rosenbluth, D. B.; Walker, P. A.; McColley, S. A.; Knoop, C.; Quattrucci, S.; Rietschel, E.; Zeitlin, P. L.; Barth, J.; Elfring, G. L.; Welch, E. M.; Branstrom, A.; Spiegel, R. J.; Peltz, S. W.; Ajayi, T.; Rowe, S. M.

Lancet Respir Med. 2014 May 20; 2(7):539-47

Abstract

BACKGROUND: Ataluren was developed to restore functional protein production in genetic disorders caused by nonsense mutations, which are the cause of cystic fibrosis in 10% of patients. This trial was designed to assess the efficacy and safety of ataluren in patients with nonsense-mutation cystic fibrosis. METHODS: This randomised, double-blind, placebo-controlled, phase 3 study enrolled patients from 36 sites in 11 countries in North America and Europe. Eligible patients with nonsense-mutation cystic fibrosis (aged >/=6 years; abnormal nasal potential difference; sweat chloride >40 mmol/L; forced expiratory volume in 1 s [FEV1] >/=40% and

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