NCT00803205 | III | Ataluren | Nonsense mutations | ≥6 | No difference in primary or secondary outcomes Improved FEV1 seen in subgroup |
NCT00865904 | II | VX-809 | Phe508del (homozygous) | ≥18 | No significant difference observed |
NCT00909532 | III | Ivacaftor | Gly551Asp (one or more allele) | ≥12 | 10.4% increase in FEV1 48.7 mmol/L decrease in sweat chloride Well tolerated |
NCT00909727 | III | Ivacaftor | Gly551Asp (one or more allele) | 6–11 | 10.7% increase in FEV1 56-mmol·L−1 decrease in sweat chloride levels Well tolerated |
NCT00953706 | II | Ivacaftor | Phe508del (homozygous) | ≥12 | No significant difference observed |
NCT01225211 | II | VX-809 with or without ivacaftor | Phe508del (one or more allele) | ≥18 | April 2013 |
NCT01531673 | II | VX-661 with or without ivacaftor | Phe508del (homozygous) | ≥18 | August 2013 |
NCT01614457 | III | Ivacaftor | Arg117His (one or more allele) | ≥6 | July 2013 |
NCT01614470 | III | Ivacaftor | Non-Gly551Asp gating mutations (G178R, S549N, S549R, G551S, G970R, G1244E, S1251N, S1255P and G1349D) | ≥6 | February 2014 |
NCT01705145 | III | Ivacaftor | Any gating mutation | 2–5 | October 2013 |