@article {McKone1842, author = {Edward McKone and Sally Rodriguez and Karl Yen and Jane Davies}, title = {Long-term safety and efficacy of ivacaftor in subjects with CF who have the G551D-CFTR mutation}, volume = {40}, number = {Suppl 56}, elocation-id = {1842}, year = {2012}, publisher = {European Respiratory Society}, abstract = {Objectives: Ivacaftor was evaluated in 2 48-week Phase 3 studies. This 96-week open-label extension (PERSIST) evaluated the safety and efficacy of long-term ivacaftor in subjects who completed the prior trials (STRIVE and ENVISION).Methods: 192 subjects who completed treatment in STRIVE or ENVISION enrolled in PERSIST. All subjects received ivacaftor 150 mg q12h in addition to prescribed therapies. Here we report the results of 96 weeks of treatment for subjects who were in STRIVE (n=144) and 72 weeks for those in ENVISION (n=48).Results: For subjects who received ivacaftor in the placebo-controlled studies, the FEV1 improvements were sustained in PERSIST. For STRIVE subjects, the mean (SD) absolute change from STRIVE baseline in \% predicted FEV1 was 9.4\% (8.3\%) on Day 1 of PERSIST (Week 48 in STRIVE), 10.3\% (9.3\%) at Week 72, and 9.5\% (10.1\%) at Week 96. For ENVISION subjects, the mean (SD) absolute change from ENVISION baseline was 10.2\% (15.7\%) on Day 1 of PERSIST, 9.1\% (15.6\%) at Week 60, and 10.1\% (14.2\%) at Week 72. For placebo subjects in STRIVE or ENVISION, the FEV1 improvements in PERSIST were similar to those observed in ivacaftor subjects during the placebo-controlled studies. The mean (SD) absolute change from PERSIST baseline was 9.4\% (8.5\%) at Week 96 for STRIVE placebo subjects and 8.1\% (12.5\%) at Week 72 for ENVISION placebo subjects. The safety profile observed in PERSIST was generally consistent with the safety profile observed during ivacaftor treatment in STRIVE and ENVISION.Conclusions: Ivacaftor-related improvements in lung function were sustained with additional ivacaftor treatment. No new clinically important safety concerns were identified.Sponsored by Vertex.}, issn = {0903-1936}, URL = {https://erj.ersjournals.com/content/40/Suppl_56/1842}, eprint = {https://erj.ersjournals.com/content/40/Suppl_56/1842.full.pdf}, journal = {European Respiratory Journal} }