Study 1 was a 48-week study in which 161 people age 12 and older with a G551D mutation took KALYDECO (150-mg tablets) or placebo with fat-containing food every 12 hours, along with their other cystic fibrosis (CF) treatments. Results were measured at different time points, including 24 and 48 weeks. All people took all their other CF treatments except for hypertonic saline through the entire length of the study (48 weeks). The study evaluated lung function (FEV1). Other results studied were CF respiratory symptoms, pulmonary exacerbations, weight, and sweat chloride. The study also evaluated the safety of KALYDECO.
Mutation eligible to enroll and included in this study was:
Other study results
Study 7 was an 8-week study in which people age 12 and older with 1 copy of the F508del mutation and a second mutation predicted to respond to KALYDECO took either KALYDECO (150-mg tablets) or placebo with fat-containing food every 12 hours. All people took all their other CF treatments except for hypertonic saline. The study evaluated lung function (FEV1). CF respiratory symptoms and safety were also studied.
In this study, 156 people took KALYDECO (150-mg tablets) and 161 people took placebo.
Mutations enrolled in this study were:
2789+5G→A, 3272-26A→G, 3849+10kBc→T, 711+3A→G, A455E, D1152H, D579G, E831X, L206W, P67L, R1070W, R117C, R347H, R352Q, S945L, S977F.