Study design (if review, criteria of inclusion for studies)
Review, HTA report
List of included studies (3)
TRANSPORT, NCT01807949, VX12-809-104; lumacaftor and ivacaftor vs placebo; phase III. TRAFFIC, NCT01807923, VX12-809-103; lumacaftor and ivacaftor vs placebo; phase III. NCT01931839, VX12-809-105; phase III extension.
patients aged 12 years and older with cystic fibrosis (CF) who are homozygous for the F508del-CFTR mutation.
Lumacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) corrector and ivacafor is a CTFR potentiator.
Primary: Relative change in percent predicted forced expiratory volume in 1 second (FEV1); safety. Secondary: Absolute change in percent predicted FEV1, change in body mass index (BMI), number of pulmonary exacerbations, cystic fibrosis questionnaire (CFQ-R) score, safety.