CFTR potentiator for cystic fibrosis
- Correspondence to Dr Siu P Cho, ST3, East Kent Hospitals University NHS Foundation Trust, Kent and Canterbury Hospital, Department of Chest Medicine, Ethelbert Road, Canterbury CT1 3NG, UK;
This multicentre, double blinded, randomised controlled trial assessed the efficacy of ivacaftor, a cystic fibrosis transmembrane conductance regulator (CFTR) protein potentiator.
The primary outcome of the trial was the change in the forced expiratory volume in 1 s (FEV1). The secondary outcomes were time to the first pulmonary exacerbation and subject-reported respiratory symptoms. The trial …