The purpose of this study is to assess whether inhalation of 7% hypertonic saline (HS) twice a day will improve lung function in comparison with inhalation of 0.9% isotonic saline (IS) in preschool children with cystic fibrosis. This is a multicenter, randomized, double-blind, controlled, parallel group clinical study. The Lung Clearance Index measured by Nitrogen Multiple Breath Washout will be used to assess the efficacy of 7% HS vs. 0.9% IS. This study is open to patients from other Cystic Fibrosis Centres.

The VX15-809-115 Study is a Phase three, two-part study that will be evaluate the pharmacokinetics and safety of lumacaftor and ivacaftor combination therapy in subjects aged two through five years with cystic fibrosis, and are homozygous for F508del CFTR mutation. Part A of the initial study will last for 15 days and will focus on how the body processes the drug. Part B will last for six months and will focus on the safety and effectiveness of the drug. This study is open-label, meaning that all study participants will receive the study drug. This study is open to patients from the Cystic Fibrosis Centres throughout Canada.

The present study is designed to obtain long-term efficacy and safety information in this young age group to further understand the impact of ivacaftor on lung and pancreatic function. The study will also examine how long-term treatment with ivacaftor impacts the course of cystic fibrosis disease progression. The VX15-770-123 study will evaluate the efficacy of ivacaftor in children three to five years of age with respect to lung function as measured by Lung Clearance Index and CT scan. This study is open to patients from other Cystic Fibrosis Centres.

This study assesses the efficacy of the CFTR Modualtor Ataluren in patients with Class I (stop) mutations who are currently not on inhaled antibiotics. The study enrollment is completed and patients are now in a rollover study to assess long term safety and efficacy.

The PIPE is a randomized, double blind, placebo controlled study that aims to determine whether oral corticosteroids (prednisone) can be an adjunctive therapy for pulmonary exacerbation’s in cystic fibrosis patients. We hypothesize that using oral corticosteroids as an adjunctive therapy in cystic fibrosis patients not responding to antibiotic treatment will improve lung function recovery during PExs by suppressing excessive inflammation in the lung.