This is a Phase 2, randomized, double-blind, placebo- and active-controlled, parallel group, multicenter study to evaluate the safety, tolerability, and efficacy of VX-440 in dual and triple combination with tezacaftor (TEZ; VX-661) and ivacaftor (IVA; VX-770) in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation of the CF transmembrane conductance regulator (CFTR) gene (F508del/F508del), or who are heterozygous for the F508del mutation and a minimal function (MF) CFTR mutation not likely to respond to TEZ and/or IVA therapy
This study will assess two emerging lung function tests. The main test uses lung MRI after inhaling a hyperpolarized gas (HP MRI), which images gas distribution within the lung. This test has been demonstrated in CF patients to show lung disease before other conventional test. So far this technique has been limited to small cohorts and therefore needs to be measured in larger cohorts. The second test is a breathing test entitled multiple breath washout (MBW). This test is similar to HP MRI and is also able to detect lung disease prior to conventional tests. It also measures gas distribution...
The study will evaluate a new hearing test - the High Frequency Digit Triplet (HFDT) test. This test mimics how individuals hear in real life situations (such as a noisy room), requires only a computer and does not need clinically trained personnel. We will evaluate the new test in stable patients and in those about to receive intravenous antibiotics for a chest infection. We will also see if it is feasible to do the test in primary school children. If the HFDT test is accurate and practical we will apply for a new research grant which would allow us to...
This study is a two arm, multicentre parallel group, randomised controlled trial comparing ten days of intravenous therapy to three months of oral therapy in Cystic Fibrosis patients whose lungs have become infected with a bacterium called Pseudomonas(P. aeruginosa). 280 patients (children and adults)in total will be randomised, 140 in each arm of the trial. Background and study aims: Cystic fibrosis (CF) is a genetic condition in which the lungs and digestive system become clogged with thick sticky mucus. CF patients are at risk of developing infection in their lungs which can cause health problems. A common cause of infection...
Ethan, 9, who has cystic fibrosis (CF), recently completed his 100th parkrun. Ahead of the big day, we caught up with Ethan and his mum, Fiona, to chat all things running, Ethan’s hopes for the future, and his advice for other young people with CF.
This week is World Antimicrobial Resistance Awareness Week and we’re raising awareness of why preventing and tackling antimicrobial resistance is so important – and how Cystic Fibrosis Trust is funding research to improve lung health for everyone with cystic fibrosis (CF).
For many people, Christmas is the most wonderful time of the year. But for people with CF, the festive season can be the most worrying. Ruth is mum to Lissy, 19, who has cystic fibrosis. She shares their story – and how research could make a difference for everyone with cystic fibrosis.
Care is changing rapidly in CF. In this blog, Jana Witt, Clinical Quality Improvement Manager at Cystic Fibrosis Trust, reflects on insights from our first ever CF workforce survey of more than 150 CF professionals.
