Evaluation of potential mucus clearing treatment for all patients with CF regardless of mutation.
This is a Phase 2, randomized, double-blind, placebo- and tezacaftor/ivacaftor (TEZ/IVA)-controlled, parallel-group, 3-part, multicenter study designed to evaluate the safety and efficacy of VX-659 in triple combination (TC) with TEZ and IVA in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation of the CF transmembrane conductance regulator (CFTR) gene (F/F genotype), or who are heterozygous for the F508del mutation and a minimal function (MF) CFTR mutation not likely to respond to TEZ, IVA, or TEZ/IVA (F/MF genotypes).
A randomised controlled trial and parallel process evaluation to determine whether CFHealthHub, an intervention to help CF patients build better treatment habits, offers any benefit over usual care to adults with CF (1711532)
Cystic Fibrosis (CF) is a genetic condition that causes the lungs and digestive system to be clogged with mmucus. It affects 10000 people in the UK. The lungs of people with CF (PWCF) are prone to infections and require daily medication to stay healthy. Average adherence to medication has been shown to be only 36%. Poor adherence is associated with poor clinical outcomes including distressing unscheduled hospitalisations. Researchers have designed an intervention that will feedback adherence data to PWCF using chipped nebulisers displaying real time treatment use on smartphones and the website (CFHealthhub). Feedback is a powerful support to habit formation and the use the CFHealthHub and a behaviour change manual to support PWCF in building successful treatment habits. The aim of this study is to assess whether use of the CFHealthHub intervention, which includes the toolkit and website, reduces the amount of unscheduled emergency care PWCF require compared to those receiving standard care.
Interstitial lung disease (ILD), which is characterized by inflammation and scarring of the lungs, is the leading cause of death in systemic sclerosis (SSc), and a major cause of morbidity in many other connective tissue diseases (CTDs) a group of conditions that are caused by over activity of the immune system. If connective tissue disease associated interstitial lung disease (CTD-ILD) is severe or progressive, immunosuppressive treatment (treatment used to damp down the immune system), such as intravenous cyclophosphamide, is required to suppress inflammation and minimise progressive lung scarring.
RAMPP is an interventional multi-centre (UK only) randomised controlled trial comparing ambulatory to standard (in-patient treatment with aspiration/chest drain) management of primary pneumothorax. Primary outcome is total hospital stay up to 30 days post-randomisation. The study will also investigate whether digitally measured air leak can predict short term outcome (prolonged air leak and requirement for surgery) and whether radiological evidence (on CT scanning) of emphysema-like change and inflammation can predict long term outcome (recurrence rates at 12 months). Patients will have baseline observations and blood tests and then will be reviewed daily either on the ward (standard management group) or as an outpatient (ambulatory group) with chest x-ray, air leak measurement and assessment of breathlessness and chest discomfort. Patients who do not require treatment, after their initial assessment, can be discharged but return for follow up. All patients will be followed-up at 1 week post treatment completion (including a CT scan), and then at 1, 6 & 12 months post enrolment.