This month, our dedicated supporters joined us in raising awareness of cystic fibrosis for CF Week, and continue to take on fundraising challenges for the Trust.
Ataluren is a novel investigational drug which works by helping the body to ignore the nonsense mutation and make a complete and functional CFTR protein. So far the results from clinical trials in ataluren have shown that the drug appears to be safe and well tolerated. There is currently an ongoing clinical trial (PTC124-GD-021-CF) which assesses the safety and effectiveness of ataluren in 208 patients worldwide with nmCF who are 6 years or older. After 48 weeks of treatment with ataluren or placebo, patients who successfully complete this study will have the chance to go onto this extension study (PTC124-GD-021e-CF)...
Over the next five years, our Translational Innovation Hub Network for lung health and infection in CF will address challenges around finding new lung infection treatment options, detecting and treating flare-ups, and developing new ways to diagnose infections.
The purpose of this study is to find out whether GLPG2222, taken by mouth as tablet once a day, is safe, well-tolerated and effective in improving your signs and symptoms of cystic fibrosis. The study will also evaluate how the body takes in and breaks down the study drug (GLPG2222). The amount of drug in your blood will be measured at specific study visits after you start taking the drug. These measurements will show how quickly the drug is taken up by the body, how quickly it is broken down, when it reaches a steady level in the blood, and...
Cystic fibrosis (CF) is a life-shortening genetic disease that damages many of the body’s organs, including the lungs, pancreas and digestive system. Cystic fibrosis related diabetes (CFRD) is the most common complication of CF, affecting 45-50% of adults. CFRD is different from type 1 and 2 diabetes and needs specialist dietary and clinical management. CFRD has a significant effect on nutrition, lung function and survival. The need to educate people with CFRD is important because, keeping blood glucose controlled results in improvements in nutrition, and reduces the development of long term complications from CFRD, including death. NICE recommends that everyone...
This study focuses on the usage of the PAM-13 which is being used as one of the secondary outcome measures to evaluate the intervention we developed. While we would expect that people with higher patient activation scores or levels would have higher levels of adherence no such pattern could be discerned from an initial analysis of the pilot trial baseline data (WP 3.1, Arden et al., unpublished). This has led to the question of how patients with Cystic Fibrosis in the UK interpret and respond to the PAM-13. Given the limited existing evidence of the effectiveness of the PAM-13 in...
This is a randomized, placebo controlled, double-blinded study. 24 CF subjects will be assigned randomly to two groups, active drug or placebo. Three doses of FDL169 or placebo will be given 3 times a day for 28 days. The aim of this clinical study is to test how well CF subjects tolerate FDL169. In addition, the study will also test how subjects’ bodies absorb and digest FDL169 by measuring study subjects’ blood at specific intervals after taking the tablet orally.
