Clinical Studies

Trials

The importance of measuring CFTR expression and function in patients with CF over a period of time has been highlighted in our own work and the work of others. To this end, we completed 2 studies in 2006/2007.

The Sample Validation study assessed the reproducibility and sensitivity of our markers in human samples. The Tracking study aimed to look at simple ways of following changes in the lungs of patients with an exacerbation that didn't involve tests that were complicated or challenging for the patient. This study helped us understand how the lungs change when patients are unwell, and which markers of lung function are best at following improvements following antibiotic treatment.

We have now started a phase of pre-testing in CF patients before proceeding to gene therapy. The aim of this Run In Study is to define the level of disease activity in individual patients. This will help with selection of the most effective methods of measuring changes in the lung.

We are also in the process of designing gene therapy trials for our leading product. These trials are due to begin mid-2008.

We are very grateful to the CF Trust for funding this work, and to all the patients who have taken part in our studies already.