Multi-dose Clinical Trial. Update July 2012

Our multi-dose clinical trial is underway at the Royal Brompton Hospital in London and at the Western General Hospital and Royal Hospital for Sick Children in Edinburgh. We hope to recruit 130 people with CF (aged 12 years and above) into the trial.

The majority of these participants will be from the run-in study or from the Royal Brompton Hospital or from Scottish CF centres.

The trial will be a blinded, placebo-controlled study, meaning that each participant will have a 50:50 chance of receiving gene therapy or 'dummy' treatment, with neither participants nor investigators knowing which.

Participants will receive a dose of gene therapy or 'dummy' treatment via a nebuliser once a month for 12 months.

Participation will be staggered. Some participants have already started their course of nebulisation but some will not start for several months yet. Therefore the trial is not due to conclude until mid 2014.

The gene therapy will consist of the healthy CFTR gene which will be carried into the cells of the airways by a liposome (a fatty substance) called GL67A. This is the same liposome which we used in clinical trials in the 1990s and we therefore have results on its safety and efficacy. The gene however, has been substantially improved and has recently been used in the single dose clinical trial to ensure its safety and to see how long each dose lasts. You can learn more about our gene therapy product GL67A/pGM169 in this section of our website.


Patients on the multi-dose trial will undergo the following tests:

Medical history/clinical examination
Blood and urine samples
Spirometry (Lung function tests)
Sputum analysis
Completion of diary cards and Quality of Life Questionnaires
Lung clearance index (LCI)
Activity Monitoring via armband
Gas Transfer tests
Exercise bike tests
CT scans of the chest

A small sub-group of participants will be invited to take an additional dose by nasal spray. These patients will also undergo nasal potential difference analysis and nasal brushings to look for molecular evidence of gene transfer. We will also look for evidence of this in the lower airways of a subgroup of patients who will be asked to undergo two bronchoscopies.

This will be the first trial of its kind to give multiple repeated doses of gene therapy and look for clinical benefit.


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