The UK Cystic Fibrosis Gene Therapy Consortium: The Run In Study

The Run In Study

Run-in to multi-dose clinical trial

labanalysis

A run-in sample being analysed in our laboratory.

Part of the planning for the major multi-dose clinical trial of gene therapy was the recruitment and performing of tests on up to 200 people with Cystic Fibrosis.

This Run-in study was to assess a new range of sophisticated and sensitive tests, which could be used to measure the effect of gene therapy, and to decide which patients would be best suited to go through to the multi-dose clinical trial.

People on the Run-in study underwent various tests to assess lung function, bacterial infection, inflammation, and other tests of clinical severity.

piko_spirometry

A lung function test performed by patients at home on a hand held PiKo machine.

The procedures patients underwent either at home prior to their visit or during their study visits were:

- Chest physiotherapy
- Sputum collection and sputum induction (if required)
- Lung function measurement
- Blood tests and urine sample
- Completion of a Quality of life questionnaire and diary card
- Collection of Exhaled Breath Condensate (EBC)
- Lung Clearance Index (LCI)
- Shuttle exercise test
- Chest Computed Topographic (CT) scan
- Mucociliary Clearance (MCC) scan
- Bicycle tests
- Completion of a symptom score card and undergoing a basic clinical examination

Each patient had to travel to Southampton for their MCC scan to test how well their lungs clear mucus. The Consortium and the CF Trust are very grateful to those who have undergone this long and demanding study.

Out of the 192 patients recruited, 153 have completed the four visits for the study. Based on the data collected, the Consortium are now in a position to select the around 100 patients who will go forward onto the multi-dose clinical trial. Each patient on the Run-in is being contacted by the Consortium to discuss the selection criteria and to be thanked for all their help in the study so far. The choice is made on two main criteria: the ability to deliver the gene to the lungs, and the ability to measure any improvement following gene therapy.

Based on the criteria mentioned above, the patients on the Run-in study have been split into three patient groups. For information on these groups and how they relate to the future multi-dose clinical trial, please visit the patient info page of this website.