The Run-in Study

Run In

Run-in to multi-dose clinical trial
Part of the planning for the major multi-dose clinical trial of gene therapy was the recruitment and measuring of 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 to the clinical trial had to have various measurements to assess lung function, bacterial infection, inflammation, and other tests of clinical severity including quality of life questionnaires. Each patient also had to travel to Southampton for a special 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.

Progress to April 2010
Out of the 192 patients recruited, 153 completed the four visits for the study. Based on the data collected, the Consortium are now in a position select the 100 patients for 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. Around 100 of the people with CF will be chosen to go through to the trial. 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.

The people with CF on the Run-in have been split into the following groups:

Group 1 Patients asked to take part in the multi-dose trial
These people have healthy enough lungs to allow efficient deliver of the gene, but also their lung disease will allow measurement of improvement after gene delivery.

Group 2 Patients not suitable to take part in the multi-dose trial
These people are split into two groups: those who are extremely well and have very healthy lungs, and those who have quite advanced lung disease. The first group are excluded because although it would be very easy to deliver the gene, there would be very little to measure as the lungs are already healthy. Those with quite advanced lung disease are excluded as delivery of the gene would be more difficult and therefore any improvement may be harder to measure. Also, the Consortium is concerned that the treatment may be less well tolerated in this group.

Group 3 Patients for whom a decision has not yet been made
These people are split into two groups: those who are quite well, and those who have had an average lung function of lower than 50% during the Run-in. The Run-in data for those who are quite well are still being analysed to see whether inclusion is possible. For those with reduced lung function, the Consortium are concerned that the gene therapy would be less well tolerated, so further work is being carried out prevent the short-lived reduction in lung function seen in the Pilot Study. This work will determine whether these patients will be suitable for the Multi-dose trial.

The Consortium will be discussing these issues with the people with CF who have taken part in the Run-in and the families will be able to discuss this and the results of the tests taken during the Run-in. All patients, whether included in the Multi-dose trial or not will be offered the chance for a final visit. For those selected to go forward into the Multi-dose trial, these visits will continue every six months until the start of the trial estimated in the second half of 2011.


Introductory Videos
Medical Futures Innovation Award 2011
Twitter Feed
About Us
Contact Us
Lab Events
Environemental Policy
About this Site

Google Site Search

Site Feedback Form

All Site Images



How the Consortium works/FAQs

Consortium Website

Centre for Molecular Medicine, Edinburgh
The Roslin Institute
Dep of Gene Therapy, Imperial



The Run-in Study

Single Dose Clinical Trial

Multi Dose Clinical Trial


Our Research

Non-viral Vector Development

Aerosol Mediated Gene Delivery

Viral Vector Development

Taqman Core Facility

Cystic Fibrosis

History of CF

Discovery of the CFTR Gene

CFTR Protein Structure

CFTR Function

CF Links


Gene Therapy

Introduction to Gene Therapy

Other CF Gene Therapy Groups

Why use Gene Therapy for CF

Target Cells for CF Gene Therapy

Barriers for CF Gene Therapy

Clinical Trials

Gene Therapy Successes

Gene Therapy Links




Papers in Journals

Conference Posters & Presentations

Book Chapters

D.Phil Theses



Gene Therapy Seminars


Directions & Venue