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The Run In Study

Run-in to multi-dose clinical trial

In preparation for the multi-dose trial of gene therapy we designed a study of almost 200 people with cystic fibrosis.

This is called the run-in study.  The purpose of this study was to help design the multi-dose trial and to choose the patients most likely to be able to demonstrate benefit.  In the run-in study we have performed a range of sophisticated tests on a number of occasions over several years.  This included tests of lung function, bacterial infection and inflammation in the lungs.

People in the study performed the following procedures:

  • Measurement of temperature, pulse, blood pressure, oxygen saturation levels and clinical examination of the lungs with a stethoscope

  • Sputum collection and sputum induction (if required)

  • Lung function measurements

  • Blood tests and urine samples

  • Completion of a Quality of Life Questionnaire (CFQ-UK) and diary card

  • Collection of Exhaled Breath Condensate (EBC)

  • Lung Clearance Index (LCI)

  • Shuttle Exercise Test

  • Computed Tomographic (CT) scanning of the chest

  • Mucociliary Clearance (MCC) scan (in Southampton)

  • Bicycle Exercise Tests

  • Completion of a symptom score card

This study helped us to decide which tests will be best at showing any benefits that might result from treatment with gene therapy.  This has also enabled us to determine how to select people with CF for the multi-dose trial in whom we will be able to detect potential treatment benefits.

Patients who were not considered suitable were asked to undergo a final exit visit.  The Consortium and the CF Trust are very grateful to all those who have participated in this important study.

The data which have been generated will prove extremely useful, not only for this study, but for CF clinical trials in general.

Find out about the discovery of

the CFTR Gene,

responsible for Cystic Fibrosis

Find out how

the CFTR Gene

causes Cystic Fibrosis

Find out about

the structure of the

CFTR Protein

Why use Gene Therapy

to treat

Cystic Fibrosis?

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