Trials Week: An introduction to clinical trials

All this week the Cystic Fibrosis Trust and cf/Aware are joining forces to highlight the importance of clinical trials and why it’s vital for people with cystic fibrosis to get involved where they can. To get the ball rolling, Professor Alan Smythof the University of Nottingham, explains exactly what a clinical trial is and how it works.

A clinical trial is a fair test of a treatment. This treatment can be compared with a dummy treatment (placebo) or an alternative treatment (eg another similar drug). The results tell us if the treatment is effective and whether any harmful effects are seen. Clinical trials have brought about effective treatment for a range of conditions, which used to be killers, such as tuberculosis and leukaemia. More recently, clinical trials have shown that a new drug (ivacaftor) is effective in some people with cystic fibrosis (CF). Clinical trials must have a minimum number of patients. If there are too few patients then we may miss a real difference with the treatment we are testing. Drugs may work differently in children and adults, so we need to do clinical trials in both age groups.

Doctors know that taking part in a trial is a big ask for patients. Extra visits or additional tests may be needed – though we keep these to a minimum. However, we must do these trials if we want to improve the treatment of cystic fibrosis. There are many questions which we still need to answer - from evaluating exciting new treatments like ivacaftor, to testing which antibiotic works best for lung infections.

TORPEDO-CF (http://www.torpedo-cf.org.uk) is an important cystic fibrosis trial which is recruiting at the minute. TORPEDO-CF compares intravenous and oral antibiotics to see which is most effective in getting rid of early lung infection with Pseudomonas. Patients in both arms of the trial also get a nebulised antibiotic. The trial is important because Pseudomonas has a huge effect on patients’ lives, if it becomes chronic, and because intravenous treatment is a much bigger hassle than oral antibiotics. To see which treatment is best, the trial must recruit 280 patients. So far we have almost three quarters of this total. With your help we can finish the trial!

Our friends at cf/Aware have produced a glossary of clinical trial terms, to clear up some of the more complicated terminology.