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PCT 124 Trials

Could you possibly let me know about the progress on the PCT 124 drug, if any? Regards Tandey Muller"
The title of the study that PTC124 is being tested in is "A Phase 3 Efficacy and Safety Study of PTC124 as an oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis".

PTC124 is a new drug being tested as a potential treatment for CF which is caused by a certain kind of genetic mutation called a nonsense mutation. Having a nonsense mutation means that the body stops part of the way through making the protein "CFTR". This abnormal protein is responsible for CF. PTC124 causes the body to continue making the protein until a fully functioning "CFTR" is produced. This is the first drug to be studied that could potentially treat the cause of CF and not just the effects of having CF.

The trial is a randomised placebo controlled study which means that participants will be randomly allocated to either a group receiving PTC124, or a group receiving a placebo (a dummy medicine with no action). The study consists of 12 months of treatment with a total duration of approximately 14 months. Only around 10% of patients with CF have nonsense mutations and this study plans to recruit 208 participants from a number of countries around the globe.

The study was submitted to the Main Research Ethics Committee in May 2009 and following a successful ethics approval, recruitment is due to commence Autumn/ Winter 2009. Please speak to your local CF Centre if you would like more details on which centres are participating in this trial
Best wishes

Judy Bradley

The answer is edited by: Prof Judy Bradley