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Results from Vertex clinical trial for patient with delF508 mutation

Question
Hello

I’m a CF patient and I’m carrying 2 different mutations: delF508 and 1717-1GA of exon 11.
I would like to know if you have the results of the Vertex‘s clinical trials. Are they encouraging? If not, when will they be available?

Best regards
Answer
Hello

A phase 3 clinical trial has been performed for patients carrying 2 copies of the delF508 mutation (homozygous patients). The goal of this trial is to evaluate the combination of Ivacaftor (VX770) and Lumacaftor (VX809). Inclusions are completed and the results are being analyzed and should be communicated by the end of the year.

A phase 2 clinical trial, whose inclusions have just ended, aims to evaluate the effectiveness of VX770 associated to VX809 for patients carrying one copy of the deltaF508 mutation and a second well-defined mutation.

If the results (not published to date) are promising, the next step is the implementation of a phase 3 to confirm results on a larger number of patients.

We have no information on the publication of preliminary results for these two studies.

Best regards,
Anna Ronayette
14.07.2014