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VX Combo trial 770/809


how long will the combo trial phase III last?
When will the results be presented?

Thank you,
Best regards,

Dear Questioner

Thank you for your question.

VX-770 and VX-809 are two compounds that are being studied in combination as a therapy for patients with the CFTR mutation F508del. VX-770, also known as ivacaftor, is a CFTR potentiator. VX-809 (lumacaftor) is a CFTR corrector. The purpose of this study is to evaluate of the safety, efficacy, pharmacokinetics (what the body does to the drug) and pharmacodynamic (effect of the drug on the body) effects of VX-809 alone and when administered in combination with VX-770. This study started in October 2010 with the aim of recruiting 240 adult patients with CF and at least one copy of F508del. It is being conducted in centres across USA, Germany, Belgium, Australia and New Zealand. Whilst the study is still running it is no longer recruiting new patients. It is anticipated that by April 2013 all data will have been collected and the final analysis can begin. This can take some time and the earliest we can hope to hear the full results is by summer 2013.

Some early results were presented in Orlando in at the North American CF Conference (Boyle et al 2012). This early data indicate that in CF patients homozygous for F508del, combination therapy with lumacaftor and ivacaftor produced improvements in lung function. They also indicate that it was safe to administer lumacaftor alone or with ivacaftor for 28 days in these patients.

More information is available on:

Dr Lisa Kent
Prof Stuart Elborn

Boyle, M.P.; Bell, S.; Konstan, M.; McColley, S.A.; Kang, L.; Patel, N. The Investigational CFTR Corrector, VX-809 (Lumacaftor) Co-administered with the Oral Potentiator Ivacaftor Improved CFTR and lung function in F508DEL Homozygous Patients: Phase II study results. Pediatr Pulmonol 2012; 47 suppl 35
The answer is edited by: PhD Lisa Kent