VX12-770-112 Phase 3 rollover study of ivacaftor in CF patients 6yrs+

• Research type

  Research Study

• Full title

  A Phase 3, Two-Arm, Rollover Study to Evaluate the Safety of Long-Term Ivacaftor Treatment in Subjects 6 Years of Age and Older with Cystic Fibrosis and a Non-G551D CFTR Mutation

• IRAS ID

  108557

• Contact name

  Stuart Elborn

• Sponsor organisation

  Vertex Pharmaceuticals Incorporated

• Eudract number

  2012-000389-39

• ISRCTN Number

  No number provided

• Clinicaltrials.gov Identifier

  No number provided

• Research summary

  Cystic fibrosis (CF) is a disease that causes repeated lung infection and leads to premature mortality. It affects approximately 30,000 individuals in the United States and 36,000 in the European Union. There is currently no cure. CF is caused by mutations (changes) in the CF transmembrane conductance regulator gene (CFTR). Ivacaftor is the first CFTR modulator to show an improvement in CFTR function and clinical benefit in patients with CF. The current study will enroll subjects with CF who participated in a qualifying previous study of ivacaftor (VX11-770-110 [Study 110]). For subjects who had completed their assigned treatment in the previous study, Study VX12-770-112 (Study 112) offers an opportunity to continue ivacaftor treatment or, for subjects who received placebo in their qualifying study, to receive ivacaftor treatment. For subjects who had prematurely discontinued their assigned treatment in the previous study, Study 112 is an opportunity to obtain data to allow evaluation of any major safety issues that arise after stopping prolonged treatment with ivacaftor. Results from this study will provide information of the safety and efficacy of long-term ivacaftor treatment in subjects with CF who are aged 6 years and older. Participants will be enrolled into one of the following two arms of the study: ? Ivacaftor arm: Subjects who completed assigned study drug treatment duration in the previous study will be enrolled. The treatment duration will be approximately 60 weeks. ? Observational arm: Subjects who prematurely discontinued study drug treatment and received at least 4 weeks of treatment in the previous ivacaftor study will be enrolled. Subject participation in the observational arm is planned for approximately 1 year.

• REC name

  London - Brighton & Sussex Research Ethics Committee

• REC reference

  12/LO/1084

• Date of REC Opinion

  25 Oct 2012

• REC opinion

  Further Information Favourable Opinion