the type of treatment or therapy being studied. A therapy could range from a medication addressing a particular characteristic of CF to a device or activity e.g. exercise
Restore CFTR Function
the different stages involved in the development of a new medication. Phase I focuses on initial safety in people. Phase 2 evaluates safety, correct dose and early signs of whether the medication works. Phase 3 is the stage before medication licensing and looks at safety and medication effectiveness. Phase 4 evaluates longer term use of a medication after it has been licensed for use
A Phase 3, Openlabel, Rollover Study to Evaluate the Safety and Efficacy of Long term Treatment with VX 661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis , Homozygous or Heterozygous for the F508del CFTR Mutation
This is a Phase 3, OpenLabel, Rollover study to look at how safe and effective longterm
treatment with VX-661 together with Ivacaftor is in paticipants aged 12 years and older with cystic fibrosis (CF) that have a F508de-lCFTR mutation.
The study involves treating all participants who meet all the rules of the study from one of the parent studies (VX-661-106,108,109 or 111) with a combination of VX-661 and Ivacaftor for approximately 96 weeks with a follow-up 28 days after the last dose. Approximately 1375 participants are potentially eligible to be enrolled.
There is also an observational cohort (group) that will enable participants under 18 years of age that received at least 4 weeks of the study drug in the parent study who meet the other rules of the study to enroll. These participants will not receive any study drug but they will have regularly scheduled telephone calls to assess post treatment safety of the combination of VX661/Ivacaftor.
Trial Reference Number
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Length of participation
the length of time a participant will take part in a trial, from the first to the last appointment
the name of the treatment or therapy being researched
Ivacaftor and VX-661
the number of participants who need to be recruited for the trial in the UK
Last edited date
14 September 2018
Vertex Pharmaceuticals Incorporated
CF sponsor type