VX14-661-110 Cystic Fibrosis

• Research type

  Research Study

• Full title

  A Phase 3, Open-label, 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

• IRAS ID

  186033

• Contact name

  Damian Downey

• Contact email

  damian.downey@belfasttrust.hscni.net

• Sponsor organisation

  Vertex Pharmaceuticals Incorporated

• Eudract number

  2014-004827-29

• Duration of Study in the UK

  3 years, 1 months, days

• Research summary

  Research Summary:
  This is a Phase 3, Open-Label, Rollover study to look at how safe and effective long-term treatment with VX-661 together with Ivacaftor is in paticipants aged 12 years and older with cystic fibrosis (CF) that have a F508del-CFTR 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.

  Summary of Research:
  VX14-661-110 was a Phase 3, open-label, rollover study to evaluate the safety and efficacy of long-termtreatment with VX-661 in combination with ivacaftor in subjects aged 12 years and older with cysticfibrosis, homozygous or heterozygous for the F508del- cystic fibrosis transmembrane conductanceregulator (CFTR) mutation. A total of 1044 (Part A), 464 (Part B), and 204 (Part C) subjects enrolled inthe study. Results for safety: Overall, TEZ/IVA was generally safe and well tolerated in Parts A and B forup to 96 weeks of treatment, as well as in Part C for up to the 192 weeks of treatment. Safety resultswere consistent with the known safety profile of TEZ/IVA, and no new safety concerns were identified.

• REC name

  South West - Central Bristol Research Ethics Committee

• REC reference

  15/SW/0248

• Date of REC Opinion

  23 Oct 2015

• REC opinion

  Further Information Favourable Opinion