Vertex - VX14-661-109 - Cystic Fibrosis

• Research type

  Research Study

• Full title

  A Phase 3, Randomized, Double-Blind, Ivacaftor-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation and a Second CFTR Allele With a Gating Defect That Is Clinically Demonstrated to be Ivacaftor Responsive

• IRAS ID

  174447

• Contact name

  Tim Lee

• Contact email

  timlee@doctors.org.uk

• Sponsor organisation

  Vertex Pharmaceuticals Incorporated

• Eudract number

  2014-004838-25

• Duration of Study in the UK

  0 years, 8 months, 1 days

• Research summary

  This is a phase 3 study to evaluate the safety and effectiveness of VX-661 in combination with ivacaftor in patients aged 12 years and older with cystic fibrosis, heterozygous for the F508del mutation (i.e. they have one copy of the F508del-CFTR mutation TR and a second CFTR allele (different form of the same gene) with a gating mutation responsive to ivacaftor.

  The study is carried out in 2 periods, including an initial Ivacaftor Period for 4 weeks, followed by the 661/ivacaftor active treatment period for 8 weeks. All subjects will receive ivacaftor twice daily throughout the duration of the trial.

  Approximately 200 subjects will be randomly placed into two groups (arms):

  • Treatment Arm A: 100 mg VX-661 once a day + 150 mg ivacaftor twice a day
  • Treatment Arm B: 150 mg ivacaftor twice a day

  Patients have an equal chance of being placed in to these arms. Neither the patient nor the study doctor will know which arm the patient is in. The study will take about 32 weeks (4 weeks for screening, 4 weeks for Ivacaftor Run-in Period, 8 weeks for Active Comparator Treatment Period and a follow up period of about 4 weeks.

  Patients who remain in the study through the Week 8 Visit of the Active Comparator Treatment Period, regardless of whether they have stopped taking the study drug before the treatment ended, will be offered the opportunity to enrol in an extension study, if they meet the eligibility criteria.

• REC name

  Yorkshire & The Humber - Leeds West Research Ethics Committee

• REC reference

  15/YH/0178

• Date of REC Opinion

  29 May 2015

• REC opinion

  Further Information Favourable Opinion