Vertex - VX14-661-106 - Cystic Fibrosis

• Research type

  Research Study

• Full title

  A Phase 3, Randomized, Double-Blind, Placebo-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, Homozygous for the F508del-CFTR Mutation

• IRAS ID

  172879

• Contact name

  Damian Downey

• Contact email

  Damian.Downey@belfasttrust.hscni.net

• Sponsor organisation

  Vertex Pharmaceuticals Incorporated

• Eudract number

  2014-004837-13

• Clinicaltrials.gov Identifier

  NCT02347657

• Clinicaltrials.gov Identifier

  IND Number (VX-661), 108,105

• Duration of Study in the UK

  0 years, 8 months, 31 days

• Research summary

  This is a phase 3 study to evaluate the efficacy of VX-661 in combination with ivacaftor through 24 weeks of treatment in patients aged 12 years and older with Cystic Fibrosis (CF), homozygous (having identical pairs of genes for any given pair of hereditary characteristics) for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.

  The following countries are taking part in this global study:
  Canada, Denmark, France, Germany, Ireland, Italy, Netherlands, Spain, Switzerland, Sweden, UK and USA

  Approximately 490 subjects will be randomly placed into 1 of 2 treatment groups (arms):

  • VX-661/ivacaftor: 100 mg VX-661 once daily + 150 mg ivacaftor every 12 hours
  • Placebo: Placebo regimen with visually matched tablets

  Patients have an equal chance of being placed into 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, 24 weeks of treatment and a follow up period of about 4 weeks).

  Participants who complete the Week 24 visit, regardless of whether they have prematurely discontinued study drug treatment, will be offered the opportunity to enrol in an extension study, if they meet the eligibility criteria for the extension study.

• REC name

  HSC REC B

• REC reference

  15/NI/0064

• Date of REC Opinion

  12 May 2015

• REC opinion

  Further Information Favourable Opinion