Vertex - VX14-661-108 - Cystic Fibrosis

• Research type

  Research Study

• Full title

  A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Efficacy and Safety of Ivacaftor and 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 Allele With a CFTR Mutation Predicted to Have Residual Function

• IRAS ID

  173824

• Contact name

  Jane Davies

• Contact email

  j.c.davies@imperial.ac.uk

• Sponsor organisation

  Vertex Pharmaceuticals Incorporated

• Eudract number

  2014-004788-18

• Clinicaltrials.gov Identifier

  NCT02392234

• Duration of Study in the UK

  1 years, 7 months, 29 days

• Research summary

  This is a phase 3 study to evaluate the efficacy of VX-661 in combination with ivacaftor and ivacaftor monotherapy through 8 weeks of treatment in patients aged 12 years and older with Cystic Fibrosis (CF), heterozygous for the F508del-CFTR Mutation on the CF transmembrane conductance regulator (CFTR) gene and a second allele with a CFTR mutation predicted to have residual function.

  The following countries are taking part in this global study:
  Canada, France, Germany, Israel, Italy, Netherlands, Switzerland, UK and USA
  
  Approx 300 subjects will be randomly placed into 1 to 6 treatment sequences:

  • Sequence 1: VX-661/ivacaftor in Treatment Period 1 → washout → ivacaftor monotherapy in Treatment Period 2
  • Sequence 2: ivacaftor monotherapy in Treatment Period 1 → washout → VX-661/ivacaftor in Treatment Period 2
  • Sequence 3: VX-661/ivacaftor in Treatment Period 1 → washout → placebo in Treatment Period 2
  • Sequence 4: placebo in Treatment Period 1 → washout → VX-661/ivacaftor in Treatment Period 2
  • Sequence 5: ivacaftor monotherapy in Treatment Period 1 → washout → placebo in Treatment Period 2
  • Sequence 6: placebo in Treatment Period 1 → washout → ivacaftor monotherapy in Treatment Period 2

  There will be two treatment periods, each of these with a duration of 8 weeks.

  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, 8 weeks of treatment followed by a washout period of 8 weeks and a subsequent treatment period of 8 weeks and a follow up period of about 4 weeks).

  Patients 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

  London - Westminster Research Ethics Committee

• REC reference

  15/LO/0699

• Date of REC Opinion

  8 Jun 2015

• REC opinion

  Further Information Favourable Opinion