VX15-770-123 - Cystic Fibrosis study for 3-5 year olds

• Research type

  Research Study

• Full title

  A Phase 3b, 2-part, Randomized, Double-blind, Placebo-controlled Crossover Study With a Long-term Open-label Period to Investigate Ivacaftor in Subjects With Cystic Fibrosis Aged 3 Through 5 Years Who Have a Specified CFTR Gating Mutation

• IRAS ID

  189383

• Contact name

  Jane Davies

• Contact email

  j.c.davies@imperial.ac.uk

• Sponsor organisation

  Vertex Pharmaceutical (Europe) Limited

• Eudract number

  2015-001267-39

• Duration of Study in the UK

  5 years, 4 months, 18 days

• Research summary

  This is a study to evaluate the efficacy and safety of Ivacaftor in children with cystic fibrosis (CF) aged 3 to 5 years old who have a specified CFTR gating mutation. It is a phase 3b, 2-part, randomised, double-blind, placebo-controlled crossover study with a long-term open-label period.

  Ivacaftor (Kalydeco®) is a first-in-class therapy that potentiates the activity of the CFTR protein. Ivacaftor is currently one of very few approved treatment for patients with CF that targets the underlying cause of the disease.

  Subjects will be randomised 1:1 to receive ivacaftor or placebo in one of two treatment periods:
  During Treatment Period 1, subjects will be dosed with placebo or ivacaftor. During Treatment Period 2, subjects who were dosed with ivacaftor in Treatment Period 1 will be dosed with placebo, and subjects who were dosed with placebo will be dosed with ivacaftor.

  Part 2 will be open-label and subjects will therefore be treated ivacaftor. After completion of Part 1, subjects will immediately begin Part 2; there is no washout period in between Parts 1 and 2.

  The study is designed to obtain long-term efficacy and safety information in this young age group to further understand the impact of ivacaftor on lung and pancreatic function. The study will also examine how long-term treatment with ivacaftor impacts the course of CF disease progression.

  A maximum of approximately 50 subjects will be enrolled and following a screening period, each subject will participate in the study for 148 weeks (± 7 days) (from Day 1 to last day of Follow-up, Week 148 [± 7 days]).

• REC name

  London - Riverside Research Ethics Committee

• REC reference

  15/LO/1729

• Date of REC Opinion

  16 Nov 2015

• REC opinion

  Favourable Opinion