VX14-809-109 Cystic Fibrosis
Research type
Research Study
Full title
A Phase 3, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects Aged 6 Through 11 Years With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
IRAS ID
182411
Contact name
Alastair Reid
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Eudract number
2015-000543-16
Duration of Study in the UK
0 years, 9 months, 0 days
Research summary
This is a phase 3 study to evaluate the safety and effects of the combination of Lumacaftor/Ivacaftor in participants aged between 6 through to 11 years old with cystic fibrosis homozygous (having similar pairs of genes for any given pair of inherited characteristics) for the F508del-CFTR mutation.
The study involves 2 phases followed by a safety follow up visit 4 weeks after the end of treatment. The 2 phases consist of a Screening phase (28 days) and Treatment phase for 24 weeks.
Approximately 200 participants will be randomised (1:1) to 1 of 2 treatments arms; either Lumacaftor in combination with Ivacaftor or the matching placebo (a tablet that does not contain any active ingredients).
Patients have an equal chance of being placed in to these arms. Neither the participant nor the study doctor will know which arm the participant is in. The study will take about 32 weeks in total (4 weeks screening, 24 weeks treatment period and safety follow up 4 weeks following last study drug dose). Participants who complete the study drug treatment and the visits in the Treatment Period will be offered the opportunity to enroll in an extension study, if they meet the eligibility criteria.
REC name
HSC REC B
REC reference
15/NI/0113
Date of REC Opinion
19 Jun 2015
REC opinion
Favourable Opinion