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Clinical trials identifierNCT02514473   
Study codesVX14-809-109
Study titleA 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
Drug (brand name)Lumacaftor, ivacaftor and placebo
Drug administrationTablet
Eligible CF patientsChildren with CF aged 6-11 years with 2 copies of the F508del CFTR mutation
Controlled study?Yes
Randomised study?Yes
Blinded study?Yes (blind - neither medical staff, outcomes assessors nor participants knew which treatment participants were taking)
Extension study?No
Study timeframeThe study was conducted between July 2015 and September 2016

Study:This Phase 2 study was conducted in 54 sites across 9 countries.
Aim:The aim was to assess the safety and efficacy of lumacaftor in combination with ivacaftor in children with CF aged 6-11 years with 2 copies of the F508del CFTR mutation 
Participants:206 children were enrolled and 2014 were treated (101 with placebo and 103 with lumacaftor/ivacaftor). There were 49 participants from 13 ECFS-CTN sites in France, Denmark, Germany, Sweden, Belgium and the UK (Bordeaux, Copenhagen, Lyon, Debré, Necker, Berlin, Koln, Munich, Stockholm, Belfast, Leeds, London, Brussels UZB).
Treatment:Children were randomised to receive either:
•    Lumacaftor 200 mg and ivacaftor 250 mg every 12 hours for 24 weeks
•    Placebo every 12 hours for 24 weeks
Safety:    Serious adverse events were experienced by 13% of children taking lumacaftor/ivacaftor and 11% taking placebo. In total 3% of children in the lumacaftor/ivacaftor group stopped treatment due to an adverse event versus 2% in the placebo group.
Respiratory symptoms and increased liver enzymes were common adverse events. 
The safety profile of lumacaftor/ivacaftor was in line with previous studies with this combination.
Efficacy:Children treated with lumacaftor/ivacaftor had significant improvements in lung function (measured by lung clearance index; LCI) compared to children treated with placebo (least squares mean difference was −1·09 units; p<0·0001). This was the primary endpoint. Sweat chloride also improved significantly in the lumacaftor/ivacaftor group.
Data source:The summary of data provided here is from a peer-reviewed publication, available here:
and from