| Clinical trials identifier | NCT02514473 |
|---|---|
| Study codes | VX14-809-109 |
| Study 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 |
| Sponsor | Vertex |
| Drug (brand name) | Lumacaftor, ivacaftor and placebo |
| Drug administration | Tablet |
| Eligible CF patients | Children 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 timeframe | The 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: http://www.thelancet.com/journals/lanres/article/PIIS2213-2600(17)30215-1/abstract and from clinicaltrials.gov. |