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Clinical trials identifierNCT01931839
Study codesVX12-809-105, PROGRESES
Study titleA Phase 3, Open-label, Rollover Study to Evaluate the Safety of Long-Term Treatment with Lumacaftor in Combination with Ivacaftor in Subjects Aged 12 Years and Older with Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
Drug (brand name)Lumacaftor in combination with ivacaftor (Orkambi®)
Drug administration
Eligible CF patientsAged at least 12 years old, homozygous for the F508del CFTR mutation, participated in feeder study
Controlled study?Yes
Randomised study?Yes
Blinded study?Yes (double blind - neither medical staff, nor participants knew which treatment participants were taking)
Extension study?Yes. This extension study includes patients who  participated in the phase 3 studies TRAFFIC and TRANSPORT.
Study timeframeThis study ran between October 2013 and April 2016


What does this mean for me?For people with 2 copies of the F508del CFTR mutation, Orkambi® shows a good safety profile over the two years of study. Clinical benefits were maintained over this two year period, in terms of a reduced rate of pulmonary exacerbations and reduction in decline in lung function compared to control data from the US CF Registry.
Study:This Phase 3 trial was conducted in 191 sites in 15 countries.
Aim:To assess the long term safety and efficacy of lumacaftor in combination with ivacaftor.
Participants:1030 participants were enrolled into the extension study from the feeder studies TRAFFIC and TRANSPORT. There were 233 participants from 29 ECFS-CTN sites in Belgium, Czech Republic, Denmark, France, Germany, Italy, Netherlands, Spain, Sweden and the UK.
Treatment:Participants who had received lumacaftor/ivacaftor in the feeder studies TRANSPORT and TRAFFIC remained on the same dose in the extension study. Participants who had received placebo were randomly assigned to receive either:
-    lumacaftor (400mg every 12 hours) and ivacaftor (250mg every 12 hours) 
-    or lumacaftor (600 mg once daily) and ivacaftor (250mg every 12 hours).
Participants were treated for up to 96 weeks.
Results are available only for patients who took the 400mg lumacaftor/250mg ivacaftor dose since this is the commercially available product.
Safety:The primary endpoint was the long-term safety of lumacaftor/ivacaftor. Safety results were similar to previous studies with no new safety concerns observed.
The most common adverse events were infective pulmonary exacerbations, cough, increased sputum and haemoptysis.
Efficacy:Long term treatment with lumacaftor/ivacaftor appeared to protect from lung function decline. Patients in this study had 42% slower rate of lung function decline (ppFEV1) compared with a similar group of control patients from the US patient registry.
Data source:The summary of data provided here is from peer reviewed publication, available here: