|Clinical trials identifier||NCT01931839|
|Study codes||VX12-809-105, PROGRESES|
|Study title||A 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®)|
|Eligible CF patients||Aged at least 12 years old, homozygous for the F508del CFTR mutation, participated in feeder study|
|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 timeframe||This 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: http://www.thelancet.com/journals/lanres/article/PIIS2213-2600(16)30427-1/abstract|