Safety, pharmacokinetics, and pharmacodynamics of lumacaftor and ivacaftor combination therapy in children aged 2–5 years with cystic fibrosis homozygous for F508del-CFTR: an open-label phase 3 study

Publication date: Available online 24 January 2019Source: The Lancet Respiratory MedicineAuthor(s): John J McNamara, Susanna A McColley, Gautham Marigowda, Fang Liu, Simon Tian, Caroline A Owen, David Stiles, Chonghua Li, David Waltz, Linda T Wang, Gregory S SawickiSummaryBackgroundThe efficacy, safety, and tolerability of lumacaftor and ivacaftor are established in patients aged 6 years and older with cystic fibrosis, homozygous for the F508del-CFTR mutation. We assessed the safety, pharmacokinetics, pharmacodynamics, and efficacy of lumacaftor and ivacaftor in children aged 2–5 years.MethodsIn this multicentre, phase 3, open-label, two-part study, we enrolled children aged 2–5 years, weighing at least 8 kg at enrolment, with a confirmed diagnosis of cystic fibrosis who were homozygous for the F508del-CFTR mutation. Children received lumacaftor 100 mg and ivacaftor 125 mg (bodyweight
Source: The Lancet Respiratory Medicine - Category: Respiratory Medicine Source Type: research

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