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Drug & research development » Clinical trials » NCT01897233

Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects 6 to 11 Years of Age With F508del-CFTR Mutation

Official title: A Phase 1, Open-Label Study to Evaluate the Pharmacokinetics and Safety of Lumacaftor in Combination With Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

Clinical Trials gov number: NCT01897233

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Purpose: To evaluate the pharmacokinetics and safety of lumacaftor in combination with ivacaftor in CF patients aged 6–11 years who have the F508del-mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Phase: 1

Type: Interventional, open-label, single group assignment

Study sponsor: Vertex Pharmaceuticals Incorporated

  • Confirmed diagnosis of CF with 2 CF-causing mutations and chronic sinopulmonary disease or gastrointestinal/nutritional abnormalities
  • Weigh >15kg without shoes at screening visit
  • Homozygous for the F508del-CFTR mutation
  • Forced expiratory volume in 1 second (FEV1) of 70% to 105% (inclusive) of predicted normal for age, gender, and height (Knudson standards) at screening
  • Stable CF disease and who are willing to remain on stable CF medication regimen
  • Able to swallow tablets

Intervention
Ivacaftor, Lumacaftor

Geographical Location
United States

Number of Participants
≤50 (6-11 years)

Primary Endpoint

Lumacaftor and Ivacaftor PK parameters, including Cmax and AUC up to 14 days

Secondary Endpoint

Up to 14 days

  • Lumacaftor and ivacaftor metabolite PK parameters including Cmax and AUC

Up to 24 days

  • Safety and tolerability of lumacaftor in combination with ivacaftor as determined by clinical laboratory values, standard 12-lead electrocardiogram, vital signs, pulse oximetry, spirometry and adverse events

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  • Experimental: 200mg of lumacaftor every 12 hours in combination with 250mg of ivacaftor every 12 hours for 14 days
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