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A Study to Confirm the Long-term Safety and Effectiveness of Kalydeco in Patients With Cystic Fibrosis Who Have an R117H-CFTR Mutation, Including Pediatric Patients

Vertex Pharmaceuticals Incorporated · 2015
ClinicalTrials.gov · Datasets · License: Public Domain · 2015
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cystic-fibrosis
The purpose of this study is to confirm the long-term safety and effectiveness of Kalydeco® (ivacaftor) in US CF patients with the R117H-CFTR mutation \<18 years of age and to describe the long-term safety and effectiveness of Kalydeco in CF patients with the R117H-CFTR mutation overall and in patients ≥18 years. The long-term safety and effectiveness of Kalydeco will be examined in totality through the evaluation of the primary outcome measures.
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