Study of Ivacaftor in Cystic Fibrosis Subjects 2 Through 5 Years of Age With a CFTR Gating Mutation
Status:
Completed
Trial end date:
2014-03-01
Target enrollment:
Participant gender:
Summary
The purpose of this study is to evaluate the safety, pharmacokinetics (PK), and
pharmacodynamics (PD), of ivacaftor in children with cystic fibrosis (CF) who are 2 through 5
years of age and have a CF Transmembrane Conductance Regulator (CFTR) gating mutation in at
least 1 allele.
Part A is designed to evaluate the safety and PK of multiple-dose administration of ivacaftor
in participants 2 through 5 years of age and to confirm the doses for Part B. Part B is
designed to evaluate the safety, PK, PD, and efficacy of ivacaftor in participants 2 through
5 years of age.
Phase:
Phase 3
Details
Lead Sponsor:
Vertex Pharmaceuticals Incorporated
Collaborators:
Cystic Fibrosis Foundation Cystic Fibrosis Foundation Therapeutics