A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have a CFTR Gating Mutation (VX15-770-124)

Details

Therapeutic category
Restore CFTR Function
Trial status
Suspended Participating Centres
Phase
Phase III

Full title

A Phase 3, 2 Part, Open-Label Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who are Less than 24 Months of Age at Treatment Initiation and Have a CFTR Gating Mutation

The purpose of this study is to evaluate the safety of ivacaftor treatment, and PK of ivacaftor and metabolites in subjects with cystic fibrosis (CF) who are <24 months of age at treatment initiation and have a CF transmembrane conductance regulator (CFTR) gene gating mutation
Trial Reference Number
71722
Trial type
Medication
Intervention
CFTR Modulator (Ivacaftor)
Recruitment target
35
Last edited date
22/02/2019
CF sponsor
Vertex Pharmaceuticals Incorporated
CF sponsor type
Commercial

Who can take part?

Age
0 - 24 months
Top inclusion criteria
  • Age 0 - 24 months
  • Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D
Top exclusion criteria
  • History of solid organ or hematological transplantation
  • History of abnormal liver function or abnormal liver function at screening

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