A Study looking at the long-term use of a CFTR modulator (Ivacaftor) in children with cystic fibrosis who are less than 24 months and have a CFTR gating mutation
Details
- Therapeutic approach
- Restore CFTR Function
- Trial status
- Recruitment complete Participating Centres
- Trials Tracker ID
- TT001851
- Last updated
- 16/10/2019
Full title
A Phase 3, 2-Arm, Open-label Study to Evaluate the Safety and Pharmacodynamics of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation
Study detailsThis is a Phase 3, 2-arm, multicenter study with an open-label ivacaftor arm and an observational arm to evaluate the safety and efficacy of long-term ivacaftor treatment in subjects with cystic fibrosis (CF) who are <24 months of age at treatment initiation and have an approved Ivacaftor-Responsive mutation
- Phase
- Phase 3
- Length of participation
- 104 weeks
- CF sponsor
- Vertex Pharmaceuticals Incorporated
- CF sponsor type
- Commercial
Who can take part?
- Age range
- Aged under 2 years
- Including people
Subjects transitioning from Study 124 Part B must have completed the last study visit of Study 124 Part B.
An ivacaftor- responsive CFTR mutation on at least 1 allele. Subjects will be eligible in countries/regions where ivacaftor is approved for use in subjects 2 years of age and older- Excluding people
Subjects receiving commercially available ivacaftor treatment
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