A study to investigate the safety of Kaftrio® in children with CF ages 12 months and older
Details
- Therapeutic approach
- Restore CFTR Function
- Trial status
- Recruitment complete Participating Centres
- Trials Tracker ID
- TT014545
- Last updated
- 21/03/2025
Full title
A phase 3, open-label study evaluating the long-term safety and efficacy of Elexacaftor/Tezacaftor/Ivacaftor in children with cystic fibrosis 12 months of age and older (VX22-445-123)
Study detailsThis study is a continuation of parent study (VX22-445-122) looking at the long term safety, tolerability, efficacy, and pharmacodynamics of Kaftrio in children with cystic fibrosis ages 12 months and older.
This treatment is already approved for use in the UK for certain groups of people with CF, but has not yet been approved for children less than 24 months.
Participants from the parent study who chose to take part in this extension will take Kaftrio in the morning and ivacaftor (IVA) in the evening.
Note: Recruitment to this study is by invitation only.
- Phase
- Phase 3
- Length of participation
- Up to 100 weeks: 96 weeks of treatment and 4 weeks of follow-up
- Recruitment target
- 12
- CF sponsor
- Vertex
- CF sponsor type
- Commercial
Who can take part?
- Age range
- 12 months and older
- Including people
- Participants must have participated in the parent study: VX22-445-122
- Completed study drug treatment in parent study OR had study drug interruptions during the parent study (but did not permanently stop treatment) AND completed all parent study follow-up
- Excluding people
- History of drug intolerance in the parent study that would pose an additional risk to the participant
- Current participation in an investigational drug trial other than parent study