A study to investigate the safety of Kaftrio® in children with CF aged 12 to less than 24 months (VX22-445-122)
- Therapeutic approach
- Restore CFTR Function
- Trial status
- In setup Participating Centres
- Trials Tracker ID
- Last updated
A Phase 3, open-label study evaluating the pharmacokinetics, safety, and tolerability of Elexacaftor/Tezacaftor/Ivacaftor in children with cystic fibrosis aged 12 to less than 24 monthsStudy details
This study is aiming to learn more about the safety and tolerability of the combination modulator treatment made up of Elexacaftor (ELX), Tezacaftor (TEZ) and Ivacaftor (IVA) (also known by its brand name Kaftrio) in children aged 12 to less than 24 months.
This treatment is already approved for use in the UK for certain groups of people with CF, but has not yet been approved for this age group.
There will be two parts to the study: Part A and Part B.
Children taking part in Part A will receive ELX/TEZ/IVA in the morning and IVA in the evening. Participation will last for around 10 weeks.
Children taking part in Part B will receive ELX/TEZ/IVA in the morning and IVA in the evening, with the dose(s) to be based on the outcome of Part A. Participation will last for around 32 weeks.
- Phase 3
- Length of participation
- 10 to 32 weeks
- Recruitment target
- CF sponsor
- CF sponsor type
Who can take part?
- Age range
- Aged 12 to less than 24 months
- Including people
Aged 12 to less than 24 months
Two copies of the F508del mutation or one copy of F508del and a minimal function (MF) mutation (F/F or F/MF genotypes)
At least one F508del mutation in the CFTR gene or another ELX/TEZ/IVA-responsive CFTR mutation
- Excluding people
Clinically significant cirrhosis with or without portal hypertension
Lung infection with organisms associated with a more rapid decline in pulmonary status
Solid organ or hematological transplantation
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