A study to investigate the safety of Kaftrio® in 2 to 5 year olds with CF (VX20-445-111)
Details
- Therapeutic approach
- Restore CFTR Function
- Trial status
- Closed with results Participating Centres
- Trials Tracker ID
- TT002106
- Last updated
- 22/12/2021
A Phase 3 Study Evaluating the Safety, Tolerability, and Pharmacokinetics of Kaftrio® in children with cystic fibrosis 2 to 5 years old
Study detailsThis study is to learn more about the safety of taking Kaftrio® (elexacaftor in combination with tezacaftor and ivacaftor) and how well it works in 2- to 5-year-olds with CF. All three of the medicines which make up Kaftrio® are called CFTR modulators. CFTR modulators help the faulty CFTR protein to function properly.
Everyone in the study will receive the study medications. Study participants will be asked to visit the CF clinic around 8 times over 6 months (24 weeks), with an additional follow-up visit approximately 4 weeks after finishing the study medications. During these visits, the research team will look at how the study medications are affecting overall health as well as specific assessments to check lung, pancreas and kidney functions. Bloods and other samples will be collected at some of the visits.
- Phase
- Phase 3
- Length of participation
- 7 months (28 weeks)
- Recruitment target
- 6
- CF sponsor
- Vertex Pharmaceuticals Incorporated
- CF sponsor type
- Commercial
Who can take part?
- Age range
- 2 to 5 years
- Including people
2 to 5 years old
Homozygous for the F508del mutation or heterozygous for F508del and a minimal function (MF) mutation (F/F or F/MF genotypes)
- 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