A trial investigating the long-term safety and effectiveness of a combined therapy in people with CF
Details
- Therapeutic approach
- Restore CFTR Function
- Trial status
- Recruitment complete Participating Centres
- Trials Tracker ID
- TT014176
- Last updated
- 16/04/2024
A Phase 3, open-label study investigating the long-term safety and effectiveness of VX-121 combination therapy in people with CF (Vertex VX20-121-104)
Study detailsThe aim of this study is to measure the long-term safety, tolerability, and effectiveness of a combination of medicines (VX-121/TEZ/D-IVA) in people with CF.
The treatment is made up of three medications: VX-121, tezacaftor and deutivacaftor. Deutivacaftor is a slightly modified version of ivacaftor that is potentially more stable in the body. This means deutivacaftor can be taken once a day instead of twice a day.
Patients will receive a single, daily dose of VX-121/TEZ/D-IVA in tablet form. Participants will be asked to visit the CF clinic around 12 times over a period of 96 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 and lung function. Bloods and other samples (urine, sputum) will be collected at some of the visits. Some assessments will be conducted over the telephone.
- Phase
- Phase 3
- Length of participation
- 100 weeks
- Recruitment target
- 73
- CF sponsor
- Vertex
- CF sponsor type
- Commercial
Who can take part?
- Age range
- 12 years and older
- Including people
- Patients must have participated in parent study VX20-121-102 or VX20-121-103
- Patients are homozygous or heterozygous for the F508del mutation, or who have at least 1 other triple combination responsive CFTR mutation and no F508del mutation (TCR/non-F)
- Excluding people
- History of drug intolerance in a parent study
- Women who are pregnant or breastfeeding