A study to investigate the effectiveness and safety of a new Vertex once daily triple therapy in people with CF (VX20-121-103)

A Phase 3 Study Evaluating the Efficacy and Safety of a new once daily triple therapy (VX-121/TEZ/D-IVA) in people with CF who have two copies of F508del, one copy of F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or have at least 1 other Triple Combination Responsive CFTR Mutation and no F508del Mutation

This study aims to look at the safety and effectiveness of a new triple combination CFTR modulator treatment, which is made up of the 3 medications VX-121, tezacaftor (TEZ) and deutivacaftor (D-IVA). Deutivacaftor is a slightly modified version of ivacaftor which makes it potentially more stable in the body. This means deutivacaftor can be taken once per day instead of twice daily. The safety and effectiveness of this new triple combination treatment will be compared against Kaftrio® (which consists of the 3 CFTR modulators elexacaftor, tezacaftor and ivacaftor).



CFTR modulators help the faulty CFTR protein to function properly.



To take part in this study you must be aged 12 years or older, with certain mutations of the CF gene (see details within the Top Inclusion Criteria list). Study participants will be randomly allocated into one of two groups which will determine whether they take the new treatment (VX-121/TEX/D-IVA) or Kaftrio®. Throughout the study, participants will not know which treatment group they are in.



Study participants will be asked to visit the CF clinic around 14 times over a period of 1 year and 2 months, 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. For Week 12, a home health visit is also an option.