A study to investigate the effectiveness and safety of a new Vertex once daily triple therapy in people with CF (VX20-121-102)

A Phase 3 study evaluating the efficacy and safety of a new once daily triple therapy (VX-121/TEZ/D-IVA) in people aged 12 years and older with cystic fibrosis who are heterozygous for F508del and a minimal function mutation (F/MF)

This study aims to look at the safety and effectiveness of taking 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 to make it potentially more stable in the body, which 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, and have one F508del CFTR mutation plus a minimal function mutation. 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 12 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.