A study of the combined effects of three CFTR modulator medicines in children with cystic fibrosis aged 6 to 11 (VX18-445-106)
VX-445 is an investigational medicine being studied to see how well it works and how safe it is in children with cystic fibrosis aged 6 to 11 years, when taken in combination with tezacaftor and ivacaftor.
All three medicines are CFTR modulators which means that they help the faulty CFTR protein found in cystic fibrosis to work properly.
Tezacaftor is already approved for use and is what is known as a ‘corrector’. Ivacaftor is also already approved for use and is what is known as a ‘potentiator’. VX-445 is a corrector.
Trial participants will be asked to take VX-445, tezacaftor and ivacaftor in the morning (2 tablets), and ivacaftor in the evening (1 tablet). Everyone in the study will receive all three of the study medications, no placebo is involved.
This study is divided into two parts. Part A will recruit 12 people who will be asked to come into clinic around 6 times over 10 weeks. Part B will recruit 56 people who will be asked to come into clinic around 11 times over 32 weeks. During visits, the effect of the treatment on your CF will be measured with sample collection, lung function, questionnaires, and other medical checks.