A study to evaluate the safety and effectiveness of a new triple combination CFTR modulator therapy in children aged 1 to 11 (VX21-121-105)

This study, also known as RIDGELINE 105, assessed the safety and effectiveness of a new triple combination therapy: vanzacaftor/tezacaftor/deuticaftor (VX121/TEZ/D-IVA or ‘vanza triple’).

78 children with CF aged 6 to 11 with at least one CFTR gene variant (including F508del) that’s responsive to triple combination CFTR modulator treatments like Kaftrio took part. This was following previous similar studies on people aged 12 and older, SKYLINE 102 and 103. 

All participants were treated with Kaftrio for 4 weeks and then treated with the new vanza triple for up to 24 weeks. 

In the RIDGELINE 105 study, the safety of the vanza triple in children aged 6 to 11 years old was similar to the safety of the vanza triple in people ages 12 and older. The vanza triple reduced sweat chloride levels further compared to after 4 weeks of taking Kaftrio. 95% of children in the study had sweat chloride levels that were below the level to be diagnosed as having CF and a majority achieved sweat chloride levels below the level to be diagnosed as a carrier.

The results of this study shows that the new vanza triple therapy is at least as good as Kaftrio in improving lung function in people with CF and has better results in reducing sweat chloride levels. Vertex announced in a press release (linked below) that they will be looking to submit an application to regulatory authorities to bring this potential medicine to patients as quickly as possible.