TOPLINE:
A 24-week phase 3 trial has found that once-daily vanzacaftor-tezacaftor-deutivacaftor is generally safe and well tolerated in children with cystic fibrosis aged 6-11 years. The treatment maintains normal baseline lung function while improving cystic fibrosis transmembrane conductance regulator (CFTR) function.
METHODOLOGY:
- Researchers conducted a phase 3 trial across 33 clinical sites in eight countries to evaluate the safety and efficacy of once-daily vanzacaftor-tezacaftor-deutivacaftor in children with cystic fibrosis aged 6-11 years.
- A total of 78 children (median age, 9.3 years; 44% girls; 91% White) with a confirmed diagnosis of cystic fibrosis were enrolled. All participants had at least one elexacaftor-tezacaftor-ivacaftor─responsive CFTR variant and forced expiratory volume in 1 second (FEV1 %)…