
A phase 3 study evaluated the safety, pharmacokinetics, pharmacodynamics, and efficacy of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) in children aged 2-5 years with cystic fibrosis (CF). The study consisted of two parts: part A (15-day treatment period) and part B (24-week treatment period). Children received different doses based on their weight. The primary endpoints were safety and tolerability, and secondary endpoints included changes in sweat chloride concentration and lung clearance index2.5 (LCI2.5). The study confirmed the appropriate dosing regimen and showed that ELX/TEZ/IVA was generally safe and well tolerated in younger children, resulting in significant reductions in sweat chloride concentration and LCI2.5.
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