EPS6.05 A phase 3b study of the effects of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) on cough and physical activity in people with cystic fibrosis (CF)

Objectives: We assessed the impact of ELX/TEZ/IVA treatment on cough frequency and physical activity using wearable devices in people with CF. Methods: VX20-445-126 (NCT04969224) was a Phase 3b, open-label, hybrid, decentralized study CFTR modulator-naïve CF aged ≥12 years heterozygous for F508del minimal function mutation. A wrist-worn actigraphy sensor (worn continuously during period) an ambulatory cough-monitoring device once/week 24 continuous hours) were used monitoring. Primary endpoint percent reduction from baseline frequency/day to average Week 8 through 12. Secondary absolute change total step count/day Results: Eighty-one participants enrolled received ≥1 dose (mean [SD] age 25.7 [9.6] years; ppFEV1 67.8 [14.3]). Two discontinued due adverse events (AEs). Mean reduced by 91.7% (95% CI: 89.2, 93.6), 241.3 coughs/day at 20.3 Cough 80.6% 2. count increased 638 steps/day 298, 977), 5,279 (SD: 2,132) 5,907 1,909) Time above sedentary, time moderate vigorous activity, improved. Most AEs consistent common manifestations Conclusion: In receiving ELX/TEZ/IVA, mean decreased rapidly coughs/day, which is similar healthy without Activity level, as measured steps/day, clinically important manner compared baseline. These results show has meaningful levels