(169) A Phase 4, Fully Decentralized Clinical Trial to Evaluate Physical Activity and Cough Frequency in Patients with Cystic Fibrosis Using Wearable Technology

Background: Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) has been approved for use in people with cystic fibrosis (CF) age ≥ 6 years with ≥ 1 F508del-CFTR mutation or another ELX/TEZ/IVA-responsive mutation. Here we report results from a Phase 4, fully decentralized pilot study designed to evaluate the performance of wearable technology in assessing physical activity and cough frequency in people with CF age ≥ 18 years treated with commercial ELX/TEZ/IVA (NCT04923464).

Methods: Wearable devices were used to measure daily physical activity and cough frequency over a 12-week measurement period. Participants, who had been taking commercial ELX/TEZ/IVA for at least 4 weeks prior to screening, were provided with a wrist-worn actigraphy sensor (worn continuously during measurement period) and an ambulatory cough monitoring system (worn once per week for 24 continuous hours). The primary endpoint was compliance with the actigraphy device (defined as percent time wearing device during measurement period). Secondary endpoints included compliance with the cough measurement device (defined as percent time wearing device during 24-hour measurement period each week, averaged across 12 weeks), number and variability of steps per day, and number and variability of coughs per day.

Results: Fifty-one participants were enrolled (mean [SD] age 34.6 [11.6] years; mean [SD] percent predicted FEV₁ at screening 68.8 [23.0]). Five participants discontinued the study (adverse event [skin reaction from a device adhesive; n=1]; withdrawal of consent [not due to adverse event; n=2]; other reasons [n=2]). Mean compliance with the actigraphy device was 91.3% and the mean compliance with the cough measurement device was 84.8%. During the 12-week measurement period, mean step count ranged from 4400 to 5500 steps/day (SD, 2300 to 3000 steps/day), and geometric mean cough count ranged from 21 to 33 coughs/day (geometric SD, 4.1 to 7.0 coughs/day).

Conclusions: Overall, compliance was high for use of the actigraphy sensor and ambulatory cough monitoring system. The variability observed in step counts and cough counts obtained from these wearable devices was generally consistent with previous reports. Notably, cough counts for these participants stable on ELX/TEZ/IVA were comparable with reported counts from healthy adults. These results show that performing fully decentralized trials in people with CF using wearable technology is feasible and indicate the potential for such trials to provide novel clinical insights on CF disease progression and the efficacy of CFTR modulators.

Acknowledgements: This work was funded by Vertex Pharmaceuticals Incorporated.

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