Reliable, valid, and sensitive clinical outcome assessments (COAs) are essential to monitor disease progression and measure therapeutic interventions. Traditional COAs capture a single day's performance, which may not reflect a person's daily abilities. Wearable sensors offer continuous monitoring in a home environment, providing real-world evidence of function and more sensitive assessments of gait and balance.

This cross-sectional cohort study was performed at the Hereditary Neuropathy Foundation CMT Clinical Trial Readiness Summit. Participants completed an in-person assessment (CMT-FOM and instrumented gait and balance assessments using LEGSys^TM and BalanSens^TM (BioSensics, Newton MA)). Additionally, physical activity was monitored continuously for 14 days at home using PAMSys^TM pendant sensor.

26 Participants (19-70yrs, 81% female, 58% CMT1A) participated in this study. Participants ranged from mildly to severely affected as measured by the CMT-FOM (53.2 ± 8.0, range: 37 - 73). Compliance with PAMSys for at home monitoring was excellent with mean non-compliance of 22 minutes per day and 16 patients with 100% compliance. Disease severity, measured by the CMT-FOM, was significantly correlated with daily step count in the community (ρ=-0.608, p=0.001), average cadence (ρ=-0.579, p=0.002) and centre of mass ranges measured by BalanSens (ρ>0.4, p<0.05).

The wearable pendant sensor, due to its ease of use, non-invasive nature, and long battery life, resulted in high patient compliance. These wearables provided real-world evidence by continuously capturing long-term data that accurately reflects patients' natural environments and daily activities. Sensor-derived metrics were validated against established COAs. In future trials, the sensitivity of these measures to change should be further explored in longitudinal studies.