Objective: This study aimed to identify balance and gait measures sensitive to disease progression in a multicenter clinical trial of Friedreich Ataxia (FRDA).

Background: The utility of digital balance measures as progression biomarkers in clinical trials remains uncertain. While digital gait and balance measures have shown sensitivity in distinguishing individuals with FRDA from healthy controls in cross-sectional studies, their ability to detect longitudinal changes over short time frames (e.g., one year) is not well established.

Method: Eleven individuals with genetically confirmed FRDA, who were not on any disease-modifying drugs (age: 16 [12,19] years[VL1] , disease duration: 4.5 [7,9] years, modified Friedreich Ataxia Rating Scale (mFARS) total score: 37 [30,38], able to sit and stand unassisted for 30 seconds, and walk 10 meters unassisted), participated in the study. Each participant wore six inertial sensors (one on each foot and wrist, as well as on the sternum and lower back) during laboratory assessments. They completed a 30-second standing balance task (natural stance, eyes open), and a subset of participants (n=7) completed 2-min walk test along with assessments of mFARS total and the Friedreich’s Ataxia Rating Scale – Activities of Daily Living (FARS-ADL). These measures were evaluated at baseline and at a one-year follow-up.

Results: Several digital balance and gait measures demonstrated significant changes after 12 months, with greater effect sizes than the clinical measures. For instance, sway range increased, showing a larger effect size compared to the mFARS total score (0.91 vs. 0.33). Similarly, foot elevation at mid-swing during gait increased showing a larger effect size compared to the mFARS total score (0.98 vs. 0.01). Additionally, the patient-reported FARS-ADL scale showed a significantly increased change, with an effect size of 0.85-0.89. Foot elevation at mid-swing was significantly correlated with FARS-ADL (r=0.87, p=0.012) and mFARS total score (r=0.82, p=0.045).

Conclusion: Wearable sensor-based balance and gait measures successfully captured FRDA progression within one year, detecting changes that mFARS could not. These digital biomarkers show promise as outcome measures for future multicenter interventional trials.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/digital-balance-and-gait-measures-show-promise-to-track-progression-of-friedreichs-ataxia/