Feasibility of measuring functional performance of FSHD patients using wearable sensors to quantify physical activity

Clinical Trials
91
Joost Kools, MD, Radboud University Medical Centre (Radboudumc), Nijmegen, NL
Nicol Voermans, MD, Radboud University Medical Centre (Radboudumc), Nijmegen, NL
Karlien Mul, MD, Radboud University
Michelle L Mellion, MD, Fulcrum Therapeutics, Inc.
John Jiang, PhD, Fulcrum Therapeutics, Inc.
Jennifer Shoskes, PharmD, Fulcrum Therapeutics, Inc.
Kelly Marshall, Fulcrum Therapeutics, Inc.
David Jackson, MD, Fulcrum Therapeutics, Inc.
Yuxi Zhao, Fulcrum Therapeutics, Inc.
Anil Tarachandani, PhD, UsinLife LLC, Whitehouse Station, NJ, USA
Joanita Figueredo, PhD, UsinLife LLC, Whitehouse Station, NJ, USA
Damien Eggenspieler, Sysnav SAS, Vernon, France
Baziel van Engelen, MD, PhD, Radboud University Medical Centre (Radboudumc), Nijmegen, NL

Objective: Evaluate feasibility to monitor daily activity and assess functional outcomes using wearable sensor devices in an open label study (OLS) of losmapimod in facioscapulohumeral muscular dystrophy (FSHD).

Background: Evaluation of clinical outcome assessments in FSHD is challenging due to the variable onset, severity and progression.:. Fulcrum is evaluating the safety and efficacy of losmapimod for the treatment of FSHD with selected clinical outcome assessments in the ongoing OLS. Assessment with wearables may be a sensitive measure of upper extremity function, activities of daily living and mobility in FSHD.

Methods: Actimyo wearable activity monitoring devices were provided to participants in the OLS to be placed on 1 wrist, and on 1 ankle. Activity was monitored, during the day, in outpatient setting, intermittently - alternating two weeks on and two weeks off until the end of the study. An 8-week baselining period was collected, with similar wear frequency, prior to initiating treatment. Participants performed prescribed movements of upper and lower extremities twice daily.

Results: Fourteen subjects ages 18 to 65 years with genetically confirmed FSHD1, clinical severity score of 2 to 4 (range 0-5) received 15 mg losmapimod twice daily completed the study over 60 weeks. Compliance for wearing the devices was 99%. All 14 participants were monitored of a total of 2941 days or 36758 hours, for an average of 2626 hours per participant. Multiple parameters calculated from the upper and lower extremities were found to be reliable (ICC > .90). Analysis of correlations with clinical outcome assessments will be reported.

Conclusion: Measurement of functional performance in FSHD patients using wearable sensors is feasible, reliable, and correlates with multiple clinical outcome assessments, activities of daily living and mobility. This assessment could provide critical data about disease progression and treatment efficacy in future clinical trials.