Objective: Our goal was to qualify digital and wearable devices to assess the Timed Up and Go (TUG) test and gait related parameters in healthy volunteers to enable a decentralized natural history study in G2019S LRRK2 PD – a rare disease that can benefit from sensitive endpoints due to small sample size trials. Digital phenotyping using capacity and performance measures done in a subject’s home will provide higher density longitudinal data and sensitive measures of PD progression to support treatment trials.

Background: TUG is a capacity assessment that measures the time taken by a subject to stand up from sitting position, walk 3 meters, turn around, walk back, and sit down. A decline in TUG performance is evident in PD subjects despite use of dopaminergic therapies to relieve symptoms. Given technological advances, we can measure the TUG, including gait and postural instability, outside the clinic.

Method: To confirm validity of the endpoints, evaluate the devices and support implementation in a clinical trial, we performed Alpha and Beta testing using TUG and gait related parameters. TUG data was collected using video recordings and wearable Smartphone-based app utilizing the sensors in the phone. TUG tests were done every other day over a 1-2 week period (3 tests/day). Insoles with built-in sensors were used, ~6 hours/day for a week, to assess real world performance in various gait parameters. In addition to collecting data, we sought feedback on usability and comfort from participants.

Results: Quantitative TUG measurements were obtained from 12/16 participant using video and phone apps. The total time to complete the TUG was in the range of 9-15 seconds with no differences by age, sex, or modality of measurement.  Our data corroborates independent TUG data collected with the phone app, showing a clear deficit in the total TUG time in PD (N=54) vs controls (N=70), which was correlated with Hoehn and Yahr stage of disease. Performance measurements of gait included cadence, stride length, single and dual support.

Conclusion: These results provide a strong impetus to initiate the natural history study using digital wearable devices to assess the TUG and gait in G2019S LRRK2 PD, with the goal of understanding disease progression and identification of novel digital endpoints for therapeutic trials.

« Back to 2022 International Congress

MDS Abstracts - https://www.mdsabstracts.org/abstract/qualification-of-digital-endpoints-of-function-in-healthy-volunteers-for-use-in-a-decentralized-natural-history-study-in-genetic-parkinsons-disease-pd/