Objective: To identify candidate wearable devices or digital solutions suitable for conducting the Timed Up and Go (TUG) test and evaluate real-world performance remotely during a natural history study of G2019S LRRK2 Parkinson’s disease (PD).

Background: ESB5070 is an experimental precision medicine in development for G2019S LRRK2 PD, offering the potential for disease modification in this targeted genetic population. The Unified Parkinson’s Disease Rating Scale (UPDRS) is a widely accepted clinical scale used in trials of symptomatic treatments; however, dopaminergic induced improvements in rigidity, bradykinesia, and tremor can confound its sensitivity for measuring disease modification. This is problematic for rare genetic PD populations due to sample size limitations in clinical trials. In PD, postural instability is an important manifestation of disease with less influence from dopaminergic therapies and can be measured by the TUG which has good test-retest reliability and shows differences between PD patients and controls. The TUG alongside other real world functional measures may be suitable as endpoints in studies for disease modifying therapies in PD.

Method: We performed a structured assessment of 30 devices and/or mobile applications potentially suitable for a decentralized study to assess change over 12 months in the TUG or TUG subcomponents as well as acquisition of real world performance parameters . The evaluation process involved a structured literature review, consensus ratings, and alpha/beta testing of devices for feasibility and usability. A custom questionnaire was used to evaluate the devices, as well as the published System Usability Scale.

Results: Of the 30 devices studied, three were identified as highly suitable for a natural history study (Encephalog™, Atom5™ Video TUG with Machine Learning, and FeetMe™). These were integrated with other decentralized solutions to help meet the challenges of COVID-19 including telemedicine assessments, patient reported outcomes and home genetic testing.

Conclusion: Digital phenotyping has the potential to provide quality data through longitudinal collection and can be deployed remotely. Decentralized studies raise different challenges and considerations which can be addressed through study design and execution.

« Back to MDS Virtual Congress 2021

MDS Abstracts - https://www.mdsabstracts.org/abstract/overcoming-clinical-research-challenges-during-a-pandemic-selection-and-implementation-of-digital-phenotyping-solutions-in-a-natural-history-study-of-g2019s-lrrk2-parkinsons-disease/