Implementation and Validation of a Biometric Solution for Remote Monitoring of Motor Symptoms in Patients with Huntington's disease in a Phase II Clinical Trial

Objective: To implement and validate quantification of motor dysfunction in a Huntington’s disease (HD) clinical trial using machine learning algorithmic analysis derived from biometric monitoring through a smartphone and wearable sensor combination. Background: Motor symptoms in HD are typically evaluated by clinicians using rating scales, such as the Unified Huntington’s Disease Rating Scale total motor score (UHDRS-TMS). Assessments are infrequent, inherently subjective, may lead to intra- and inter-rater variability, and are prone to placebo response. The use of biometric health solutions could enable objective, real-time monitoring of motor dysfunction in both clinical research and patient care. Design/Methods: Up to 60 HD patients with a pre-defined spectrum of motor dysfunction will be included in a sub-study of Open Pride-HD (NCT02494778; Phase II, open-label, extension study) after meeting a set of pre-specified inclusion and exclusion criteria. Participants will be asked to use a biometric monitoring platform for 6 months. High-frequency movement-tracking data will be collected using an iPhone and wearable Pebble smartwatch combination and streamed continuously to a secure cloud-based analytics platform. 3D accelerometer and gyroscope data that reflect the intensity and direction of movements will be collected at the patients’ homes and during in-clinic visits. Results: The digital health sub-study of Open Pride-HD will begin in December 2016 and is anticipated to conclude within 2 years. The collected information, comprising accelerometer data, app-enabled patient-reported severity assessments of motor dysfunction, and in-clinic sessions, will be used to assess the validity of biometric monitoring as a means of providing reliable information on motor dysfunction in HD patients between and during clinic visits. Conclusions: The design of this innovative study in HD is poised to set the foundation for the development of biometric monitoring solutions as a key component of HD management in clinical trials and everyday care. Study Supported by: This study was funded by Teva Branded Pharmaceutical Products R&D, Inc. Petach Tikva, Israel Disclosure: Dr. Papapetropoulos has received personal compensation for activities with Teva Pharmaceuticals as an employee. Dr. Fine has received personal compensation for activities with Teva as an employee. Dr. Taylor has received personal compensation for activities with Teva Pharmaceuticals as an employee. Dr. Blatt has received personal compensation for activities with Teva Pharmaceuticals as an employee. Dr. Cohen has received personal compensation for activities with Intel Corporation as an employee. Dr. Admati has received personal compensation for activities with Intel Corporation as an employee. Dr. Dolan has received personal compensation for activities with Intel Corporation as an employee. Dr. Lemieux has received personal compensation for activities with Intel Corporation as an employee. Dr. Grachev has received personal compensation for activities with Teva Pharmaceuticals. Dr. Grossman has received personal compensation for activities with Teva Neuroscience as an employee. Dr. Hayden has received personal compensation for activities with Teva Neuroscience as an employee.