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Abstract Details

Preliminary Reliability and Validity of a Novel Digital Biomarker Smartphone Application to Assess Cognitive and Motor Symptoms in Huntington’s Disease (HD)
Movement Disorders
P1 - Poster Session 1 (5:30 PM-6:30 PM)
10-042
To determine the reliability and convergent validity of a novel digital biomarker smartphone application for objective, daily assessment of cognition and motor symptoms in Huntington’s disease (HD).

Objective, more precise, longitudinal tracking of HD signs and symptoms through frequent, ecologically valid, at-home testing promises to augment standard clinical testing methods. Standard methods may fail to capture natural symptom fluctuations and be influenced by inter-rater variability, and the clinical visit itself.

A smartphone application composed of seven active tests (Symbol Digit Modalities Test, Stroop Word Reading, Speeded Tapping, Chorea, Balance, U-turn, and 2-Minute-Walk) and continuous passive monitoring is deployed in an ongoing, open-label extension study of huntingtin antisense oligonucleotide RG6042 in early manifest HD (ClinicalTrials.gov. NCT03342053). Forty-one of 46 participants were enrolled and available for the reported baseline analysis. Sensor features were extracted from each active test and aggregated over 2-week periods and compared with standard clinical measures at baseline. Intra-class correlation coefficients and Spearman correlations quantified test-retest reliability and convergent validity of novel digital measures compared with equivalent standard in-clinic tests.

Average adherence was high, with 80% of all active tests performed each week in the first 20 weeks. The test-retest reliability of active tests ranged from 0.74–0.97, median 0.95. Digital versions of standard in-clinic tests showed strong correlations ranging from r=0.69 (P <0.001, Stroop Word Reading) to r=0.86 (P <0.001, Speeded Tapping). Active tests showed good correlations with established clinical motor measures, ranging from r=0.34 (P=0.03, U-Turn) to r=0.64 (P <0.001, Chorea, non-dominant hand). Updated results from ongoing data collection will be presented.

The results suggest that frequent remote patient monitoring yields reliable and valid measures of cognition and motor symptoms in HD when compared with standard clinical measures. The digital biomarker smartphone application has potential to improve measurement of disease progression in HD.

Authors/Disclosures
Florian Lipsmeier
PRESENTER
No disclosure on file
No disclosure on file
No disclosure on file
Anne Smith Anne Smith has received personal compensation for serving as an employee of Ionis Pharmaceuticals. Anne Smith has stock in Ionis Pharmaceuticals.
Scott Schobel Scott Schobel has received personal compensation for serving as an employee of F. Hoffman-La Roche Ltd.
Christian Czech, PhD No disclosure on file
No disclosure on file
No disclosure on file
Edward J. Wild, MBBS, PhD, MRCP (UCL Institute of Neurology) Dr. Wild has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Annexon Biosciences. Dr. Wild has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Takeda. Dr. Wild has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Teitur Trophics. Dr. Wild has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Remix Therapeutics. Dr. Wild has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Hoffman La Roche. Dr. Wild has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Triplet Therapeutics. Dr. Wild has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Vico Therapeutics. Dr. Wild has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Spark Therapeutics. Dr. Wild has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for PTC Therapeutics. Dr. Wild has received personal compensation in the range of $10,000-$49,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for HDBuzz (a registered non-profit). The institution of Dr. Wild has received research support from CHDI Foundation (non-profit). The institution of Dr. Wild has received research support from Hoffman La Roche Ltd.
No disclosure on file