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. 2022 Jan 10;6(1):1-8.
doi: 10.1159/000521232. eCollection 2022.

A Smartphone Application as an Exploratory Endpoint in a Phase 3 Parkinson's Disease Clinical Trial: A Pilot Study

Alex Page  1   2 , Norman Yung  1 , Peggy Auinger  1   2 , Charles Venuto  1   2 , Alistair Glidden  1 , Eric Macklin  3 , Larsson Omberg  4 , Michael A Schwarzschild  5 , E Ray Dorsey  1   2
Affiliations

A Smartphone Application as an Exploratory Endpoint in a Phase 3 Parkinson's Disease Clinical Trial: A Pilot Study

Alex Page et al. Digit Biomark. .

Abstract

Background: Smartphones can generate objective measures of Parkinson's disease (PD) and supplement traditional in-person rating scales. However, smartphone use in clinical trials has been limited.

Objective: This study aimed to determine the feasibility of introducing a smartphone research application into a PD clinical trial and to evaluate the resulting measures.

Methods: A smartphone application was introduced part-way into a phase 3 randomized clinical trial of inosine. The application included finger tapping, gait, and cognition tests, and participants were asked to complete an assessment battery at home and in clinic alongside the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS).

Results: Of 236 eligible participants in the parent study, 88 (37%) consented to participate, and 59 (27 randomized to inosine and 32 to placebo) completed a baseline smartphone assessment. These 59 participants collectively completed 1,292 batteries of assessments. The proportion of participants who completed at least one smartphone assessment was 61% at 3, 54% at 6, and 35% at 12 months. Finger tapping speed correlated weakly with the part III motor portion (r = -0.16, left hand; r = -0.04, right hand) and total (r = -0.14) MDS-UPDRS. Gait speed correlated better with the same measures (r = -0.25, part III motor; r = -0.34, total). Over 6 months, finger tapping speed, gait speed, and memory scores did not differ between those randomized to active drug or placebo.

Conclusions: Introducing a smartphone application midway into a phase 3 clinical trial was challenging. Measures of bradykinesia and gait speed correlated modestly with traditional outcomes and were consistent with the study's overall findings, which found no benefit of the active drug.

Keywords: Clinical trial; Cognition; Gait; Inosine; Movement; Parkinson disease; Smartphone; Telemedicine.

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Conflict of interest statement

Dr. L. Omberg has received research support from the National Institutes of Health/National Institute of Neurological Disorders and Stroke. Dr. M.A. Schwarzschild has been (or expects to be) paid by the following commercial entities for service on a data monitoring committee (by Eli Lilly and Co.) and for service on scientific advisory boards both directly (by Prevail Therapeutics and Denali Therapeutics) and indirectly (via the Parkinson Study Group; by nQ Medical, Chase Therapeutics, Partner Therapeutics and Bial Biotech). He has also received royalty payments (via the Massachusetts General Hospital) for licensing of an adenosine A2A knockout mouse line. Dr. E.R. Dorsey has received honoraria for speaking at the American Neurological Association, Excellus BlueCross BlueShield, International Parkinson's and Movement Disorders Society, National Multiple Sclerosis Society, Northwestern University, Stanford University, Texas Neurological Society, and Weill Cornell; received compensation for consulting services from Abbott, Abbvie, Acadia, Acorda, Alzheimer's Drug Discovery Foundation, Ascension Health Alliance, Biogen, BluePrint Orphan, Clintrex, Curasen Therapeutics, DeciBio, Denali Therapeutics, Eli Lilly, Grand Rounds, Huntington Study Group, medical-legal services, Medical Communications Media, Medopad, Medrhythms, Michael J. Fox Foundation, MJH Holding LLC, NACCME, Olson Research Group, Origent Data Sciences, Otsuka, Pear Therapeutic, Praxis, Prilenia, Roche, Sanofi, Spark, Springer Healthcare, Sunovion Pharma, Sutter Bay Hospitals, Theravance, University of California Irvine, and WebMD; research support from Acadia Pharmaceuticals, Biogen, Biosensics, Burroughs Wellcome Fund, CuraSen, Greater Rochester Health Foundation, Huntington Study Group, Michael J. Fox Foundation, National Institutes of Health, Patient-Centered Outcomes Research Institute, Pfizer, PhotoPharmics, Safra Foundation, and Wave Life Sciences; editorial services for Karger Publications; and ownership interests with Grand Rounds (second opinion service). The remaining authors have no conflicts to report.

Figures

Fig. 1
Fig. 1
Examples from the mPower application, including (from left to right) an evaluation of finger tapping, a voice assessment, and a memory test.
Fig. 2
Fig. 2
Participant flow.
Fig. 3
Fig. 3
Tapping speed and variability in active (inosine) and placebo arms. a Tapping speed in best hand. b Tapping speed in worst hand. c Tapping variability in best hand. d Tapping variability in worst hand.
Fig. 4
Fig. 4
Gait speed at baseline and month 6 among active smartphone participants.
See this image and copyright information in PMC

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