Evidence map›Paper›PMID 35763342›Full record

ArticleJournal of medical Internet research2022

A Remote Digital Monitoring Platform to Assess Cognitive and Motor Symptoms in Huntington Disease: Cross-sectional Validation Study.

Florian Lipsmeier, Cedric Simillion, Atieh Bamdadian, Rosanna Tortelli, Lauren M Byrne, Yan-Ping Zhang, Detlef Wolf, Anne V Smith, Christian Czech, Christian Gossens and 5 more

2 registry-linked trialsAbstract read
In one paragraph

Article in Journal of medical Internet research, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 21 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
21citing papers in PubMed, 3 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT03342053 phase2completednot on this map

An Open-Label Extension Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RO7234292 (ISIS 443139) in Huntington's Disease Patients Who Participated in Prior Investigational Studies of RO7234292 (ISIS 443139)

TypeinterventionalSponsorHoffmann-La RocheRan2018 to 2019Enrolled46ConditionsHuntington's DiseaseArmsRO7234292 (RG6042)
NCT03664804 phase1completednot on this map

A Multi-Site, Prospective, Longitudinal, Cohort Study Measuring Cerebrospinal Fluid-Mutant Huntingtin Protein in Patients With Huntington's Disease

TypeinterventionalSponsorHoffmann-La RocheRan2018 to 2022Enrolled95ConditionsHuntington's DiseaseArmsNo Study Drug was Administered in this Study
3 · Its place in the literature

Who cites it

21 citing papers in PubMed, 3 syntheses or guidelines pooled it.

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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

15 authors.

Florian LipsmeierRoche Pharma Research and Early Development, pRED Informatics, Pharmaceutical Sciences, Clinical Pharmacology, and Neuroscience, Ophthalmology, and Rare Diseases Discovery and Translational Area, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland.ORCID 0000-0002-8663-819X
Cedric SimillionRoche Pharma Research and Early Development, pRED Informatics, Pharmaceutical Sciences, Clinical Pharmacology, and Neuroscience, Ophthalmology, and Rare Diseases Discovery and Translational Area, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland.ORCID 0000-0002-2026-6741
Atieh BamdadianRoche Pharma Research and Early Development, pRED Informatics, Pharmaceutical Sciences, Clinical Pharmacology, and Neuroscience, Ophthalmology, and Rare Diseases Discovery and Translational Area, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland.ORCID 0000-0003-0459-9244
Rosanna TortelliHuntington's Disease Centre, UCL Queen Square Institute of Neurology, University College London, London, United Kingdom.ORCID 0000-0003-4499-9891
Lauren M ByrneHuntington's Disease Centre, UCL Queen Square Institute of Neurology, University College London, London, United Kingdom.ORCID 0000-0003-1650-4273
Yan-Ping ZhangRoche Pharma Research and Early Development, pRED Informatics, Pharmaceutical Sciences, Clinical Pharmacology, and Neuroscience, Ophthalmology, and Rare Diseases Discovery and Translational Area, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland.ORCID 0000-0001-7602-4453
Detlef WolfRoche Pharma Research and Early Development, pRED Informatics, Pharmaceutical Sciences, Clinical Pharmacology, and Neuroscience, Ophthalmology, and Rare Diseases Discovery and Translational Area, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland.ORCID 0000-0002-4487-8419
Anne V SmithIonis Pharmaceuticals Inc, Carlsbad, CA, United States.ORCID 0000-0001-8190-3006
Christian CzechRoche Pharma Research and Early Development, pRED Informatics, Pharmaceutical Sciences, Clinical Pharmacology, and Neuroscience, Ophthalmology, and Rare Diseases Discovery and Translational Area, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland.ORCID 0000-0001-7746-0134
Christian GossensRoche Pharma Research and Early Development, pRED Informatics, Pharmaceutical Sciences, Clinical Pharmacology, and Neuroscience, Ophthalmology, and Rare Diseases Discovery and Translational Area, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland.ORCID 0000-0003-2660-5895
Patrick WeydtDepartment of Neurology, University of Ulm Medical Center, Ulm, Germany.ORCID 0000-0003-2344-5662
Scott A SchobelF. Hoffmann-La Roche Ltd, Basel, Switzerland.ORCID 0000-0001-6182-1943
Filipe B RodriguesHuntington's Disease Centre, UCL Queen Square Institute of Neurology, University College London, London, United Kingdom.ORCID 0000-0002-5567-7938
Edward J WildHuntington's Disease Centre, UCL Queen Square Institute of Neurology, University College London, London, United Kingdom.ORCID 0000-0002-6921-7887
Michael LindemannRoche Pharma Research and Early Development, pRED Informatics, Pharmaceutical Sciences, Clinical Pharmacology, and Neuroscience, Ophthalmology, and Rare Diseases Discovery and Translational Area, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland.ORCID 0000-0003-1010-3890

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundRemote monitoring of Huntington disease (HD) signs and symptoms using digital technologies may enhance early clinical diagnosis and tracking of disease progression, guide treatment decisions, and monitor response to disease-modifying agents. Several recent studies in neurodegenerative diseases have demonstrated the feasibility of digital symptom monitoring.

objectiveThe aim of this study was to evaluate a novel smartwatch- and smartphone-based digital monitoring platform to remotely monitor signs and symptoms of HD.

methodsThis analysis aimed to determine the feasibility and reliability of the Roche HD Digital Monitoring Platform over a 4-week period and cross-sectional validity over a 2-week interval. Key criteria assessed were feasibility, evaluated by adherence and quality control failure rates; test-retest reliability; known-groups validity; and convergent validity of sensor-based measures with existing clinical measures. Data from 3 studies were used: the predrug screening phase of an open-label extension study evaluating tominersen (NCT03342053) and 2 untreated cohorts-the HD Natural History Study (NCT03664804) and the Digital-HD study. Across these studies, controls (n=20) and individuals with premanifest (n=20) or manifest (n=179) HD completed 6 motor and 2 cognitive tests at home and in the clinic.

resultsParticipants in the open-label extension study, the HD Natural History Study, and the Digital-HD study completed 89.95% (1164/1294), 72.01% (2025/2812), and 68.98% (1454/2108) of the active tests, respectively. All sensor-based features showed good to excellent test-retest reliability (intraclass correlation coefficient 0.89-0.98) and generally low quality control failure rates. Good overall convergent validity of sensor-derived features to Unified HD Rating Scale outcomes and good overall known-groups validity among controls, premanifest, and manifest participants were observed. Among participants with manifest HD, the digital cognitive tests demonstrated the strongest correlations with analogous in-clinic tests (Pearson correlation coefficient 0.79-0.90).

conclusionsThese results show the potential of the HD Digital Monitoring Platform to provide reliable, valid, continuous remote monitoring of HD symptoms, facilitating the evaluation of novel treatments and enhanced clinical monitoring and care for individuals with HD.

Indexed as

Huntington DiseaseMotor SkillsCognitionCross-Sectional StudiesHumansOligonucleotidesReproducibility of ResultsSensitivity and SpecificityOligonucleotidesclinical trialscognitiondigital biomarkersdigital monitoringHuntington diseasemobile phonemotorremote monitoringsmartphonesmartwatch

Identifiers

PMID35763342
PMCPMC9277525

What OpenQuestion holds

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LicenceCC BY
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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.