Evidence map›Paper›PMID 41933359›Full record

ArticleOrphanet journal of rare diseases2026

Prospective pilot safety, feasibility study of an optic-to-audio device for children with CLN3 disease.

Thuy Tien Nguyen, Andrea Munoz, Kisha Jenkins, Ibukun Agbede, Wadih M Zein, Laryssa A Huryn, Brian P Brooks, Jennifer Chisholm, Julie Christensen, Christopher Zalewski and 4 more

Registry-linked trialAbstract readClinical Study
PubMed Full text at PMC Publisher
In one paragraph

Article in Orphanet journal of rare diseases, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04974307 (Pilot Study of an Optic-to-Audio Device in a Pediatric Cohort With CLN3-related Conditions or Low Vision), which is not on this map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–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.

NCT04974307 naterminatednot on this map

Pilot Study of an Optic-to-Audio Device in a Pediatric Cohort With CLN3-related Conditions or Low Vision

TypeinterventionalSponsorEunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)Ran2021 to 2023Enrolled11ConditionsCLN3-related Disorders, Non-CLN3 Related Low VisionArmsOrCam MyEye 2
3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

14 authors.

Thuy Tien Nguyen *Occupational Therapy Service, Mark O. Hatfield Clinical Center, National Institutes of Health, Bethesda, MD, USA.
Andrea Munoz *Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD, USA.
Kisha JenkinsEunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD, USA.
Ibukun AgbedeEunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD, USA.
Wadih M ZeinOphthalmic Genetics and Visual Function Branch, National Eye Institute, National Institutes of Health, Bethesda, MD, USA.
Laryssa A HurynOphthalmic Genetics and Visual Function Branch, National Eye Institute, National Institutes of Health, Bethesda, MD, USA.
Brian P BrooksOphthalmic Genetics and Visual Function Branch, National Eye Institute, National Institutes of Health, Bethesda, MD, USA.
Jennifer ChisholmAuditory and Vestibular Clinical Research Section, National Institute on Deafness and Communication Disorders, National Institutes of Health, Bethesda, MD, USA.
Julie ChristensenAuditory and Vestibular Clinical Research Section, National Institute on Deafness and Communication Disorders, National Institutes of Health, Bethesda, MD, USA.
Christopher ZalewskiAuditory and Vestibular Clinical Research Section, National Institute on Deafness and Communication Disorders, National Institutes of Health, Bethesda, MD, USA.
M Teresa MagoneConsult Services Section, National Eye Institute, National Institutes of Health, Bethesda, MD, USA.
Colby ChlebowskiNational Institute of Mental Health, National Institutes of Health, Bethesda, MD, USA.
Audrey ThurmNational Institute of Mental Health, National Institutes of Health, Bethesda, MD, USA.
An N Dang DoUnit on Cellular Stress in Development and Diseases, Eunice Kennedy Shriver National Institute of Health and Human Development, National Institutes of Health, 10 Center Drive, Bethesda, MD, 20892-1103, USA. an.dangdo@nih.gov.ORCID http://orcid.org/0000-0002-4171-0493

Funding

Clinical Analysis Of Disorders Of Hearing And BalanceZIADC000064 · NIDCD · NATIONAL INSTITUTE ON DEAFNESS AND OTHER COMMUNICATION DISORDERS · PI HERTZANO, RONNA · 2009 to 2025
$25.2M
Research Investigations of Cellular Stress in Development and DiseasesZIAHD009001 · NICHD · EUNICE KENNEDY SHRIVER NATIONAL INSTITUTE OF CHILD HEALTH & HUMAN DEVELOPMENT · PI DANG DO, AN · 2022 to 2025
$4.9M
Intramural NIH HHS ZIA HD009001NIDCD NIH HHS ZIA DC000064
6 · The paper itself

Abstract

backgroundLow-vision rehabilitative support for children with multiple-disability conditions is underexplored. We conduct a pilot study of an assistive device in children with CLN3 disease, a multisystemic pediatric blindness and neurodegenerative condition. The prospective, pilot study (NCT04974307) evaluated the safety, feasibility and preliminary efficacy of the OrCam MyEye 2 in aiding daily living tasks. We used multimodal assessments of feasibility and efficacy to assess nine participants with CLN3 disease and one with non-CLN3 low vision (age 8.1-17.4 years; females:males 2:8) at baseline (Day 1, without the device) and Day 5 (with the device).

resultsSurveyed parents and affected children reported desire for future research addressing the vision loss in CLN3 disease, and lower quality of life in vision-related domains. One grade 1 adverse event was recorded during the study. Feasibility and Function assessments showed that > 90% of evaluable participants scored above thresholds on feasibility assessments and > 25% had improved scores and performance time on device-specific tasks.

conclusionsThe data provide indications for the safety, feasibility and task-specific efficacy for the OrCam MyEye 2. They support further, including longitudinal, evaluation and consideration of such optic-to-audio devices as part of the toolbox for low vision therapy for children with multiple disabilities such as CLN3 disease.

trial registrationclinicaltrials.gov, NCT04974307. Registered 23 July 2021, https//clinicaltrials.gov/study/NCT04974307 .

Indexed as

Neuronal Ceroid-LipofuscinosesAdolescentChildFeasibility StudiesFemaleHumansMalePilot ProjectsProspective StudiesQuality of LifeAssistive deviceJuvenile battenLow visionPatient-reported outcomes

Identifiers

PMID41933359
PMCPMC13173725

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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.