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

BACKGROUND: Low-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). RESULTS: Surveyed 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. CONCLUSIONS: The 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 REGISTRATION: clinicaltrials.gov, NCT04974307. Registered 23 July 2021, https//clinicaltrials.gov/study/NCT04974307 .