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

Low Vision Clinical Trial

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

Status Completed

Clinical Trial Summary

Background:

CLN3 involves vision loss observed around the preschool years, with eventual progression to blindness within 1-3 years. Researchers want to test an assistive device that may help children with CLN3 or blindness.

Objective:

To learn if it is safe, easy, and useful for children with CLN3 or blindness to use the OrCam.

Eligibility:

People aged 6-18 years who have either CLN3-related disease or blindness.

Design:

Participants will be screened with the following:

Medical history

Physical exam

Family history

Eye exam and vision tests. They will get eyedrops to dilate their eyes.

Psychological and neurocognitive tests. They will be asked questions and observed for how they do various tasks, such as talking, playing, writing, drawing, and solving problems.

Hearing tests. They may wear headphones or earplugs. Electrodes may be taped to their head.

Blood samples

Skin biopsy, if needed

Cheek cell, saliva, or urine samples

The OrCam is the size of and weighs about half as much as a pack of gum. It is attached to eyeglass frames by magnets. Participants will do tasks before and after they have been trained on the OrCam. They will do these tasks without or with using the OrCam.

Participants will be given an OrCam to use for 1 week or 1 month. They will have check-in sessions with the study team.

Participants and/or their caregivers will be asked about abilities, behaviors, social skills, learning methods, intelligence, and health-related quality of life.

Participants samples may be used for genetic testing and/or to make a type of stem cell.

Participation will last for 1-5 weeks.

Clinical Trial Description

Study Description:

This is a pilot study of an augmentative visual device, OrCam MyEye 2 (OrCam), in pediatric individuals with CLN3-related conditions or low vision. We hypothesize that, given the relatively simple design and operating procedure of the device, the use of the OrCam by study participants will be safe and feasible. We also hypothesize that the device will enhance their ability to obtain visually based information.

Objectives:

Primary:

Assess the safety and feasibility of OrCam use by children with CLN3 or low vision.

Secondary:

Assess the effectiveness of OrCam use by children with CLN3 or low vision.

Exploratory:

Assess the feasibility and effectiveness of OrCam use in CLN3 versus non-CLN3 groups. Optimize methods for assessing efficacy of visual accommodation/assistive devices for the CLN3/NCL population

Endpoints:

Primary:

1) Adverse events during the use of OrCam. 2) Feasibility test. 3) Feasibility questionnaire. 4) Device use diary. The assessment periods will be 1 week at study site and 1 month at home.

Secondary:

1) Function test of using OrCam. 2) Ability questionnaire.

Exploratory:

1) Function test. 2) Feasibility, Ability, Applicability questionnaires. ;

Related Conditions & MeSH terms

• Low Vision
• Vision, Low

• NCT number: NCT04974307
• Study type: Interventional
• Source: National Institutes of Health Clinical Center (CC)
• Status: Completed
• Phase: N/A
• Start date: September 30, 2021
• Completion date: October 7, 2023