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

Primary Purpose

CLN3-related Disorders, Low Vision

Status
Recruiting
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
OrCam MyEye 2
Sponsored by
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional device feasibility trial for CLN3-related Disorders focused on measuring OrCam, Legal blindness, Safety, Feasibility, Efficacy

Eligibility Criteria

6 Years - 18 Years (Child, Adult)All SexesDoes not accept healthy volunteers
  • INCLUSION CRITERIA:

To participate in the screening portion of this study, an individual must meet all of the following criteria:

-Has a diagnosis or suspected diagnosis of any genetically based condition causing low vision to the level specified in criteria 2.

If the genetic condition is CLN3-related, the individual must have one of the following:

  • Two CLN3 pathogenic variants,

  • One CLN3 pathogenic variant AND

    • clinical presentation suggestive of CLN3, OR

    • characteristic electron microscopy (EM) findings (such as curvilinear body, fingerprint profile, granular osmiophilic deposits).

      • Has an estimated visual acuity in the better seeing eye < 20/200, without the use of an assistive or augmentative device.
      • Is between 6 to 18 years of age.
      • To participate in the intervention/device use portion of this study, an individual must meet the above screening criteria and the following criteria:
  • Has an appropriate cognitive developmental ability to participate based on Investigators screening assessment.

EXCLUSION CRITERIA:

An individual who meets any of the following criteria will be excluded from participation in this study:

  • Has any of the following auditory dysfunctions: non-reversible or non-correctable hearing loss, tinnitus that is chronic or occurring daily, auditory hallucinations that occur daily.
  • Uses an optic-to-audio assistive device at the time or within 3 months of screening and enrollment.
  • Is unable to travel to the NIH because of medical condition for required in-person portions of the study.
  • Is unable to comply with or have medical conditions that would potentially increase the risk of participation.

Sites / Locations

  • National Institutes of Health Clinical CenterRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Device

Arm Description

use of OrCam MyEye 2.0

Outcomes

Primary Outcome Measures

To assess: a) safety and b) feasibility of using OrCam MyEye 2 by children with CLN3 or low vision
a) Adverse events; b) Feasibility test, Feasibility questionnaire, and Device use diary

Secondary Outcome Measures

To assess the efficacy of using the OrCam by children with CLN3 or low vision
1) Function Test - Efficacy Scores; 2) Ability questionnaire

Full Information

First Posted
July 22, 2021
Last Updated
April 4, 2023
Sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
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1. Study Identification

Unique Protocol Identification Number
NCT04974307
Brief Title
Optic-to-Audio Device in a Pediatric Cohort With CLN3-related Conditions or Low Vision
Official Title
Pilot Study of an Optic-to-Audio Device in a Pediatric Cohort With CLN3-related Conditions or Low Vision
Study Type
Interventional

2. Study Status

Record Verification Date
April 3, 2023
Overall Recruitment Status
Recruiting
Study Start Date
September 30, 2021 (Actual)
Primary Completion Date
January 20, 2025 (Anticipated)
Study Completion Date
January 20, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

Brief 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.
Detailed 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
CLN3-related Disorders, Low Vision
Keywords
OrCam, Legal blindness, Safety, Feasibility, Efficacy

7. Study Design

Primary Purpose
Device Feasibility
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
50 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Device
Arm Type
Experimental
Arm Description
use of OrCam MyEye 2.0
Intervention Type
Device
Intervention Name(s)
OrCam MyEye 2
Intervention Description
The study uses the OrCam MyEye 2, a 22.5-g portable, eyeglass-mounted device that converts camera-captured inputs to auditory outputs. Its advertised functions include text reading, facial recognition, and product identification. The study participants and parents/guardians will be trained on how to use the device. Evaluations for primary and secondary endpoints will be done following 1 week and 1 month of device use.
Primary Outcome Measure Information:
Title
To assess: a) safety and b) feasibility of using OrCam MyEye 2 by children with CLN3 or low vision
Description
a) Adverse events; b) Feasibility test, Feasibility questionnaire, and Device use diary
Time Frame
1 week, 1 month
Secondary Outcome Measure Information:
Title
To assess the efficacy of using the OrCam by children with CLN3 or low vision
Description
1) Function Test - Efficacy Scores; 2) Ability questionnaire
Time Frame
1 week, 1 month

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
INCLUSION CRITERIA: To participate in the screening portion of this study, an individual must meet all of the following criteria: -Has a diagnosis or suspected diagnosis of any genetically based condition causing low vision to the level specified in criteria 2. If the genetic condition is CLN3-related, the individual must have one of the following: Two CLN3 pathogenic variants, One CLN3 pathogenic variant AND clinical presentation suggestive of CLN3, OR characteristic electron microscopy (EM) findings (such as curvilinear body, fingerprint profile, granular osmiophilic deposits). Has an estimated visual acuity in the better seeing eye < 20/200, without the use of an assistive or augmentative device. Is between 6 to 18 years of age. To participate in the intervention/device use portion of this study, an individual must meet the above screening criteria and the following criteria: Has an appropriate cognitive developmental ability to participate based on Investigators screening assessment. EXCLUSION CRITERIA: An individual who meets any of the following criteria will be excluded from participation in this study: Has any of the following auditory dysfunctions: non-reversible or non-correctable hearing loss, tinnitus that is chronic or occurring daily, auditory hallucinations that occur daily. Uses an optic-to-audio assistive device at the time or within 3 months of screening and enrollment. Is unable to travel to the NIH because of medical condition for required in-person portions of the study. Is unable to comply with or have medical conditions that would potentially increase the risk of participation.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
An N Dang Do, M.D.
Phone
(301) 496-8849
Email
an.dangdo@nih.gov
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
An N Dang Do, M.D.
Organizational Affiliation
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Official's Role
Principal Investigator
Facility Information:
Facility Name
National Institutes of Health Clinical Center
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20892
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR)
Phone
800-411-1222
Ext
TTY8664111010
Email
prpl@cc.nih.gov

12. IPD Sharing Statement

Links:
URL
https://clinicalstudies.info.nih.gov/cgi/detail.cgi?A_000414-CH.html
Description
NIH Clinical Center Detailed Web Page

Learn more about this trial

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

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