CLN3-related DisordersNCT04974307
Optic-to-Audio Device in a Pediatric Cohort With CLN3-related Conditions or Low Vision
A study testing OrCam MyEye 2 in people with CLN3-related Disorders and Non-CLN3 Related Low Vision, run by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) at 1 site in United States.
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 with non-CLN3 related blindness. Read more about this study
- Who is it for?
- Ages 6 to 18 years
- Conditions the study is for
- CLN3-related Disorders, Non-CLN3 Related Low Vision
- What is being tested?
- OrCam MyEye 2
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Sep 2021to 7 Oct 2023
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- To participate in the screening portion of this study, an individual must meet all of the following criteria:
- Have 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:
- a. Two CLN3 pathogenic variants, OR
- b. One CLN3 pathogenic variant AND
- i. clinical presentation suggestive of CLN3, OR
Show all 11Show fewer
- ii. characteristic electron microscopy (EM) findings (such as curvilinear body, fingerprint profile, granular osmiophilic deposits).
- Have 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:
- Have an appropriate cognitive developmental ability to participate based on Investigators screening assessment.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
About this study
Official title: Pilot Study of an Optic-to-Audio Device in a Pediatric Cohort With CLN3-related Conditions or Low Vision
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 with non-CLN3 related blindness.
Read more
Objective:
To learn if it is safe, easy, and useful for children with CLN3 or with non-CLN3 related blindness to use the OrCam.
Eligibility:
People aged 6-18 years who have either CLN3-related disease or non-CLN3 related 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.
Read the 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.
Endpoints:
Primary:
- Adverse events during the use of OrCam
- Feasibility test
- Feasibility questionnaire
The assessment periods will be 1 week at study site.
- Being tested
- OrCam MyEye 2
- Keywords
- OrCam, Legal blindness, Safety, Feasibility, Efficacy
- Medical subject headings
- Blindness
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OrCam MyEye 2Device
The OrCam MyEye 2 is 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.
| Study group | Role | What they receive |
|---|---|---|
| Children with CLN3 disease | Experimental | Children with CLN3 use the OrCam MyEye 2, a 22.5-g portable, eyeglass-mounted device that converts camera-captured inputs to auditory outputs for text reading, facial recognition, and product identification. The study participants and parents/guardians were trained on how to use the device. Receives: Device: OrCam MyEye 2 |
| Children with Low vision without CLN3 | Experimental | Children with low vision not related to CLN3 use the OrCam MyEye 2, a 22.5-g portable, eyeglass-mounted device that converts camera-captured inputs to auditory outputs for text reading, facial recognition, and product identification. The study participants and parents/guardians were trained on how to use the device. Receives: Device: OrCam MyEye 2 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Participants With Adverse Events
Number of participants with adverse events related to the use of OrCam MyEye 2. Adverse events were collected from parental report.
Measured: 1 week
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jul 2021
- Study start30 Sep 2021
- Primary completion6 Oct 2023
- Study completion7 Oct 2023
Why it stopped: Early termination due to low enrollment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for CLN3-related DisordersStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Device feasibility
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- An N Dang Do, M.D., Principal investigator, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04974307
- Sponsor's protocol ID
- 10000414
- Other IDs
- 000414-CH
- Status last verified
- Feb 2024
- First submitted
- 22 Jul 2021
- Last updated
- 23 Oct 2024
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- No
- FDA-regulated device
- Yes
- Individual data sharing
- Yes: De-identified individual participant data (IPD) that underlie results in a publication will be shared.
Publications
Nguyen TT, Munoz A, Jenkins K, Agbede I, Zein WM, Huryn LA, Brooks BP, Chisholm J, Christensen J, Zalewski C, Magone MT, Chlebowski C, Thurm A, Dang Do AN. Prospective pilot safety, feasibility study of an optic-to-audio device for children with CLN3 disease. Orphanet J Rare Dis. 2026 Apr 3;21(1):199. doi: 10.1186/s13023-026-04319-0.
PubMed 41933359
Study at a glance
From the registry record, last updated by the sponsor on 23 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study of an Optic-to-Audio Device in a Pediatric Cohort With CLN3-related Conditions or Low Vision
- ClinicalTrials.gov ID
- NCT04974307
- Acronym / sponsor ID
- 10000414
- Keywords
- OrCam, Legal blindness, Safety, Feasibility, Efficacy
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Early termination due to low enrollment
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)US National Institutes of Health
- Study start
- 30 Sep 2021 (actual)
- Primary completion
- 6 Oct 2023 (actual)
- Study completion
- 7 Oct 2023 (actual)
- First posted
- 23 Jul 2021 (actual)
- Results
- Posted 23 Oct 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 with non-CLN3 related blindness.
Objective:
To learn if it is safe, easy, and useful for children with CLN3 or with non-CLN3 related blindness to use the OrCam.
Eligibility:
People aged 6-18 years who have either CLN3-related disease or non-CLN3 related 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.
Endpoints:
Primary:
- Adverse events during the use of OrCam
- Feasibility test
- Feasibility questionnaire
The assessment periods will be 1 week at study site.
Study design
- Primary purpose
- Device feasibility
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Children with CLN3 disease | Experimental | Device: OrCam MyEye 2Children with CLN3 use the OrCam MyEye 2, a 22.5-g portable, eyeglass-mounted device that converts camera-captured inputs to auditory outputs for text reading, facial recognition, and product identification. The study participants and parents/guardians were trained on how to use the device. |
| Children with Low vision without CLN3 | Experimental | Device: OrCam MyEye 2Children with low vision not related to CLN3 use the OrCam MyEye 2, a 22.5-g portable, eyeglass-mounted device that converts camera-captured inputs to auditory outputs for text reading, facial recognition, and product identification. The study participants and parents/guardians were trained on how to use the device. |
Who may qualify
- Ages
- 6 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (11 listed)
- To participate in the screening portion of this study, an individual must meet all of the following criteria:
- Have a diagnosis or suspected diagnosis of any genetically based condition causing low vision to the level specified in criteria 2.
- Have 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:
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| OrCam MyEye 2The OrCam MyEye 2 is 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. | Device | Children with CLN3 disease, Children with Low vision without CLN3 |
Primary outcome
Participants With Adverse Events
Time frame: 1 week
Number of participants with adverse events related to the use of OrCam MyEye 2. Adverse events were collected from parental report.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Oct 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 41933359Nguyen TT, Munoz A, Jenkins K, Agbede I, Zein WM, Huryn LA, Brooks BP, Chisholm J, Christensen J, Zalewski C, Magone MT, Chlebowski C, Thurm A, Dang Do AN. Prospective pilot safety, feasibility study of an optic-to-audio device for children with CLN3 disease. Orphanet J Rare Dis. 2026 Apr 3;21(1):199. doi: 10.1186/s13023-026-04319-0.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. De-identified individual participant data (IPD) that underlie results in a publication will be shared.
Related trials for CLN3-related Disorders
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
RecruitingObservational study
Investigations of Juvenile Neuronal Ceroid Lipofuscinosis
Juvenile Neuronal Ceroid Lipofuscinosis (CLN3), Batten Disease
RecruitingObservational study
NYSCF Scientific Discovery Biobank
ALS, Amyotrophic Lateral Sclerosis
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
An N Dang Do, M.D. principal investigator
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing OrCam MyEye 2 in people with CLN3-related Disorders and Non-CLN3 Related Low Vision, run by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 23 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 23 Oct 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Participants With Adverse Events (time frame: 1 week). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04974307, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Oct 2024.