What side effects are associated with this type of intervention?

Batten Disease, particularly CLN3-related conditions, often involves treatments aimed at managing symptoms and improving quality of life. Assistive devices like the OrCam, which provide visual and auditory assistance, are generally safe but may cause minor issues such as ocular discomfort or vision problems. Broadly, treatments for Batten Disease can include enzyme replacement therapy, gene therapy, and symptom management strategies, which may have side effects like immune reactions, infection risks, and discomfort at the injection site. It is important to consult with a healthcare provider to understand the specific risks and benefits of each treatment option.

What prior FDA approvals exist?

The FDA has approved cerliponase alfa (Brineura) for the treatment of Batten Disease, specifically for CLN2, a form of the disease. Brineura is an enzyme replacement therapy that helps slow the progression of the disease. While there are no specific FDA-approved treatments for visual and auditory assistance in Batten Disease, assistive devices like the OrCam are being studied to help improve the quality of life for patients by aiding in daily activities despite vision loss.

What other types of research exist?

Promising alternatives to OrCam for Batten Disease patients include: 1) Virtual Reality Orientation and Mobility (VR-O&M) tests, which help quantify and improve functional vision. 2) Game platforms for vision therapy, such as those using modified game applications on devices like the Apple iPod Touch. 3) Low-intensity laser light projection devices, which have shown improvements in reading abilities for low-vision patients.

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Optic-to-Audio Device for Low Vision

Q: What is the mechanism of action of this treatment?

Batten Disease, particularly CLN3-related conditions, leads to progressive vision loss and eventual blindness. The OrCam device, an assistive technology, is being studied for its potential to aid children with CLN3 or blindness by converting visual information into audio cues. This mechanism of action is crucial as it provides an alternative sensory input to compensate for the loss of vision, thereby enhancing the patient's ability to interact with their environment and maintain a degree of independence. The emphasis on visual and auditory assistance in treatments like OrCam is significant because it directly addresses the sensory deficits caused by Batten Disease, potentially improving the quality of life for patients.

N/A

Waitlist Available

Led By An N Dang Do, M.D.

Research Sponsored by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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

Must not have

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

Is unable to travel to the NIH because of medical condition for required in-person portions of the study

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 week, 1 month

Awards & highlights

No Placebo-Only Group

Summary

This trial tests the OrCam MyEye 2, a small device that attaches to glasses and helps people with vision problems by reading text and recognizing faces and objects. It targets children aged 6-18 who have CLN3-related disease or blindness. The device works by using a camera to capture visual information and then providing audio descriptions to the user. Researchers aim to see if it is safe, easy to use, and effective for these children.

Who is the study for?

This trial is for children aged 6-18 with CLN3-related disease or severe vision loss, having visual acuity less than 20/200. They should have cognitive abilities to participate and not use similar devices recently. Those with certain hearing issues, travel restrictions due to health, or conditions increasing study risks are excluded.

What is being tested?

The OrCam MyEye 2 device is being tested on kids with CLN3 or low vision to see if it's safe and helpful. It's a small device that attaches to glasses and helps the visually impaired by converting visual information into audio cues.

What are the potential side effects?

Since the OrCam MyEye 2 is a non-invasive assistive device, there may be minimal side effects related mainly to comfort of wearing the device or potential psychological impact from dependency on technology for assistance.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My best eye sees worse than 20/200 without help.

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I am between 6 and 18 years old.

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I have or might have a genetic condition causing significant vision loss.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I experience daily tinnitus, hearing loss that can't be corrected, or daily auditory hallucinations.

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I cannot travel to NIH for the study due to my health condition.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 week, 1 month

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1 week, 1 month

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 week, 1 month for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Participants With Adverse Events

Secondary study objectives

To assess the efficacy of using the OrCam by children with CLN3 or low vision

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Children with Low vision without CLN3Experimental Treatment1 Intervention

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.

Group II: Children with CLN3 diseaseExperimental Treatment1 Intervention

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.

0 / 5Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Batten Disease, particularly CLN3-related conditions, leads to progressive vision loss and eventual blindness. The OrCam device, an assistive technology, is being studied for its potential to aid children with CLN3 or blindness by converting visual information into audio cues. This mechanism of action is crucial as it provides an alternative sensory input to compensate for the loss of vision, thereby enhancing the patient's ability to interact with their environment and maintain a degree of independence. The emphasis on visual and auditory assistance in treatments like OrCam is significant because it directly addresses the sensory deficits caused by Batten Disease, potentially improving the quality of life for patients.

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