Eye injection may save sight in kids with rare batten disease
NCT ID NCT05152914
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests whether injecting a replacement enzyme directly into the eye can slow or stop vision loss in children with CLN2 Batten disease, a rare genetic disorder that causes blindness and brain damage. Five children aged 2 to 6 who already receive brain infusions of the enzyme will get eye injections over two years. The goal is to see if the treatment is safe and helps preserve retinal structure and eyesight.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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About 5 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Nov 2021
- Expected to finish
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Mar 2027
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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24 to 72 months
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Genotypic confirmation of classical CLN2 Batten's disease from a CLIA certified lab. * Enzyme level deficiency of tripeptidyl-peptidase * Minimum age requirement: 24 months of age at enrollment * Maximum age requirement: 72 months of age at enrollment * Currently receiving intraventricular cerliponase alfa * Willing to participate in the proposed study visits over the 2-year period * Minimum central retinal thickness (CRT) of 140μm based upon OCT assessment * Clear ocular media * No ocular pathology present to account for vision loss other than optic atrophy and pigmentary retinopathy that is felt to be due to the CLN2 disease process Exclusion Criteria: * Any opacities in the clear ocular media including vitreous debris. * History of ocular trauma or prior ocular surgery. * Episode of generalized motor status epilepticus within four weeks before the First Dose visit * Severe infection (e.g., upper respiratory tract infection, pneumonia, pyelonephritis, or meningitis) within four weeks before the First Dose visit (enrollment may be postponed) * Those with a history of bleeding disorders. * History of or current chemotherapy, radiotherapy or other immunosuppression therapy within the past 30 days (corticosteroid treatment may be permitted at the discretion of the PI) * Has a medical condition, or extenuating circumstance that, in the opinion of the investigator, might compromise the subject's ability to comply with the protocol required testing or procedures or compromise the subject's wellbeing, safety, or clinical interpretability
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.