Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2

Running, not enrolling · Phase 1/Phase 2

Conditions studied: Neuronal Ceroid Lipofuscinosis Type 2

In brief

This is a phase I/II randomized, masked, clinical trial to determine the safety and efficacy of intravitreal administration of cerliponase alfa.

Key facts

Study ID

NCT05152914

Run by

David L Rogers, MD

People needed

5

Starts

2021-11-01

Expected to finish

2027-03-06

Last updated by the study team

2026-04-30

Who can join

Age: 2 and older, up to 6. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Nationwide Children's Hospital — Columbus, Ohio, United States

Full record on ClinicalTrials.gov

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