Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2 (NCT05152914) | Clinical Trial Compass
Active — Not RecruitingPhase 1/2
Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2
United States5 participantsStarted 2021-11-01
Plain-language summary
This is a phase I/II randomized, masked, clinical trial to determine the safety and efficacy of intravitreal administration of cerliponase alfa.
Who can participate
Age range24 Months – 72 Months
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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 proc…
What they're measuring
1
Monitoring for the development of unacceptable toxicity.