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 range
24 Months – 72 Months
Sex
ALL
See this in plain English?
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…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Monitoring for the development of unacceptable toxicity.