WithdrawnNot Applicable

An Observational Study in Children With CLN2 Batten Disease

Stopped: study stopped due to company decision

0Started 2021-03-31

Plain-language summary

This is a prospective, longitudinal natural history study to document the progression of ocular manifestations of CLN2 disease among a community-dwelling population of pediatric participants affected by this disease.

Who can participate

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

. His or her legal guardian(s) is(are) willing and able to provide them written, signed informed consent.
. Has documented diagnosis of CLN2 disease due to TPP1 deficiency, or has a relative clinically diagnosed with CLN2 with the same mutation as the participant
. Is currently receiving biweekly ERT treatment with cerliponase alfa

Exclusion criteria

. Has had prior treatment with an adeno-associated virus-based AAV gene therapy
. Is currently participating in a clinical trial of investigational product for the treatment of CLN2 disease

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

Change in retinal structure in children with CLN2 Batten disease

Timeframe: 96 weeks

Trial details

NCT IDNCT04462692

SponsorREGENXBIO Inc.

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2023-10

View on ClinicalTrials.gov More Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) trials

Who can participate

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

. His or her legal guardian(s) is(are) willing and able to provide them written, signed informed consent.
. Has documented diagnosis of CLN2 disease due to TPP1 deficiency, or has a relative clinically diagnosed with CLN2 with the same mutation as the participant
. Is currently receiving biweekly ERT treatment with cerliponase alfa

Exclusion criteria

. Has had prior treatment with an adeno-associated virus-based AAV gene therapy
. Is currently participating in a clinical trial of investigational product for the treatment of CLN2 disease