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

SexALL

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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

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