WithdrawnNot Applicable

A Retrospective, Natural History Study in Children With CLN2

Stopped: study stopped due to company decision

0Started 2021-03-31

Plain-language summary

This is a multi center, retrospective, chart review study to document the evolution of ocular disease progression in pediatric patients with CLN2.

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. The participant's legal guardian(s) is(are) willing and able to provide them written, signed informed consent.
✓. The participant has a documented diagnosis of CLN2 disease due to TPP1 deficiency, or has a relative clinically diagnosed with CLN2 disease who has the same CLN2 mutations as the participant
✓. The participant has had one or more eye examinations by an eye care specialist at any time since birth.

Characterize retinal structural changes in children with CLN2

Timeframe: From first available medical chart through informed consent, an average of 10 years

NCT IDNCT04480476

SponsorREGENXBIO Inc.

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2022-04