Eye injections aim to save sight in kids with rare batten disease

NCT ID NCT05152914

First seen May 01, 2026 · Last updated May 05, 2026 · Updated 1 time

Summary

This study tests whether injecting a replacement enzyme directly into the eye can slow or stop vision loss in children with CLN2 Batten disease, a rare genetic disorder that causes blindness and brain damage. Five children aged 2 to 6 who already receive enzyme therapy into the brain will get eye injections over two years. The goal is to see if the treatment is safe and can preserve the retina and eyesight.

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Contacts and locations

Locations

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

Conditions

Explore the condition pages connected to this study.