Gene therapy trial targets blindness in rare batten disease
NCT ID NCT05791864
First seen Jan 06, 2026 · Last updated May 07, 2026 · Updated 22 times
Summary
This study tests a new gene therapy, TTX-381, for vision problems caused by CLN2 disease (a form of Batten disease). About 16 people who already receive brain enzyme infusions will get a single eye injection to see if it is safe and can slow vision loss. The goal is to protect the retina while continuing standard treatment.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Greater Ormond Street Hospital
RECRUITINGLondon, Wc1N 3JH, United Kingdom
Contact Email: •••••@•••••
Contact
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University Medical Center Hamburg-Eppendorf (UKE)- Childrens Hospital
RECRUITINGHamburg, Germany
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
Conditions
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