Gene therapy shot aims to save sight in batten disease kids
NCT ID NCT05791864
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early study tests a one-time gene therapy injection under the retina for children with CLN2 Batten disease who already receive enzyme therapy. The main goal is to check safety in 16 participants, with a secondary look at whether it can preserve vision. It is a first-in-human trial, so results are uncertain but could point to a new way to treat the eye symptoms of this rare disease.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
TTX-381 (gene therapy given as a one-time injection under the retina)
What this could lead to
If it works, this could slow or stop vision loss in children with CLN2 Batten disease, improving their quality of life.
What could go wrong
This is a very early, small trial (16 people) focused on safety, not yet on effectiveness. Gene therapy carries risks like inflammation or vision damage, and it may not help everyone.
Disclaimer
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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.
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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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Other studies related to the condition(s) this trial covers.
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