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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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.
Contacts and locations
Locations
-
Greater Ormond Street Hospital
RECRUITINGLondon, Wc1N 3JH, United Kingdom
-
University Medical Center Hamburg-Eppendorf (UKE)- Childrens Hospital
RECRUITINGHamburg, Germany
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