Experimental gene therapy aims to halt rare fatal brain disease in children
NCT ID NCT03770572
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-stage trial tests a one-time gene therapy called CLN-301 in 7 children aged 3 to 10 with CLN3 Batten disease, a rare genetic disorder that causes progressive loss of vision, movement, and thinking skills. The therapy delivers a working copy of the CLN3 gene directly into the spinal fluid to help brain cells function better. The main goals are to check safety and see if the treatment can slow the disease's progression.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- CLN-301 (a gene therapy that delivers a working copy of the CLN3 gene using a modified virus)
- What this could lead to
- If successful, this could slow or stabilize the devastating symptoms of CLN3 Batten disease, offering children more time with better function and quality of life.
- What could go wrong
- This is a very early, small trial (only 7 children) testing safety and dosing. Gene therapies can have side effects, and it is not yet known if the treatment will meaningfully change the course of the disease.
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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
-
Nationwide Children's Hospital
Columbus, Ohio, 43201, United States
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Other studies related to the condition(s) this trial covers.
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