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.
Disclaimer
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the original study
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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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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
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Locations
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Nationwide Children's Hospital
Columbus, Ohio, 43201, United States