Gene therapy for rare brain disease tracked over years
NCT ID NCT04273243
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study follows 10 people with CLN6 Batten disease who received a single dose of gene therapy (AT-GTX-501) in an earlier trial. Researchers are checking long-term safety and how the disease progresses over time. No new treatment is given in this follow-up.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
AT-GTX-501 (gene therapy)
What this could lead to
If successful, this follow-up could show that a single dose of gene therapy safely slows or stabilizes CLN6 Batten disease over years, pointing toward a long-term treatment option.
What could go wrong
This is a very small, early-stage follow-up study with only 10 participants, so results may not apply to everyone. The gene therapy was given years ago, and long-term safety or effectiveness is still uncertain.
Disclaimer
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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.
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 Hosptial
Columbus, Ohio, 43205, United States