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.
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
-
Nationwide Children's Hosptial
Columbus, Ohio, 43205, United States
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