One-Time gene injection holds promise for rare childhood brain disease
NCT ID NCT04737460
First seen Dec 31, 2025 · Last updated Apr 24, 2026 · Updated 18 times
Summary
This early-stage trial tests a single injection of gene therapy (AAV9/CLN7) into the spinal fluid of children with CLN7 Batten disease, a rare and severe genetic disorder that causes progressive loss of motor and mental abilities. The main goal is to check safety, but researchers will also measure changes in movement, thinking, and brain scans. Only 4 children aged 1 to 18 with mild to moderate symptoms are enrolled.
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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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Locations
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Children' Health
Dallas, Texas, 75390, United States
Conditions
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