Brain gene injection aims to halt rare childhood disease
NCT ID NCT01801709
First seen Nov 18, 2025 · Last updated Jun 23, 2026 · Updated 25 times
Summary
This study tested a gene therapy for children with early-onset metachromatic leukodystrophy (MLD), a rare and severe brain disease. Five children aged 6 months to 5 years received direct injections of a harmless virus carrying a working gene into their brains. The goal was to see if it is safe and can slow disease progression.
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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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Contacts and locations
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Locations
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Bicêtre Hospital - Paris Sud
Le Kremlin-Bicêtre, France
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
AAVrh.10cuARSA gene therapy
What this could lead to
If it works, this could slow or stop the progression of metachromatic leukodystrophy in young children, potentially preserving brain function.
What could go wrong
This is a very early, small trial (only 5 children) testing safety first. The treatment involves direct brain injections, which carries risks like bleeding or infection. It may not stop the disease.
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.