Brain gene therapy shows promise for rare childhood disease

NCT ID NCT01801709

Completed Disease control Sponsor: Institut National de la Santé Et de la Recherche Médicale, France Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

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 injections of a harmless virus carrying a working copy of the ARSA gene directly into their brains. The goal was to see if the treatment is safe and 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: gene therapy (AAVrh.10cuARSA)
What this could lead to: If successful, this could slow or stop the progression of metachromatic leukodystrophy in young children, potentially preserving motor and cognitive function.
What could go wrong: This is a very early, small trial (only 5 children) with no control group. The treatment involves direct brain injections, which carry risks like infection or brain injury. It may not work or could have unknown side effects.

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Conditions

The condition(s) this trial relates to.

metachromatic leukodystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

METACHROMATIC LEUKODYSTROPHY

Contacts and locations

Locations

Bicêtre Hospital - Paris Sud

Le Kremlin-Bicêtre, France

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