Gene therapy aims to halt fatal brain disease in children
NCT ID NCT03725670
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This trial tests a gene therapy for metachromatic leukodystrophy (MLD), a rare and life-threatening genetic disorder that damages the nervous system. The treatment uses a lentivirus to deliver a working copy of the ARSA gene directly into the spinal fluid and bloodstream. Up to 10 participants, including infants as young as 1 month, will be monitored for safety and any changes in disease progression.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- lentiviral TYF-ARSA gene therapy
- What this could lead to
- If successful, this could slow or stop the progression of MLD, a severe genetic disorder, by correcting the underlying enzyme deficiency.
- What could go wrong
- This is an early-phase trial with only 10 participants, so results may not apply broadly. Gene therapy carries risks like immune reactions or off-target effects.
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
-
Lung-Ji Chang
RECRUITINGShenzhen, Guangdong, 518000, China
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