Experimental gene therapy aims to halt fatal brain disease in children
NCT ID NCT03727555
First seen Jun 24, 2026 · Last updated Jun 26, 2026 · Updated 1 time
Summary
This trial tests a gene therapy for X-linked adrenoleukodystrophy (X-ALD), a rare genetic disease that damages the brain. The therapy uses a modified virus to deliver a working copy of the faulty gene directly into the spinal fluid and bloodstream. Up to 30 patients aged 1 year and older will be enrolled to see if the treatment is safe and can slow 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 gene therapy carrying a corrected ABCD1 gene
- What this could lead to
- If successful, this could slow or halt the progression of X-ALD, a devastating brain disease, and improve quality of life for patients.
- What could go wrong
- This is an early Phase I/II trial with only 30 participants, so safety and effectiveness are not yet proven. Gene therapy carries risks like immune reactions or unintended genetic changes.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for X-LINKED ADRENOLEUKODYSTROPHY are added.
By submitting, you agree to our Terms of use
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
-
Shenzhen Geno-immune Medical Institute
RECRUITINGShenzhen, Guangdong, 518000, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.