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.
Disclaimer
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the original study
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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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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
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Shenzhen Geno-immune Medical Institute
RECRUITINGShenzhen, Guangdong, 518000, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
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