Halted gene therapy study raises questions for AMN patients
NCT ID NCT05394064
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-stage trial tested a gene therapy called SBT101 for adrenomyeloneuropathy (AMN), a rare nerve disease that causes walking difficulties. Eight adults received either the therapy or a sham procedure. The study was terminated early, so we have limited data on safety and effectiveness. Researchers were measuring side effects and changes in walking ability over two years.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
SBT101 gene therapy (AAV9-ABCD1)
What this could lead to
If successful, this could point toward a treatment that slows or stops the progression of adrenomyeloneuropathy, a rare and disabling nerve disease.
What could go wrong
The trial was terminated early with only 8 participants, so results are very limited. Gene therapies can have unexpected side effects, and it is unclear if SBT101 will work in humans.
Disclaimer
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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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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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Locations
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Amsterdam UMC
Amsterdam, Netherlands
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University of Massachusetts Chan Medical School
Worcester, Massachusetts, 01655, United States
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