Gene therapy trial for rare nerve disease halted early
NCT ID NCT05394064
First seen Oct 31, 2025 · Last updated May 22, 2026 · Updated 28 times
Summary
This study tested a one-time gene therapy (SBT101) given into the spinal fluid to treat adrenomyeloneuropathy (AMN), a rare inherited nerve disease that causes walking problems. The trial enrolled 8 adults aged 18-65 and was terminated early. The goal was to check safety and whether the treatment could slow disease progression.
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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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Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
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Genom att skicka in godkänner du våra Användarvillkor
Locations
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Amsterdam UMC
Amsterdam, Netherlands
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University of Massachusetts Chan Medical School
Worcester, Massachusetts, 01655, United States
Conditions
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