One-Patient trial hopes to fix rare nerve disease at its genetic root
NCT ID NCT07223632
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a custom-made drug (VCA-894A) in a single person with a rare genetic nerve disease called CMT2S. The drug is designed to correct a specific genetic error and restore a missing protein. The main goals are to check if the treatment is safe and if it can improve muscle function.
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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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Locations
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Vanda Investigational Site
Madison, Wisconsin, 53792, United States