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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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
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Vanda Investigational Site
Madison, Wisconsin, 53792, United States
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