Gene therapy for giant axonal neuropathy via vagus nerve
Part of Brain & nervous system, Genetic & congenital clinical trials.
This is a follow-up study for patients who previously received an experimental gene therapy (scAAV9/JeT-GAN) for giant axonal neuropathy (GAN), a rare genetic nerve disorder. The new treatment delivers the therapy directly into the vagus nerve—a major nerve in the neck—to help manage nerve damage and related symptoms.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have been diagnosed with GAN through genetic testing showing mutations in the GAN gene
- You must have already completed the first GAN gene therapy study and finished its 5-year follow-up period
- You must be able to safely receive anesthesia (sedation) during the nerve injection procedure
- You cannot be enrolled in another drug or device trial at the same time
- Your blood work (liver and kidney function, blood cell counts) must be within acceptable ranges
- You must be up to date on childhood vaccines and willing to get annual flu and COVID-19 vaccines
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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