First human test of gene injection into vagus nerve for fatal childhood disease
NCT ID NCT07543991
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 2 times
Summary
This early-stage trial tests a gene therapy called scAAV9/JeT-GAN for giant axonal neuropathy (GAN), a rare, fatal nerve disease in children. The therapy is injected directly into the left vagus nerve to target autonomic nervous system symptoms. Only 4 people who have already received the therapy via spinal injection will take part. The main goal is to check safety, with a secondary look at whether symptoms improve.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- scAAV9/JeT-GAN (a gene therapy)
- What this could lead to
- If it works, this could help control autonomic nervous system symptoms in giant axonal neuropathy, improving quality of life and possibly slowing disease progression.
- What could go wrong
- This is a very early, small trial (only 4 people) testing a new way of giving the drug. The safety and effectiveness are unknown, and side effects could be serious.
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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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Children's Health
RECRUITINGDallas, Texas, 75235, United States