New gene therapy trial hopes to tame rare metabolic disease in kids
NCT ID NCT06217861
First seen May 16, 2026 · Last updated May 24, 2026 · Updated 2 times
Summary
This early-stage trial tests a one-time gene therapy (VGM-R02b) for children up to 6 years old with Glutaric Acidemia Type I, a rare genetic disorder that can cause brain damage. The study aims to see if the treatment is safe and can reduce symptoms like movement problems and seizures. Only 12 children will take part, and they will be closely monitored for side effects.
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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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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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The Children's Hospital Zhejiang University Shcool of Medicine
RECRUITINGHangzhou, Zhejiang, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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