New gene therapy trial hopes to tame rare metabolic disease in kids
NCT ID NCT06217861
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage trial tests a one-time gene therapy called VGM-R02b in up to 12 children aged 6 years or younger 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 improve symptoms like movement problems and seizures. Participants will be monitored closely for side effects and changes in their condition.
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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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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: •••••@•••••