New gene therapy hopes to tame rare metabolic disease in kids
NCT ID NCT06217861
First seen May 16, 2026 · Last updated May 16, 2026
Summary
This early-stage study tests a single-dose gene therapy called VGM-R02b in up to 12 children aged 6 or younger with glutaric acidemia type I, a rare genetic disorder that can cause brain damage. The treatment aims to replace the faulty gene and improve symptoms like movement problems and seizures. The main goals are to check safety and see if the therapy helps control the disease.
Disclaimer
Read more
Show less
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.
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for GLUTARIC ACIDEMIA TYPE I are added.
By submitting, you agree to our Terms of use
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
The Children's Hospital Zhejiang University Shcool of Medicine
RECRUITINGHangzhou, Zhejiang, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
Explore the condition pages connected to this study.