One-Time IV treatment aims to halt fatal childhood brain disease
NCT ID NCT03952637
Summary
This study is testing a one-time intravenous (IV) gene therapy for children with GM1 gangliosidosis, a rare and fatal genetic disorder that destroys nerve cells. The therapy aims to deliver a working copy of a missing gene so the body can produce a vital enzyme, potentially slowing or stopping disease progression. The study will enroll up to 54 children, from infants to age 12, to assess the treatment's safety and see if it improves symptoms and development.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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National Institutes of Health Clinical Center
RECRUITINGBethesda, Maryland, 20892, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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