Experimental gene therapy offers hope for rare brain disease in toddlers
NCT ID NCT04998396
First seen Apr 25, 2026 · Last updated Apr 25, 2026
Summary
This study tests a new gene therapy called BBP-812 for children with Canavan disease, a rare genetic disorder that damages the brain. The treatment uses a harmless virus to deliver a working copy of the missing gene. The trial aims to see if it is safe and can improve motor and thinking skills in up to 26 children under 30 months old.
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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: •••••@•••••
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Contact
Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITINGChicago, Illinois, 60611, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Massachusetts General Hospital (MGH); Center for Rare Neurological Diseases (CRND)
RECRUITINGBoston, Massachusetts, 02114, United States
Contact Email: •••••@•••••
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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UCSF Benioff Children's Hospital Oakland
RECRUITINGOakland, California, 94609, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Weill Cornell Medicine; Division of Pediatric Neurology
COMPLETEDNew York, New York, 10065, United States
Conditions
Explore the condition pages connected to this study.