Experimental gene therapy hopes to slow rare brain disease in toddlers
NCT ID NCT04998396
First seen Apr 25, 2026 · Last updated May 19, 2026 · Updated 4 times
Summary
This study tests a gene therapy called BBP-812 in children up to 30 months old with Canavan disease, a rare and severe brain disorder. The treatment uses a harmless virus to deliver a working copy of the ASPA gene, aiming to reduce harmful brain chemicals and improve motor and thinking skills. The main goals are to check safety and measure changes in brain chemistry over one year.
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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.