Experimental gene therapy hopes to slow rare brain disease in toddlers

NCT ID NCT04998396

Recruiting now Disease control Sponsor: Aspa Therapeutics Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a gene therapy called BBP-812 in children up to 30 months old with Canavan disease, a rare genetic 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 and development over a year.

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Conditions

The condition(s) this trial relates to.

Canavan disease Leukoencephalopathies Rare Diseases

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

CANAVAN DISEASE

Contacts and locations

Locations

Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITING

Chicago, Illinois, 60611, United States
Massachusetts General Hospital (MGH); Center for Rare Neurological Diseases (CRND)
RECRUITING

Boston, Massachusetts, 02114, United States
UCSF Benioff Children's Hospital Oakland
RECRUITING

Oakland, California, 94609, United States
Weill Cornell Medicine; Division of Pediatric Neurology
COMPLETED

New York, New York, 10065, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.