Canavan disease
MONDO:0010079A neurodegenerative disorder; its spectrum varies between severe forms with leukodystrophy, macrocephaly and severe developmental delay, and a very rare mild/juvenile form characterized by mild developmental delay.
Also known as: ACY2 deficiency, Canavan disease, Canavan-VAN Bogaert-Bertrand disease, aminoacylase 2 deficiency, aspartoacylase deficiency, spongy degeneration of central nervous system, spongy degeneration of the brain, Acy2 deficiency
6 clinical trials for this condition and its sub-types, 6 tagged with Canavan disease itself.
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Browse by category →Sub-types of Canavan disease
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Mild Canavan disease 0 trials
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Severe Canavan disease 0 trials
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Experimental gene therapy hopes to slow rare brain disease in toddlers
Disease control Recruiting nowThis 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 think…
Phase 1/2 • Sponsor: Aspa Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 12:09 UTC
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Massive leukodystrophy biobank aims to unlock disease secrets
Knowledge-focused Recruiting nowThis study collects medical information and biological samples (like blood or tissue) from up to 12,000 people with leukodystrophies—rare disorders that damage the brain's white matter. Researchers will use this data to find new genetic causes, develop biomarkers for future trial…
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:55 UTC