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Clinical Trial Summary

The main objective of this trial is to evaluate the safety, tolerability, and pharmacodynamic activity of BBP-812, an investigational AAV9-based gene therapy, in pediatric participants with Canavan disease.


Clinical Trial Description

Canavan disease is an ultra-rare, profoundly disabling and fatal disease with no approved therapy. The Sponsor is developing BBP-812, an investigational gene therapy product for systemic delivery in participants with Canavan disease. BBP-812 is a recombinant adeno-associated virus serotype 9 (rAAV9) vector engineered to deliver the aspartoacylase (ASPA) transgene under control of a ubiquitous promoter to restore ASPA expression in both neuronal and non-neuronal cell types. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT04998396
Study type Interventional
Source Aspa Therapeutics
Contact Michelle Nelken
Phone 833-764-2267 or 617-861-4617
Email CANaspire@aspatx.com
Status Recruiting
Phase Phase 1/Phase 2
Start date September 8, 2021
Completion date October 8, 2030

See also
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