A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)

NCTID	NCT04998396 (View at clinicaltrials.gov)
Description	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. (Show More)
Development Status	Inactive
Indication	Canavan Disease
Disease Ontology Term	DOID:3613
Compound Name	BBP-812
Compound Description	AAV9-ASPA
Sponsor	Aspa Therapeutics
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	26
Results Posted	Not Available

Target Gene/Variant	ASPA
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV9
Editor Type	none
Dose 1	Undisclosed dose escalation, 2 levels
Dose 2	Undisclosed expansion dose
Dose 3
Dose 4
Dose 5

No.of Trial Sites	4
Locations	United States

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
Recent Updates	Program terminated, effective Feb 2025