A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)
Recruiting now · Phase 1/Phase 2
Conditions studied: Canavan Disease
In brief
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.
Key facts
- Study ID
- NCT04998396
- Run by
- Aspa Therapeutics
- People needed
- 26
- Starts
- 2021-09-08
- Expected to finish
- 2032-10-08
- Last updated by the study team
- 2026-04-17
Who can join
Age: any, up to 3. Sex: any. Healthy volunteers: not accepted.
Where it is running
- UCSF Benioff Children's Hospital Oakland — Oakland, California, United States (enrolling)
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States (enrolling)
- Massachusetts General Hospital (MGH); Center for Rare Neurological Diseases (CRND) — Boston, Massachusetts, United States (enrolling)
- Weill Cornell Medicine; Division of Pediatric Neurology — New York, New York, United States
Full record on ClinicalTrials.gov
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