drugset / Trial / NCT04998396
A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)
Non-randomizedSequentialOpen-labelTreatment
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.
Timeline
- Start
- 2021-09-08
- Primary completion
- 2026-10-13
- Completion
- 2032-10-08
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | BBP-812 | Gene therapy (AAV / viral vector) | — | Intravenous |