drugset / Trial / NCT07530796
Safety and Efficacy of scAAV9/AGA Gene Therapy in Participants With Aspartylglucosaminuria (AGU)
NaSequentialOpen-labelTreatment
Summary
The goal of this clinical trial is to learn if the treatment is a safe, tolerable, and efficacious treatment for adults and children with Aspartylglucosaminuria (AGU).
Timeline
- Start
- 2026-05-01
- Primary completion
- 2029-05-01
- Completion
- 2032-05-01
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Danagalex | Gene therapy (AAV / viral vector) | — | Intrathecal |