drugset / Trial / NCT07479953
Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis Clinical Trial
NaSingle-groupOpen-labelTreatment
Summary
This is a study for the administration of in utero AAV9 transfer in prenatally diagnosed Type I or Type II GM1.
Timeline
- Start
- 2026-06-01
- Primary completion
- 2038-06-01
- Completion
- 2055-06-01
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | AAV9 Vector Expressing Human ß-galactosidase | Gene therapy (AAV / viral vector) | — | Intravenous |