drugset / Trial / NCT02168686
Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency
Non-randomizedSequentialOpen-labelTreatment
Summary
The ADVANCE study is being conducted by Adverum Biotechnologies, Inc. as an open-label, multicenter, dose-escalation study in order to assess the safety and protein expression of ADVM-043 following a single intravenous or intrapleural administration.
Timeline
- Start
- 2017-11-28
- Primary completion
- 2019-08-29
- Completion
- 2019-08-29
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | ADVM-043 | Gene therapy (AAV / viral vector) | 8e+13 vg | Intravenous |
| Subject | ADVM-043 | Gene therapy (AAV / viral vector) | 4e+14 vg | Intravenous |
| Subject | ADVM-043 | Gene therapy (AAV / viral vector) | 1.2e+15 vg | Intravenous |