drugset / Trial / NCT02991144
Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency
Non-randomizedParallel-groupOpen-labelTreatment
Summary
This is a Phase 1/2, open-label, single arm, multicenter, safety and dose finding study of DTX301 in adults with late-onset OTC deficiency. The primary objective of the study is to determine the safety of single intravenous (IV) doses of DTX301.
Timeline
- Start
- 2017-07-31
- Primary completion
- 2021-12-16
- Completion
- 2021-12-16
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | AVALOTCAGENE ONTAPARVOVEC | Gene therapy (AAV / viral vector) | 2e+12 vg/kg | Intravenous |
| Subject | AVALOTCAGENE ONTAPARVOVEC | Gene therapy (AAV / viral vector) | 6e+12 vg/kg | Intravenous |
| Subject | AVALOTCAGENE ONTAPARVOVEC | Gene therapy (AAV / viral vector) | 1e+13 vg/kg | Intravenous |
| Background | Prednisolone | Other / unclassified | 30 mg | Oral |
| Background | Prednisolone | Other / unclassified | 60 mg | Oral |
| Background | Prednisone | Other / unclassified | 30 mg | Oral |
| Background | Prednisone | Other / unclassified | 60 mg | Oral |