Overview

Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset OTC Deficiency

Status:
Active, not recruiting
Trial end date:
2021-12-01
Target enrollment:
Participant gender:
Summary
A Phase 1/2, open-label dose-finding safety study of single ascending doses of DTX301 in adults with late-onset OTC Deficiency
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
Ultragenyx Pharmaceutical Inc
Treatments:
Prednisolone
Prednisone