Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)
Status:
Completed
Trial end date:
2021-11-02
Target enrollment:
Participant gender:
Summary
The primary objective of the study is to determine the safety of single doses of DTX401,
including the incidence of dose-limiting toxicities (DLTs) at each dose level.