Trial Profile

A Phase 1/2, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Glucose-6- Phosphatase (G6Pase) in Adults With Glycogen Storage Disease Type Ia (GSDIa)

Status: Completed

Phase of Trial: Phase I/II

Latest Information Update: 28 Mar 2025

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At a glance

01 Mar 2025 Results assessing safety and efficacy of a single DTX401 infusion in 12 adults with Glycogen Storage Disease Type I a, published in the Journal of Inherited Metabolic Disease.
08 May 2023 According to Ultragenyx Pharmaceutical media release, data from this study will be presented at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting
19 May 2022 According to Ultragenyx Pharmaceutical media release, data from this study were presented this week at the American Society of Gene & Cell Therapy (ASGCT) 25th Annual Meeting.

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