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: 25 Sep 2023
Price :
$35
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At a glance
- Drugs Pariglasgene-brecaparvovec (Primary)
- Indications Glycogen storage disease type I
- Focus Adverse reactions; First in man
- Sponsors Ultragenyx Pharmaceutical
- 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.
- 19 May 2022 Results published in the Ultragenyx Pharmaceutical Media Release