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Clinical trial

A Long-Term Follow-up Study to Evaluate the Safety and Efficacy 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)

NCT03970278 is registered in ClinicalTrials.gov with Ultragenyx Pharmaceutical Inc as lead sponsor.

NCT NCT03970278
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Registry record

As filed in ClinicalTrials.gov.

Title
A Long-Term Follow-up Study to Evaluate the Safety and Efficacy 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)
Lead sponsor
Ultragenyx Pharmaceutical Inc
Phase
Observational
Overall status
Completed
Start date
July 15, 2019
Completion date
February 25, 2025
Enrollment
12 (actual)
Sites
6

Conditions

As the registry lists them; a condition with its own page is linked.

glycogen storage disease type ia, von gierke's disease (gsd type ia)