Trial Profile

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia

Status: Active, no longer recruiting

Phase of Trial: Phase III

Latest Information Update: 26 Feb 2025

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At a glance

28 Jan 2025 Status changed from recruiting to active, no longer recruiting.
12 Jan 2025 According to an Ultragenyx Pharmaceutical Media Release, These results have been discussed with regulatory authorities in a pre-BLA meeting and will be included as part of a BLA submission in mid-2025.
05 Nov 2024 According to an Ultragenyx Pharmaceutical Media Release, results from this trial will be discussed with regulatory authorities to support a BLA submission in mid-2025.

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