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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

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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

  • Drugs Pariglasgene-brecaparvovec (Primary)
  • Indications Glycogen storage disease type I
  • Focus Registrational; Therapeutic Use
  • Acronyms GlucoGene
  • Sponsors Ultragenyx Pharmaceutical

    • Most Recent Events

    • 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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