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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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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: 28 Nov 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

  • 04 Nov 2025 According to an Ultragenyx Pharmaceutical Media Release, rolling submission of the BLA is expected to complete in the fourth quarter of 2025.
  • 08 Sep 2025 According to an Ultragenyx Pharmaceutical Media Release, Dr. John Mitchell, scientist in the Child Health and Human Development Program at the Research Institute of the McGill University Health Centre, pediatric endocrinologist at the Montreal Children's Hospital, is an investigator on the study
  • 08 Sep 2025 According to an Ultragenyx Pharmaceutical Media Release, announced positive longer-term results from phase 3 study of DTX401 AAV gene therapy for the treatment of glycogen storage disease type Ia (GSDIa)

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