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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: Completed
Phase of Trial: Phase III

Latest Information Update: 16 Mar 2026

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At a glance

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

Most Recent Events

  • 11 Mar 2026 Status changed from active, no longer recruiting to completed.
  • 23 Feb 2026 According to an Ultragenyx Pharmaceutical Media Release, the company announced the U.S. Food and Drug Administration (FDA or the Agency) has accepted for review the Biologics License Application (BLA) seeking approval of DTX401 AAV gene therapy for the treatment of Glycogen Storage Disease Type Ia. The FDA granted the BLA Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) action date of August 23, 2026.
  • 13 Feb 2026 According to an Ultragenyx Pharmaceutical Media Release, biologics License Application (BLA) rolling submission completed in December 2025, with an anticipated Prescription Drug User Fee Act (PDUFA) action date in the third quarter of 2026.

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