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
Latest Information Update: 26 Feb 2025
At a glance
- Drugs Pariglasgene-brecaparvovec (Primary)
- Indications Glycogen storage disease type I
- Focus Registrational; Therapeutic Use
- Acronyms GlucoGene
- Sponsors Ultragenyx Pharmaceutical
- 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.