Pariglasgene brecaparvovec

DRUG3 trials

Sponsors

Ultragenyx Pharmaceutical Inc.

Conditions

Glycogen Storage Disease Type Ia (GSDIa)Glycogen storage disease type IaGlycogen storage disease type Ia (GSDIa)

Phase 1

A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Glucose-6-Phosphatase (G6Pase) in Adults with Glycogen Storage Disease Type Ia (GSDIa)

CompletedCTIS2023-504004-29-00

Ultragenyx Pharmaceutical Inc.Glycogen storage disease type Ia

Start: 2021-10-18End: 2024-11-25Target: 3Updated: 2025-01-06

Phase 3

A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adenoassociated Virus Serotype 8-mediated Gene Transfer of Glucose-6-phosphatase in Patients With Glycogen Storage Disease Type Ia.

CompletedCTIS2023-508750-25-00

Ultragenyx Pharmaceutical Inc.Glycogen Storage Disease Type Ia (GSDIa)

Start: 2022-01-11End: 2025-12-22Target: 30Updated: 2026-01-19

GSDIa Disease Monitoring Program

RecruitingCTIS2023-510219-20-00

Ultragenyx Pharmaceutical Inc.Glycogen storage disease type Ia (GSDIa)

Start: 2024-11-04Target: 14Updated: 2025-08-21