NCT03970278

Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia

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)

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 12 (actual)
• Sponsor: Ultragenyx Pharmaceutical Inc · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of this study is to determine the long-term safety of DTX401 following a single intravenous (IV) dose in adults with GSDIa.

Detailed description

Only participants who received DTX401 in study 401GSDIA01 (NCT03517085) are eligible to participate in study 401GSDIA02. No investigational product will be administered during study 401GSDIA02. Participants will be followed in study 401GSDIA02 for at least 4 years, and up to 6 years after administration of DTX401.

Conditions

• Glycogen Storage Disease Type IA
• Von Gierke's Disease (GSD Type Ia)

Interventions

Type	Name	Description
OTHER	No intervention	No intervention

Timeline

• Start date: 2019-07-15
• Primary completion: 2025-02-25
• Completion: 2025-02-25
• First posted: 2019-05-31
• Last updated: 2026-03-18
• Results posted: 2026-03-18

Locations

6 sites across 4 countries: United States, Canada, Netherlands, Spain

Regulatory

• FDA-regulated drug study