Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia
NCT03970278 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 12
Last updated 2026-03-18
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.
Conditions
- Glycogen Storage Disease Type IA
- Von Gierke's Disease (GSD Type Ia)
Interventions
- OTHER
-
No intervention
No intervention
Sponsors & Collaborators
- lead INDUSTRY
Principal Investigators
-
Medical Director · Ultragenyx Pharmaceutical Inc
Eligibility
- Min Age
- 18 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2019-07-15
- Primary Completion
- 2025-02-25
- Completion
- 2025-02-25
- FDA Drug
- Yes
Countries
- United States
- Canada
- Netherlands
- Spain
Study Locations
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