At a glance
ClinicalIndex Comparison RecordN/ACompleted· 12 enrolled
Drug / intervention
No interventionother
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
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)
In Brief
An observational study evaluating No intervention for Glycogen Storage Disease Type IA and Von Gierke's Disease (GSD Type Ia). Completed, enrolled 12 participants across 6 sites in 4 countries.
Detailed 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.
Study Details
Study Typeobservational
Allocation--
Masking--
Primary Purpose--
CountriesCanada, Netherlands, Spain, United States
Collaborators--
Timeline
N/ACompletedFinished
2020202120222023202420252026
First PostedMay 2019
Enrollment StartJul 2019
Primary CompletionFeb 2025
TodayJul 2026
First PostedMay 31, 2019
Enrollment StartJul 15, 2019
Primary CompletionFeb 25, 2025
TodayJul 2, 2026
Enrollment to primary: 5.6 yearsPosted 7.1 years ago
Interventions
No interventionother
No intervention