At a glance
ClinicalIndex Comparison RecordPhase 2Completed· 12 enrolled
Drug / intervention
DTX401 +1 moregenetic
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 Phase 1/2, Open-Label Safety and Dose-Finding Study 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
A Phase 2 clinical trial evaluating DTX401 and steroid regimen for GSD1. Completed, enrolled 12 participants across 6 sites in 4 countries.
Detailed Summary
The primary objective of the study is to determine the safety of single doses of DTX401, including the incidence of dose-limiting toxicities (DLTs) at each dose level.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsGSD1
CountriesCanada, Netherlands, Spain, United States
Collaborators--
Timeline
Phase 2CompletedFinished
20192020202120222023202420252026
First PostedMay 2018
Enrollment StartMay 2018
Primary CompletionNov 2021
TodayJul 2026
First PostedMay 7, 2018
Enrollment StartMay 18, 2018
Primary CompletionNov 2, 2021
TodayJul 2, 2026
Enrollment to primary: 3.5 yearsPosted 8.2 years ago
Interventions
DTX401genetic
DTX401 administered as a single peripheral intravenous (IV) infusion
steroid regimendrug
prednisone or prednisolone to manage alanine aminotransferase (ALT) elevation