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

Phases

Phase 1Phase 2

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2023-504004-29-00

Acronym

401GSDIA02

Enrollment

Registered

2024-02-07

Start date

2021-10-18

Completion date

2024-11-25

Last updated

2025-01-06

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Glycogen storage disease type Ia

Brief summary

The incidence of AEs and SAEs for each cohort and overall assessed by severity and relationship to IP

Detailed description

The change from Day 0 (Study 401GSDIA01) in time to first hypoglycemic event during a controlled fasting challenge over time by cohort and overall, following IV administration of DTX401

Interventions

DRUGPariglasgene brecaparvovec

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
The incidence of AEs and SAEs for each cohort and overall assessed by severity and relationship to IP	—

Secondary

Measure	Time frame
The change from Day 0 (Study 401GSDIA01) in time to first hypoglycemic event during a controlled fasting challenge over time by cohort and overall, following IV administration of DTX401	—

Countries

Netherlands, Spain

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026