NCT03517085

Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)

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)

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: Ultragenyx Pharmaceutical Inc · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Detailed description

Participants enrolled in the 401GSDIA01 study will be monitored for 52 weeks following DTX401 administration. Participants in Cohorts 1, 2, and 3 will receive reactive oral steroid treatment for possible vector-induced hepatitis following treatment with DTX401. Participants in Cohort 4 will receive prophylactic oral steroid treatment to prevent possible vector-induced hepatitis. After completion of the Week 52 visit or early withdrawal, participants will be offered enrollment into a 4-year extension study.

Conditions

• GSD1

Interventions

Type	Name	Description
GENETIC	DTX401	DTX401 administered as a single peripheral intravenous (IV) infusion
DRUG	steroid regimen	prednisone or prednisolone to manage alanine aminotransferase (ALT) elevation

Timeline

• Start date: 2018-05-18
• Primary completion: 2021-11-02
• Completion: 2021-11-02
• First posted: 2018-05-07
• Last updated: 2022-11-18
• Results posted: 2022-11-18

Locations

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

Regulatory

• FDA-regulated drug study