Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia

Glycogen Storage Disease Type IA Clinical Trial

Official title:
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 Active, not recruiting

Clinical Trial 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.

Clinical Trial Description

Only participants who received DTX401 in study 401GSDIA01 (NCT03517085) are eligible to participate in study 401GSDIA02. No investigational product will be administered during study 401GSDIA02. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT03970278
Study type	Observational
Source	Ultragenyx Pharmaceutical Inc
Contact
Status	Active, not recruiting
Phase
Start date	July 15, 2019
Completion date	December 2026

View Details

See also
Status	Clinical Trial	Phase
Active, not recruiting	`NCT05139316` - A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)	Phase 3
Completed	`NCT04708015` - Retrospective Study of Glucose Monitoring for Glycemic Control in Patients With GSDIa
Completed	`NCT01854242` - Study of the Relationship Between Glycogen Storage Disease Type Ia and Inflammatory Bowel Disease
Completed	`NCT02054832` - Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard Versus Modified Uncooked Cornstarch	N/A
Terminated	`NCT04909346` - Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease
Completed	`NCT04311307` - Endogenous Glucose Production in Patients With Glycogen Storage Disease Type Ia	Phase 1/Phase 2