DTX401

GENETIC2 trials

Sponsors

Ultragenyx Pharmaceutical Inc

Conditions

GSD1Glycogen Storage Disease Type IA

Phase 1

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

CompletedNCT03517085

Ultragenyx Pharmaceutical IncGSD1

Start: 2018-05-18End: 2021-11-02Updated: 2022-11-18

Phase 3

A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)

CompletedNCT05139316

Ultragenyx Pharmaceutical IncGlycogen Storage Disease Type IA

Start: 2021-11-08End: 2026-02-20Updated: 2026-03-25

Related Papers

Trial Interviews to Explore Glycogen Storage Disease Type Ia Patient Experiences Following Gene Therapy

Journal of health economics and outcomes research2026-02-12

Trial Interviews to Explore Glycogen Storage Disease Type Ia Patient Experiences Following Gene Therapy

Journal of health economics and outcomes research2026-02-12

Safety and Efficacy of <scp>DTX401</scp> , an <scp>AAV8</scp> ‐Mediated Liver‐Directed Gene Therapy, in Adults With Glycogen Storage Disease Type I a ( <scp>GSDIa</scp> )

Journal of Inherited Metabolic Disease2025-03-0112 citations

Gene therapy and genome editing for type I glycogen storage diseases.

2023-03-317 citations

A Prospective Study on Continuous Glucose Monitoring in Glycogen Storage Disease Type Ia: Toward Glycemic Targets

The Journal of Clinical Endocrinology & Metabolism2022-07-0425 citations

Gene therapy using a novel G6PC-S298C variant enhances the long-term efficacy for treating glycogen storage disease type Ia.

2020-05-1611 citations

An evolutionary approach to optimizing glucose-6-phosphatase-α enzymatic activity for gene therapy of glycogen storage disease type Ia.

2019-02-2210 citations