Clinical trials: GSD1
GSD1
Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)
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.
- Trial name
- NCT03517085
- Testing
- DTX401, steroid regimen
- Where
- 6 sites in United States, Canada and 2 more
- Volunteers
- 12 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Inflammatory Bowel Disease, Glycogen Storage Disease Type Ia
Study of the Relationship Between Glycogen Storage Disease Type Ia and Inflammatory Bowel Disease
The purpose of this research study is to understand the relationship between inflammatory bowel disease (IBD) and Glycogen storage disease (GSD)type Ia.
- Trial name
- NCT01854242
- Testing
- Glycogen Storage Disease type Ia patients
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- University of Florida
Glycogen Storage Disease Type IA
A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)
The primary objectives of this study are to evaluate the efficacy of DTX401 to reduce or eliminate dependence on exogenous glucose replacement therapy to maintain euglycemia and to maintain or improve the quality of glucose…
- Trial name
- NCT05139316
- Testing
- DTX401, Oral prednisolone
- Where
- 20 sites in United States, Brazil and 7 more
- Volunteers
- 52 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Glycogen Storage Disease Type IA, Von Gierke's Disease (GSD Type Ia)
Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia
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.
- Trial name
- NCT03970278
- Testing
- No intervention
- Where
- 6 sites in United States, Canada and 2 more
- Volunteers
- 12 people
- Sponsor
- Ultragenyx Pharmaceutical Inc