A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)
Completed · Phase 3 · Has a placebo group
Conditions studied: Glycogen Storage Disease Type IA
In brief
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 control.
Key facts
- Study ID
- NCT05139316
- Run by
- Ultragenyx Pharmaceutical Inc
- People needed
- 49
- Starts
- 2021-11-08
- Expected to finish
- 2026-02-20
- Last updated by the study team
- 2026-03-25
Who can join
Age: 8 and older. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Children's Hospital of Orange County — Orange, California, United States
- Children's Hospital Colorado — Aurora, Colorado, United States
- University of Connecticut Health Center — Farmington, Connecticut, United States
- Mount Sinai — The Bronx, New York, United States
- Duke University — Durham, North Carolina, United States
- Cleveland Clinic — Cleveland, Ohio, United States
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
- University of Texas — Houston, Texas, United States
- Primary Children's Hospital — Salt Lake City, Utah, United States
- Hospital de Clinicas de Porto Alegre — Porto Alegre, Rio Grande do Sul, Brazil
- McGill University — Montreal, Quebec, Canada
- Righospitalet — Copenhagen, Capital, Denmark
- University Medical Center Eppendorf — Hamburg, Germany
- Istituto Giannina Gaslini — Genova, Linguria, Italy
- University of Naples — Naples, Italy
- Kumamoto University Hospital — Kumamoto, Japan
- Osaka City General Hospital — Osaka, Japan
- Fujita Health University Hospital — Toyoake, Japan
- Groningen University — Groningen, Netherlands
- Hospital Clinico Universitario de Santiago — Santiago de Compostela, A Coruna, Spain
Full record on ClinicalTrials.gov
Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.