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Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
Sponsor: Ultragenyx Pharmaceutical Inc
Summary
The main objective of this observational study is to evaluate the long-term safety and effectiveness of DTX401 for at least 10 years after DTX401 administration.
Official title: GSDIa Disease Monitoring Program
Key Details
Gender
All
Age Range
2 Years - Any
Study Type
OBSERVATIONAL
Enrollment
140
Start Date
2024-11-04
Completion Date
2036-12
Last Updated
2026-03-03
Healthy Volunteers
No
Conditions
Interventions
No Intervention
No investigational/study product will be administered in this DMP.
Locations (19)
Children's Hospital of Orange County
Orange, California, United States
Children's Hospital Colorado
Denver, Colorado, United States
University of Connecticut Health Center
Hartford, Connecticut, United States
University of Michigan
Ann Arbor, Michigan, United States
Duke University Medical Center
Durham, North Carolina, United States
The Cleveland Clinic Foundation
Cleveland, Ohio, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
University of Texas Health Science Center at Houston
Houston, Texas, United States
Primary Children's Hospital
Salt Lake City, Utah, United States
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
Porto Alegre, Rio Grande do Sul, Brazil
McGill University Health Center
Montreal, Quebec, Canada
Rigshospitalet
Copenhagen, Denmark
Universitätsklinikum Hamburg Eppendorf
Hamburg, Germany
University of Naples
Naples, Campania, Italy
Istituto G Gaslini Ospedale Pediatrico IRCCS
Genoa, Italy
Osaka City General Hospital
Osaka, Japan
Fujita Health University Hospital
Toyoake, Japan
Groningen University
Groningen, Provincie Groningen, Netherlands
Hospital Clínico Universitario de Santiago
Santiago, Spain