Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
Start Date
11/4/2024
Completion Date
12/1/2036
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.
Detailed Description
The DTX401-CL401 Disease Monitoring Program (DMP) is a prospective, multicenter, long-term observational study to follow up participants with GSDIa for at least 10 years after the administration of DTX401.
Eligibility Criteria
Age Range: 2 years to No maximum
Interventions
No Intervention
Conditions
Locations
Children's Hospital of Orange County
Orange, California 92868
United States
Children's Hospital Colorado
Denver, Colorado 80045
United States
University of Connecticut Health Center
Hartford, Connecticut 06106
United States
University of Michigan
Ann Arbor, Michigan 48109
United States
Duke University Medical Center
Durham, North Carolina 27710
United States
The Cleveland Clinic Foundation
Cleveland, Ohio 44195
United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United States
University of Texas Health Science Center at Houston
Houston, Texas 77030
United States
Primary Children's Hospital
Salt Lake City, Utah 84108
United States
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
Porto Alegre, Rio Grande do Sul 90035-903
Brazil
McGill University Health Center
Montreal, Quebec H4A3J1
Canada
Rigshospitalet
Copenhagen, 2700
Denmark
Universitätsklinikum Hamburg Eppendorf
Hamburg, 20246
Germany
University of Naples
Naples, Campania 80131
Italy
Istituto G Gaslini Ospedale Pediatrico IRCCS
Genoa, 16147
Italy
Osaka City General Hospital
Osaka, 534-0021
Japan
Fujita Health University Hospital
Toyoake, 470-1192
Japan
Groningen University
Groningen, Provincie Groningen 9700 RB
Netherlands
Hospital Clínico Universitario de Santiago
Santiago, 15706
Spain