Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
Conditions:
Glycogen Storage Disease Type Ia
Sponsor: Ultragenyx Pharmaceutical Inc
trial.available_in:
БГ
Overview
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.
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.
Who can participate
Inclusion Criteria:
* Patient who had:
* DTX401 (full or partial dose) administered in a parent clinical study (Group 1) or
* Prescribed DTX401(full or partial dose) administered in a post-marketing setting (Group 2)
* Patient is willing and able to provide informed consent after the nature of the study has been explained, and prior to any research-related assessments or procedures. If a minor or an adult with cognitive limitations, the patient is willing and able (if possible) to provide assent and have a legally authorized representative provide informed consent after the nature of the study has been explained, and prior to any research-related assessments or procedures.
Exclusion Criteria:
* Presence of any condition that would interfere with study participation, interpretation of results or affect patient's safety in the opinion of the Investigator
Interventions
No Intervention
OTHER
Locations
19
Brazil (1)
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
Porto Alegre , Rio Grande do Sul
Canada (1)
McGill University Health Center
Montreal , Quebec
Denmark (1)
Rigshospitalet
Copenhagen
Germany (1)
Universitätsklinikum Hamburg Eppendorf
Hamburg
Italy (2)
Istituto G Gaslini Ospedale Pediatrico IRCCS
Genoa
University of Naples
Naples , Campania
Japan (2)
Osaka City General Hospital
Osaka
Fujita Health University Hospital
Toyoake
Netherlands (1)
Groningen University
Groningen
Spain (1)
Hospital Clínico Universitario de Santiago
Santiago
United States (9)
University of Michigan
Ann Arbor , Michigan
The Cleveland Clinic Foundation
Cleveland , Ohio
NOT_YET_RECRUITING
Children's Hospital Colorado
Denver , Colorado
Duke University Medical Center
Durham , North Carolina
University of Connecticut Health Center
Hartford , Connecticut
University of Texas Health Science Center at Houston