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Recruiting
NCT06636383
Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
Conditions: Glycogen Storage Disease Type Ia
Sex: All
Ages: 2 Years – N/A
Healthy volunteers: No
Enrollment: 140
Sponsor: Ultragenyx Pharmaceutical Inc
Location: Children's Hospital of Orange County Orange California
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.
Eligibility Criteria
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
Source: ClinicalTrials.gov (NCT06636383). StuddyBuddy aggregates publicly available trial information.