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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

Interested in this study? View the official listing for contact and enrollment details.

View on ClinicalTrials.gov

Source: ClinicalTrials.gov (NCT06636383). StuddyBuddy aggregates publicly available trial information.