RecruitingNCT06636383
Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
GSDIa Disease Monitoring Program
Sponsor
Ultragenyx Pharmaceutical Inc
Enrollment
140 participants
Start Date
Nov 4, 2024
Study Type
OBSERVATIONAL
Conditions
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
Min Age: 2 Years
Inclusion Criteria4
- 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 Criteria1
- Presence of any condition that would interfere with study participation, interpretation of results or affect patient's safety in the opinion of the Investigator
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Interventions
OTHERNo Intervention
No investigational/study product will be administered in this DMP.
Locations(19)
View Full Details on ClinicalTrials.gov
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NCT06636383