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Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
RecruitingObservational study
GSDIa Disease Monitoring Program
Who can join
Ages 2 and older · All sexes
Full 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
About the study
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.
What is being tested
- No Intervention (other)
Sponsor: Ultragenyx Pharmaceutical Inc · Participants: 140 · Started: Nov 4, 2024
Contact the study team
- Patients Contact: Trial Recruitment · Phone: 1-888-756-8657
- HCPs Contact: Medical Information · Phone: 1-888-756-8657
Official record on ClinicalTrials.gov — NCT06636383
Locations in the U.S.
| California | Children's Hospital of Orange County, Orange |
| Colorado | Children's Hospital Colorado, Denver |
| Connecticut | University of Connecticut Health Center, Hartford |
| Michigan | University of Michigan, Ann Arbor |
| North Carolina | Duke University Medical Center, Durham |
| Ohio | The Cleveland Clinic Foundation, Cleveland (Not yet recruiting) |
| Pennsylvania | Children's Hospital of Philadelphia, Philadelphia |
| Texas | University of Texas Health Science Center at Houston, Houston |
| Utah | Primary Children's Hospital, Salt Lake City (Not yet recruiting) |
From ClinicalTrials.gov, data retrieved Oct 2, 2026. Each study sets its own eligibility; the study team decides who can join.