GSDIa Disease Monitoring Program
Ultragenyx Pharmaceutical Inc
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.
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.
Eligibility
- Age range
- 2+ years
- Sex
- All
- Healthy volunteers
- No
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…
Interventions
- OtherNo Intervention
No investigational/study product will be administered in this DMP.
Locations (19)
- Children's Hospital of Orange CountyOrange, California
- Children's Hospital ColoradoDenver, Colorado
- University of Connecticut Health CenterHartford, Connecticut
- University of MichiganAnn Arbor, Michigan
- Duke University Medical CenterDurham, North Carolina
- The Cleveland Clinic FoundationCleveland, Ohio