Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

Launched by ULTRAGENYX PHARMACEUTICAL INC · Oct 9, 2024

Keywords

ClinConnect Summary

The Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program is a clinical trial aimed at understanding the long-term safety and effectiveness of a treatment called DTX401. This study is observational, meaning researchers will monitor participants over at least 10 years after they receive DTX401 to see how well it works and if there are any side effects.

To be eligible for this study, participants must have received DTX401 as part of a previous clinical trial or through a doctor’s prescription. They should also be able to give their consent to participate. This trial is open to individuals aged 730 days and older, regardless of gender. Participants can expect to be followed closely by the research team to gather important information about their health and the treatment's impact over time. If you or someone you know has GSDIa and has received DTX401, this study may be a valuable opportunity to contribute to important research.

Gender

ALL

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