Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
ConditionGlycogen Storage Disease Type Ia
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age2+
SponsorUltragenyx Pharmaceutical Inc
About this trial
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
Qualifiers
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.
Disqualifiers
Presence of any condition that would interfere with study participation, interpretation of results or affect patient's safety in the opinion of the Investigator
Trial design
Treatments tested in this trial
- No Intervention
Treatment groups
140 Participants
are divided into 2 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov