Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
Recruiting
Observational Study
Glycogen Storage Disease Type Ia
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
Ready to participate?
Review the details below, then apply to join this clinical trial.
At a Glance
Age
2 and older
Sex
Any
Study type
Observational
Participants needed
140 (estimated)
Sponsor
Ultragenyx Pharmaceutical Inc · Industry
Who this trial is looking for
This trial is looking for patients who have received DTX401 and want to help understand its long-term safety and effectiveness. Participation involves providing consent to be part of the study and sharing health information for at least 10 years.
Are You a Good Fit for This Trial?
You may be able to join if
I have received DTX401 in a clinical study or post-marketing setting.
I am willing and able to provide informed consent.
If I am a minor or have cognitive limitations, I can assent and have a representative consent.
You may not be able to join if
I have a condition that could interfere with the study or my safety.
Summarized in plain language from this trial's official eligibility criteria.
The full criteria are further down this page — only the research team can
confirm whether you qualify.
Think this trial could be right for you?
Answer three quick questions written from this study's own rules to see if you may qualify. No account needed to find out.
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.
Trial Locations
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City
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Status
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…
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
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