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.
At a Glance
- Age
- 2 and older
- Sex
- Any
- Study type
- Observational
- Participants needed
- 140 (estimated)
- Sponsor
- Ultragenyx Pharmaceutical Inc · Industry
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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.
Trial Locations
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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…
Contacts