Orphan Europe Carbaglu® Surveillance Protocol
Recruiting
Observational Study
N-acetylglutamate Synthase (NAGS) Deficiency
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Sex
- Any
- Study type
- Observational
- Participants needed
- 30 (estimated)
- Sponsor
- Nicholas Ah Mew · Other
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About This Trial
The purpose of this study is to conduct post-marketing surveillance of carglumic acid (Carbaglu) to obtain long-term clinical safety information. Carglumic acid was approved by the United States Food and Drug Administration (FDA) for treatment of acute hyperammonemia due to N-acetylglutamate synthase (NAGS) deficiency. Much of the FDA-required data is already collected through the Longitudinal Stu…
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* Confirmed diagnosis of NAGS deficiency or suspicion of NAGS deficiency
* Carbaglu intake for the treatment of NAGS
* Enrolled in the Longitudinal Study of Urea Cycle Disorders (RDCRN protocol #5101)
Exclusion Criteria:
* Cases of hyperammonemia caused by other urea cycle dis…
Contacts