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Orphan Europe Carbaglu® Surveillance Protocol
Sponsor: Nicholas Ah Mew
Summary
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 Study of Urea Cycle Disorders (RDCRN Protocol #5101). This study will collect additional data on adverse events (interim events), adverse reactions, pregnancy, and fetal outcomes.
Key Details
Gender
All
Age Range
Any - Any
Study Type
OBSERVATIONAL
Enrollment
30
Start Date
2012-04
Completion Date
2027-01
Last Updated
2024-02-07
Healthy Volunteers
No
Locations (3)
Children's National Medical Center
Washington D.C., District of Columbia, United States
Children's Hospital Boston (UCDC New England Center)
Boston, Massachusetts, United States
Icahn School of Medicine at Mount Sinai
New York, New York, United States