Condition: N-acetylglutamate Synthase (NAGS) Deficiency · Sponsor: Nicholas Ah Mew
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.
This description comes directly from the study's public registry record.
Jennifer Seminara, MPH · 2023066489 · jseminar@childrensnational.org
Always discuss trial participation with your own doctor first.
| Children's National Medical Center | Washington D.C., District of Columbia, United States | Recruiting |
| Children's Hospital Boston (UCDC New England Center) | Boston, Massachusetts, United States | Recruiting |
| Icahn School of Medicine at Mount Sinai | New York, New York, United States | Recruiting |
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Source record: clinicaltrials.gov/study/NCT03409003