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.
N-acetylglutamate Synthase (NAGS) Deficiency
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.
Orphan Europe Carbaglu® Surveillance Protocol
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Children's National Medical Center, Washington, District of Columbia, United States, 20010
Children's Hospital Boston (UCDC New England Center), Boston, Massachusetts, United States, 02115
Icahn School of Medicine at Mount Sinai, New York, New York, United States, 10029
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
For general information about clinical research, read Learn About Studies.
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ALL
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Nicholas Ah Mew,
Nicholas Ah Mew, MD, PRINCIPAL_INVESTIGATOR, Children's National Health System
2027-01