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Orphan Europe Carbaglu® Surveillance Protocol

Recruitingn/aAges any - anyAllNicholas Ah Mew
NCT03409003 on ClinicalTrials.gov ↗
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In plain words

What the trial is, and whether it could suit you

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.

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📋 Eligibility rules (as published)
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 disorders * Organic acidemia, lysinuric protein intolerance * Mitochondrial disorders * Congenital lactic acidemia, * Fatty acid oxidation defects * Primary liver disease will be excluded * Individuals with extreme low birth weight (\<1,500 grams) will be also excluded.
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What it tests

  • Not listed
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Where

3 sites
Children's National Medical Center
Washington D.C., United States
Children's Hospital Boston (UCDC New England Center)
Boston, United States
Icahn School of Medicine at Mount Sinai
New York, United States
🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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