Actively Recruiting
Orphan Europe Carbaglu® Surveillance Protocol
Led by Nicholas Ah Mew · Updated on 2024-02-07
30
Participants Needed
3
Research Sites
769 weeks
Total Duration
On this page
Sponsors
N
Nicholas Ah Mew
Lead Sponsor
B
Boston Children's Hospital
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Orphan Europe Carbaglu® Surveillance Protocol
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 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)
You will not qualify if you...
- 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
- Individuals with extreme low birth weight (<1,500 grams)
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 3 locations
1
Children's National Medical Center
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
2
Children's Hospital Boston (UCDC New England Center)
Boston, Massachusetts, United States, 02115
Actively Recruiting
3
Icahn School of Medicine at Mount Sinai
New York, New York, United States, 10029
Actively Recruiting
Research Team
J
Jennifer Seminara, MPH
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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