Orphan Europe Carbaglu® Surveillance Protocol
Part of paid clinical trials in Washington D.C., District of Columbia.
- Sponsor
- Nicholas Ah Mew
- Study ID
- NCT03409003
- Status
- Recruiting
Conditions
- N-acetylglutamate Synthase (NAGS) Deficiency
Eligibility Criteria
- Sex
- ALL
- Age
- N/A - N/A
- Healthy Volunteers
- Not accepted
Study Details
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 Dates
- Start date
- Apr 30, 2012
- Status verified
- Feb 2024
- Primary completion
- Jul 31, 2026
- Completion
- Jan 31, 2027
Study Design
- Enrollment
- 30 participants (estimated)
Primary Outcome Measure
Carbaglu related adverse events and adverse reactions [ Time Frame: 15 years ]
Central Contacts
- Jennifer Seminara, MPH2023066489
Locations (3)
| Facility | City | State | ZIP | Site coordinators |
|---|---|---|---|---|
| Children's National Medical Center | Washington D.C. | District of Columbia | 20010 | Nicholas Ah Mew, MD (PRINCIPAL_INVESTIGATOR) |
| Children's Hospital Boston (UCDC New England Center) | Boston | Massachusetts | 02115 | Gerry Berry, MD (PRINCIPAL_INVESTIGATOR) Harvey Levy, MD (SUB_INVESTIGATOR) |
| Icahn School of Medicine at Mount Sinai | New York | New York | 10029 | Margo Breilyn, MD (PRINCIPAL_INVESTIGATOR) |