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NCT03409003 · ClinicalTrials.gov registry record
Orphan Europe Carbaglu® Surveillance Protocol
A clinical trial of N-acetylglutamate Synthase (NAGS) Deficiency, sponsored by Nicholas Ah Mew.
- Recruiting
- Registry status
- 30
- Enrollment target
- 3
- Study locations
NCT03409003 is a study of N-acetylglutamate Synthase (NAGS) Deficiency that is actively recruiting participants, run by Nicholas Ah Mew. The registered enrollment target is 30 participants. The trial reports 3 study locations across 3 states.
The verdict
NCT03409003, a study of N-acetylglutamate Synthase (NAGS) Deficiency, is actively recruiting participants, sponsored by Nicholas Ah Mew.
- RECRUITING
- Registry status
- 30 participants
- Enrollment target
- 3
- Study locations
Study Summary
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 Studied
Study Locations (3)
District of Columbia
- Children's National Medical Center - Washington D.C.
Massachusetts
- Children's Hospital Boston (UCDC New England Center) - Boston
New York
- Icahn School of Medicine at Mount Sinai - New York
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 30 participants |
| Start Date | 2012-04 |
| Est. Completion | 2027-01 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03409003
The ClinicalTrials.gov registry entry for NCT03409003 describes a study currently listed as recruiting, categorized as an unspecified phase. The registered enrollment target is 30 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Nicholas Ah Mew, which has 2 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with N-acetylglutamate Synthase (NAGS) Deficiency appearing as the primary indexed condition, and to 0 interventions.
NCT03409003 reports 3 study locations spanning 3 distinct geographic areas - top geographies include District of Columbia, Massachusetts, New York.
Frequently Asked Questions
What is clinical trial NCT03409003 about?
NCT03409003 is a clinical study titled "Orphan Europe Carbaglu® Surveillance Protocol". 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 synthas...
What is the current status of trial NCT03409003?
This trial is currently recruiting. The enrollment target is 30 participants. The study started on 2012-04. Estimated completion is 2027-01.
What conditions does trial NCT03409003 study?
This clinical trial studies the following conditions: N-acetylglutamate Synthase (NAGS) Deficiency.
Who is sponsoring clinical trial NCT03409003?
This trial is sponsored by Nicholas Ah Mew, which has 2 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03409003 being conducted?
This trial has 3 study locations across District of Columbia, Massachusetts, New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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