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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: Recruiting study of N-acetylglutamate Synthase (NAGS) Deficiency, sponsored by Nicholas Ah Mew.
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. According to ClinicalTrials.gov, the official US trial registry.
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.
Primary Outcome
The primary outcome measure is to monitor adverse events and adverse reactions, which will be reported to the FDA to fulfill post-marketing surveillance requirements.
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 |
What NCT03409003 shows while recruiting
NCT03409003 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 30 participants, a relatively small participant target.
The record links to 1 condition, with N-acetylglutamate Synthase (NAGS) Deficiency appearing as the primary indexed condition, and to 0 interventions.
NCT03409003 reports a single indexed study location in 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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