NCT03409003|RecruitingDMC
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Sponsor study identifier
observational
30
1 country
3
Study Start
April 2012
Date type not reportedThis date has not been verified as actual or estimated.
Primary Completion
July 2026
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
Study Completion
January 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
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.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
January 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
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Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
April 2012
Date type not reportedThis date has not been verified as actual or estimated.
November 18, 2016
Actual (reported)January 24, 2018
Actual (reported)July 2026
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
January 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Carbaglu related adverse events and adverse reactions
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.
15 years
Number of hyperammonemic events
15 years
IQ
15 years
Height
15 years
Weight
15 years
Abnormal physical and neurological findings
15 years
Confirmed diagnosis of NAGS deficiency or suspicion of NAGS deficiency, taking Carbaglu for the treatment of NAGS, and enrolled in the Longitudinal Study of Urea Cycle Disorders (RDCRN protocol #5101)
Children's National Medical Center
Washington D.C., District of Columbia, 20010, United States
Children's Hospital Boston (UCDC New England Center)
Boston, Massachusetts, 02115, United States
Nicholas Ah Mew, MD
Children's National Research Institute
First Submitted
November 18, 2016
First Posted
January 24, 2018
Study Start
April 2012
Date type not reportedThis date has not been verified as actual or estimated.
Primary Completion
July 2026
Contact the study team to confirm eligibility.
Icahn School of Medicine at Mount Sinai
New York, New York, 10029, United States
This estimate has passed. The registry has not confirmed an actual date for this milestone.
Study Completion
January 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Last Updated
February 7, 2024
Record last verified: 2024-02
This is a post-marketing surveillance study being performed so that Orphan Europe (OE) can meet its post FDA approval reporting obligations. Data will be shared with OE who will then report to the FDA. There are no other plans to make this data available to other researchers.
Download the complete ClinicalTrials.gov record imported by Syfrah, including fields not shown in this page. The registry may contain a newer revision.
Source: ClinicalTrials.gov.
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