Orphan Europe Carbaglu® Surveillance Protocol
1 other identifier
observational
30
1 country
3
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for all trials
Started Apr 2012
Longer than P75 for all trials
3 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
April 1, 2012
CompletedFirst Submitted
Initial submission to the registry
November 18, 2016
CompletedFirst Posted
Study publicly available on registry
January 24, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 1, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
January 1, 2027
February 7, 2024
February 1, 2024
14.3 years
November 18, 2016
February 6, 2024
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
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
Secondary Outcomes (5)
Number of hyperammonemic events
15 years
IQ
15 years
Height
15 years
Weight
15 years
Abnormal physical and neurological findings
15 years
Eligibility Criteria
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)
You may qualify if:
- 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 may not qualify if:
- 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 will be excluded
- Individuals with extreme low birth weight (\<1,500 grams) will be also excluded.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Nicholas Ah Mewlead
- Boston Children's Hospitalcollaborator
- Icahn School of Medicine at Mount Sinaicollaborator
Study Sites (3)
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
Icahn School of Medicine at Mount Sinai
New York, New York, 10029, United States
Related Links
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Nicholas Ah Mew, MD
Children's National Research Institute
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Target Duration
- 15 Years
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR INVESTIGATOR
- PI Title
- Principal Investigator
Study Record Dates
First Submitted
November 18, 2016
First Posted
January 24, 2018
Study Start
April 1, 2012
Primary Completion (Estimated)
July 1, 2026
Study Completion (Estimated)
January 1, 2027
Last Updated
February 7, 2024
Record last verified: 2024-02
Data Sharing
- IPD Sharing
- Will not share
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.