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Orphan Europe Carbaglu® Surveillance Protocol

NCT03409003 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 30

Last updated 2024-02-07

No results posted yet for this 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

  • N-acetylglutamate Synthase (NAGS) Deficiency

Sponsors & Collaborators

Principal Investigators

  • Nicholas Ah Mew, MD · Children's National Research Institute

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-04-30
Primary Completion
2026-07-31
Completion
2027-01-31

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03409003 on ClinicalTrials.gov