Orphan Europe Carbaglu® Surveillance Protocol

Status: Recruiting
Location: See all (3) locations...
Study Type: Observational
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.

Eligibility
Participation Requirements
Sex: All
Healthy Volunteers: f
View:

• 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)

Locations
United States
Washington, D.c.
Children's National Medical Center
RECRUITING
Washington D.c.
Massachusetts
Children's Hospital Boston (UCDC New England Center)
RECRUITING
Boston
New York
Icahn School of Medicine at Mount Sinai
RECRUITING
New York
Contact Information
Primary
Jennifer Seminara, MPH
jseminar@childrensnational.org
2023066489
Time Frame
Start Date: 2012-04
Estimated Completion Date: 2027-01
Participants
Target number of participants: 30
Authors
George A. Diaz
Sponsors
Collaborators: Boston Children's Hospital, Icahn School of Medicine at Mount Sinai
Leads: Nicholas Ah Mew

This content was sourced from clinicaltrials.gov