Clinical Research Directory
Browse clinical research sites, groups, and studies.
An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics
Sponsor: RECORDATI GROUP
Summary
To obtain short-term and long-term clinical safety information, in pediatric and adult patients with PA and MMA treated with Carbaglu®.
Official title: A Non-Interventional Post-Authorization Safety Study (PASS) of Carbaglu® for the Treatment of Hyperammonemia Due to Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA) in Adult and Pediatric Patient Populations
Key Details
Gender
All
Age Range
Any - Any
Study Type
OBSERVATIONAL
Enrollment
20
Start Date
2022-06-30
Completion Date
2032-06-30
Last Updated
2025-04-15
Healthy Volunteers
No
Interventions
Carglumic Acid
Current or previous treatment with Carbaglu, the dose of Carbaglu® prescribed will be determined by the investigator for each individual patient.
Locations (5)
Children's National Hospital
Washington D.C., District of Columbia, United States
University of South Florida
Tampa, Florida, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Riley Children's Hospital
Indianapolis, Indiana, United States
Icahn School of Medicine at Mt. Sinai
New York, New York, United States