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An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics

Propionic Acidemia Clinical Trial

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

Clinical Trial Summary

To obtain short-term and long-term clinical safety information, in pediatric and adult patients with PA and MMA treated with Carbaglu®.

Clinical Trial Description

This study is being conducted to obtain short-term and long-term clinical safety information from adult and pediatric patients treated for hyperammonemia due to Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA). This is an observational/non-interventional study. Patients will be treated per the prescribing information and routine medical practice. Only available data will be collected as part of the study including developmental outcomes, details of treatment with Carbaglu® and other treatments for hyperammonemia including dietary and protein management, plasma ammonia levels, pregnancy and maternal complications, adverse effects on the developing fetus and neonate, adverse effects on the infant through first year of life. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05040178
Study type Observational
Source Recordati Rare Diseases
Contact Anne Marie Cesario
Phone 908-849-4907
Email cesario.a@recordati.com
Status Recruiting
Phase
Start date June 30, 2022
Completion date June 30, 2032
View Details
See also
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