NCT05040178

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

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

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 20 (estimated)
• Sponsor: RECORDATI GROUP · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

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

Detailed 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.

Conditions

• Hyperammonemia
• Methylmalonic Acidemia
• Propionic Acidemia

Interventions

Type	Name	Description
DRUG	Carglumic Acid	Current or previous treatment with Carbaglu, the dose of Carbaglu® prescribed will be determined by the investigator for each individual patient.

Timeline

• Start date: 2022-06-30
• Primary completion: 2032-06-30
• Completion: 2032-06-30
• First posted: 2021-09-10
• Last updated: 2025-04-15

Locations

5 sites across 1 country: United States

Regulatory

• FDA-regulated drug study