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Carglumic Acid in Propionic Acidemia or Methylmalonic Acidemia
NCT04284917Completed
- Sponsor
- National Taiwan University Hospital
- Start
- Nov 2019
- Primary completion
- Dec 2024
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Open-label, single-arm trial of carglumic acid in propionic acidemia (PA) and methylmalonic acidemia (MMA), sponsored by National Taiwan University Hospital. Primary endpoint: Number of emergency visits due to hyperammonemia at 12 months. Started Nov 2019; status: completed.
02
Drugs
carglumic acid
03
Conditions
propionic acidemia (PA)methylmalonic acidemia (MMA)
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