drugset / Trial / NCT04284917
Long-term Efficacy of Carglumic Acid in Organic Acidemia.
NaSingle-groupOpen-labelTreatment
Summary
Evaluate the Long Term Effectiveness \& Safety of the use of Carglumic Acid (Carbaglu®) in Patients with Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA).
Timeline
- Start
- 2019-11-28
- Primary completion
- 2024-12-31
- Completion
- 2025-03-11
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Carglumic Acid | Unknown | 50 mg/kg | — |