Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA) Clinical Trial
Official title:
Evaluate the Long Term Effectiveness & Safety of the Use of Carglumic Acid (Carbaglu®) in Patients With Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA).
Evaluate the Long Term Effectiveness & Safety of the use of Carglumic Acid (Carbaglu®) in Patients with Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA).
Study procedures:
A. Study period: 24 months; treatment period: 12 months.
B. Patients will receive Carglumic Acid (Carbaglu®) 50 mg/kg/day in addition to standard
therapy (protein restricted diet, L-carnitine, metronidazole and vitamin B12).
C. Patients need to regularly come back for follow up examination on Day 0, month 3, month 6,
and month 12.
D. Patients should report any adverse event that occur during treatment period.
E. Patients' number of emergency visits due to hyperammonemia, ammonia level, acylcarnitine,
urinary organic acid analysis, plasma amino acid analysis will be recorded during treatment
period.
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