Propionic Acidemia Clinical Trial
Official title:
Randomized Multicentre Comparative Trial to Evaluate the Long Term Effectiveness of the Use of Carbaglu® in Patients With Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA)
A Phase IIIb (Three b), Randomized Multicentre Comparative Trial to Evaluate the Long Term
Effectiveness & Safety of the use of Carglumic Acid (Carbaglu®) in Patients with Propionic
Acidemia (PA) or Methylmalonic Acidemia (MMA). Carbaglu® clinical experience in Organic
Acidemia (OA) is limited to a non-comparative retrospective collection of data from patients
who had received Carbaglu® for 1 to 15 days.
There is no current evidence supporting the use of carglumic acid for the chronic management
of patients with OA. The investigators are proposing a randomized multicentre prospective
clinical trial to evaluate long-term effects of the use of Carbaglu® (50mg/kg/day) combined
with standard chronic therapy in patients with PA and MMA compared to standard chronic
therapy alone.
n/a
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