Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia
Status:
Completed
Trial end date:
2019-03-19
Target enrollment:
Participant gender:
Summary
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.
Phase:
Phase 3
Details
Lead Sponsor:
King Abdullah International Medical Research Center