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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
33
Carglumic Acid 50 mg/kg/day (twice daily)
King Abdullah Specialist Children Hospital, King Abdulaziz Medical City
Riyadh, Saudi Arabia
King Fahad Medical City
Riyadh, Saudi Arabia
Number of emergency visits due to hyperammonemia within 24 months period
Time frame: 24 months
Time to first visit to the ER due to hyperammonemia from starting the treatment.
Time frame: 24 months
Plasma ammonia levels over the study treatment period.
Time frame: 24 months
Number of days of hospitalization
Time frame: 24 months
Acylcarnitine level for all patients
Time frame: once on screening visit
Measuring urine organic acid levels for both diseases.
Time frame: 24 months
Measuring Plasma aminoacids' levels for both diseases
Time frame: 24 months
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