Clinical Trial: Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia
Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
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 Aci
Brief 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.
Detailed Summary:
Sponsor: King Abdullah International Medical Research Center
Current Primary Outcome: Number of emergency visits due to hyperammonemia within 24 months period [ Time Frame: 24 months ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- 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 ]
Original Secondary Outcome: Same as current
Information By: King Abdullah International Medical Research Center
Dates:
Date Received: April 22, 2015
Date Started: November 2015
Date Completion: March 2019
Last Updated: March 14, 2017
Last Verified: March 2017