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