NCT02426775

Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia

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)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 33 (actual)
• Sponsor: King Abdullah International Medical Research Center · Academic / Other
• Sex: All
• Age: 15 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Propionic Acidemia
• Methylmalonic Acidemia

Interventions

Type	Name	Description
DRUG	Carglumic Acid	Carglumic Acid 50 mg/kg/day (twice daily)

Timeline

• Start date: 2015-11-01
• Primary completion: 2019-03-19
• Completion: 2019-03-19
• First posted: 2015-04-27
• Last updated: 2019-04-25

Locations

2 sites across 1 country: Saudi Arabia