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Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia — CAMP  

Propionic Acidemia Clinical Trial

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 Acidemia (MMA)

Clinical Trial 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.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT02426775
Study type Interventional
Source King Abdullah International Medical Research Center
Contact
Status Completed
Phase Phase 3
Start date November 2015
Completion date March 19, 2019
View Details
See also
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Recruiting NCT04159103 - Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia Phase 1/Phase 2