Carglumic Acid

DRUG3 trials

Sponsors

King Abdullah International Medical Research Center, RECORDATI GROUP

Conditions

HyperammonemiaMethylmalonic AcidemiaPropionic Acidemia

Phase 3

Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia

CompletedNCT02426775

King Abdullah International Medical Research CenterMethylmalonic Acidemia, Propionic Acidemia

Start: 2015-11-30End: 2019-03-19Updated: 2019-04-25

Unknown Phase

Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach

RecruitingNCT04176523

RECORDATI GROUPMethylmalonic Acidemia, Propionic Acidemia

Start: 2019-01-15End: 2029-07-30Target: 95Updated: 2024-02-05

An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics

RecruitingNCT05040178

RECORDATI GROUPHyperammonemia, Methylmalonic Acidemia, Propionic Acidemia

Start: 2022-06-30End: 2032-06-30Target: 20Updated: 2025-04-15

Related Papers

Real world experience of carglumic acid for methylmalonic and propionic acidurias: the second interim analysis of the multicentre observational PROTECT study

Orphanet Journal of Rare Diseases2026-08-03

Real-World Experience of Carglumic Acid for Methylmalonic and Propionic Acidurias: An Interim Analysis of the Multicentre Observational PROTECT Study.

2024-01-105 citations

Evaluation of long-term effectiveness of the use of carglumic acid in patients with propionic acidemia (PA) or methylmalonic acidemia (MMA): study protocol for a randomized controlled trial

BMC Pediatrics2019-06-1319 citations