3 completed, 2 recruiting, 1 terminated, 1 unknown. 4 Phase 2, 2 Phase 3, 1 Not applicable.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 4 | Completed 1, Terminated 1, Unknown 1, Withdrawn 1 |
| Phase 3 | 2 | Completed 1, Unknown 1 |
| Not applicable | 1 | Completed 1 |
Follow carglumic acid: new trials by email Literature Review of carglumic acid trials Research Report on carglumic acid
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics Carglumic Acid - Hyperammonemia, Methylmalonic Acidemia | Recruiting | RECORDATI GROUP | 20 | 2022-06-30 | 2025-04-15 | |
| Long-term Efficacy of Carglumic Acid in Organic Acidemia. Carglumic Acid (Carbaglu®) - Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA) | Not applicable | Completed | National Taiwan University Hospital | 3 | 2019-11-28 | 2025-03-20 |
| Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach Carglumic Acid - Methylmalonic Acidemia, Propionic Acidemia | Recruiting | RECORDATI GROUP | 95 | 2019-01-15 | 2024-02-05 | |
| Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia Carglumic Acid - Propionic Acidemia, Methylmalonic Acidemia | Phase 3 | Completed | King Abdullah International Medical Research Center | 33 | 2015-11-01 | 2019-04-25 |
| Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia Carbaglu, Placebo - Propionic Acidemia, Type I and/or Type II, Methylmalonic Acidemia | Phase 2 | Completed | Mendel Tuchman | 35 | 2012-09-01 | 2021-02-15 |
| Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia N-carbamylglutamate, Standard of Care - Propionic Acidemia, Methylmalonic Acidemia | Phase 2 | Terminated | Mendel Tuchman | 1 | 2012-09-01 | 2017-04-26 |
| Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate N-carbamylglutamate - Urea Cycle Disorders, Inborn, Inborn Errors of Metabolism | Phase 2 | Withdrawn | Children's Hospital of Philadelphia | 2010-12-01 | 2014-05-29 | |
| N-Carbamylglutamate (Carbaglu) In The Treatment Of Hyperammonemia N-carbamylglutamate - Inborn Errors of Metabolism | Phase 2 / Phase 3 | Unknown | Mendel Tuchman | 52 | 2008-08-01 | 2020-02-17 |
Methylmalonic acidemia (7) · Propionic Acidemia (7) · Hyperammonemia (3) · Carbamoyl-Phosphate Synthase I Deficiency Disease (2) · Metabolism, Inborn Errors (2) · Ornithine Carbamoyltransferase Deficiency Disease (1) · Urea Cycle Disorders, Inborn (1)
Mendel Tuchman (3) · RECORDATI GROUP (2) · Children's Hospital of Philadelphia (1) · King Abdullah International Medical Research Center (1) · National Taiwan University Hospital (1)
United States (4) · France (1) · Germany (1) · Italy (1) · Norway (1) · Saudi Arabia (1) · Spain (1) · Sweden (1) · Taiwan (1) · United Kingdom (1)
Mendel Tuchman, MD (3) · Majid A. Alfadhel, MD (1) · Marc Yudkoff, MD (1) · Ni-Chung Lee, M.D., Ph.D (1) · Nicholas Ah Mew, MD (1) · Sufin Yap, MD (1) · William Ludlum, MD (1)
PMID 18414145, PMID 19660971, PMID 20338795, PMID 20566609, PMID 31196016, PMID 33006765, PMID 42839282
R01HD058567 (2)