7 completed. 7 Phase 2, 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 7 | Completed 7 |
| Phase 1 | 1 | Completed 1 |
Follow Urea Cycle Disorders: new trials by email Literature Review of Urea Cycle Disorders trials Research Report on Urea Cycle Disorders
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 |
|---|---|---|---|---|---|---|
| Study to Evaluate the Efficacy of HepaStem in Urea Cycle Disorders Paediatric Patients (HEP002) HepaStem - Urea Cycle Disorders | Phase 2 | Completed | Cellaion SA | 5 | 2014-10-01 | 2020-10-19 |
| Safety Study of HepaStem for the Treatment of Urea Cycle Disorders (UCD) and Crigler-Najjar Syndrome (CN) HepaStem - Urea Cycle Disorders, Crigler Najjar Syndrome | Phase 1 / Phase 2 | Completed | Cellaion SA | 20 | 2012-03-01 | 2020-10-19 |
| Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders HPN-100, NaPBA - Urea Cycle Disorders | Phase 2 | Completed | Amgen | 17 | 2010-03-01 | 2024-07-11 |
| Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders Human Heterologous Liver Cells - Urea Cycle Disorders, Carbamoylphosphate Synthetase I Deficiency | Phase 2 | Completed | Cytonet GmbH & Co. KG | 12 | 2008-07-01 | 2016-02-08 |
| Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder Sodium Phenylbutyrate, Arginine - Argininosuccinic Aciduria, Amino Acid Metabolism, Inborn Errors | Phase 2 | Completed | Brendan Lee | 12 | 2008-02-01 | 2018-01-31 |
| Dose-Escalation Safety Study of HPN-100 to Treat Urea Cycle Disorders HPN-100, BUPHENYL® - Urea Cycle Disorders | Phase 2 | Completed | Amgen | 14 | 2007-10-01 | 2024-07-10 |
| Pilot Study For Hypothermia Treatment In Hyperammonemic Encephalopathy In Neonates And Very Young Infants Therapeutic Hypothermia, Standard of Care Therapy - Urea Cycle Disorders, Organic Acidemias | Phase 2 | Completed | Uta Lichter-Konecki | 5 | 2007-08-01 | 2015-06-01 |
Urea Cycle Disorders, Inborn (7) · Argininosuccinic Aciduria (2) · Citrullinemia (2) · Hyperammonemia (2) · Ornithine Carbamoyltransferase Deficiency Disease (2) · Amino Acid Metabolism, Inborn Errors (1) · Brain Diseases (1) · Brain Edema (1) · Carbamoyl-Phosphate Synthase I Deficiency Disease (1) · Coma (1) · Crigler-Najjar Syndrome (1) · Lethargy (1) · Methylmalonic acidemia (1) · Propionic Acidemia (1) · Seizures (1)
Brain Diseases, Metabolic (7) · Brain Diseases, Metabolic, Inborn (7) · Central Nervous System Diseases (7) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (7) · Genetic Diseases, Inborn (7) · Metabolic Diseases (7) · Metabolism, Inborn Errors (7) · Nervous System Diseases (7) · Nutritional and Metabolic Diseases (7) · Amino Acid Metabolism, Inborn Errors (6) · Brain Diseases (6) · Genetic Diseases, X-Linked (2) · Pathologic Processes (2) · Pathological Conditions, Signs and Symptoms (2) · Consciousness Disorders (1)
4-phenylbutyric acid (2) · glycerol phenylbutyrate (2) · Arginine (1) · Continuous Renal Replacement Therapy (1) · Cool-Down Exercise (1) · Cryotherapy (1) · Dialysis (1) · Hypothermia, Induced (1) · Renal Dialysis (1)
Amgen (2) · Cellaion SA (2) · Brendan Lee (1) · Cytonet GmbH & Co. KG (1) · Uta Lichter-Konecki (1)
United States (4) · Belgium (2) · France (2) · Canada (1) · Germany (1) · Israel (1) · Italy (1) · Poland (1) · Spain (1) · United Kingdom (1)
Brendan Lee, MD, PhD (1) · Georg Hoffmann, Prof. (1) · Uta Lichter-Konecki, MD, PhD (1)
PMID 22961727, PMID 24144944, PMID 32028991
U54HD061221 (1)