7 completed, 2 recruiting, 1 terminated. 10 Phase 2, 3 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 10 | Completed 7, Recruiting 2, Terminated 1 |
| Phase 1 | 3 | Recruiting 2, 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 |
|---|---|---|---|---|---|---|
| Phase 1/2 Study of KRRO-121 in Healthy Volunteers and Patients With UCD KRRO-121, Placebo - Urea Cycle Disorders, Inborn, Urea Cycle Disorders | Phase 1 / Phase 2 | Recruiting | Korro Bio, Inc. | 30 | 2026-09-28 | 2026-10-08 |
| Study of LNP.UCD.ABE in Patients With Urea Cycle Disorders LNP.UCD.ABE - Urea Cycle Disorders, Carbamoyl-Phosphate Synthase I Deficiency | Phase 1 / Phase 2 | Recruiting | Rebecca Ahrens-Nicklas | 7 | 2026-08-07 | 2026-08-26 |
| 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 |
| Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders HHLivC - Urea Cycle Disorders | Phase 2 | Terminated | Cytonet GmbH & Co. KG | 10 | 2010-12-01 | 2016-02-08 |
| 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 (10) · Argininosuccinic Aciduria (3) · Ornithine Carbamoyltransferase Deficiency Disease (3) · Citrullinemia (2) · Hyperammonemia (2) · Adult-onset citrullinemia type 2 (1) · 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) · Hyperargininemia (1) · Lethargy (1) · Methylmalonic acidemia (1)
Brain Diseases, Metabolic (10) · Brain Diseases, Metabolic, Inborn (10) · Central Nervous System Diseases (10) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (10) · Genetic Diseases, Inborn (10) · Metabolic Diseases (10) · Metabolism, Inborn Errors (10) · Nervous System Diseases (10) · Nutritional and Metabolic Diseases (10) · Amino Acid Metabolism, Inborn Errors (9) · Brain Diseases (9) · Genetic Diseases, X-Linked (3) · 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) · Cytonet GmbH & Co. KG (2) · Brendan Lee (1) · Korro Bio, Inc. (1) · Rebecca Ahrens-Nicklas (1) · Uta Lichter-Konecki (1)
United States (6) · Belgium (2) · Canada (2) · France (2) · Australia (1) · Germany (1) · Israel (1) · Italy (1) · Poland (1) · Spain (1) · United Kingdom (1)
Brendan Lee, MD, PhD (1) · Georg Hoffmann, Prof. (1) · Rebecca Ahrens-Nicklas, M.D., Ph.D. (1) · Uta Lichter-Konecki, MD, PhD (1)
PMID 22961727, PMID 24144944, PMID 32028991
U54HD061221 (1)