19 completed, 5 recruiting, 2 terminated, 2 unknown. 10 Phase 2, 6 Not applicable, 4 Phase 1, 3 Phase 3, 1 Phase 4.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 10 | Completed 7, Recruiting 2, Terminated 1 |
| Not applicable | 6 | Completed 4, Recruiting 2 |
| Phase 1 | 4 | Completed 2, Recruiting 2 |
| Phase 3 | 3 | Completed 3 |
| Phase 4 | 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 |
|---|---|---|---|---|---|---|
| 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 |
| A Clinical Study of Glycerol Phenylbutyrate in Chinese Patients With Urea Cycle Disorders Glycerol phenylbutyrate Oral Liquid - Urea Cycle Disorders | Recruiting | Tongji Hospital | 40 | 2025-12-09 | 2026-04-27 | |
| At Home Ammonia Monitoring of Inborn Errors of Ammonia Metabolism Ammonia Study Device - Urea Cycle Disorders, Organic Acidemias | Not applicable | Recruiting | Sequitur Health Corp. | 30 | 2025-09-23 | 2026-05-27 |
| Ureagenesis Analysis in Healthy Subjects and in Urea Cycle Disorder Patients Urea cycle flux study - Urea Cycle Disorders | Not applicable | Recruiting | University Children's Hospital, Zurich | 100 | 2019-10-31 | 2026-01-08 |
| Clinical Food Study to Evaluate the Effect of KB195 on Gut Nitrogen Metabolism in Patients With Urea Cycle Disorders KB195 (a novel mixture of oligosaccharides) - Urea Cycle Disorders | Not applicable | Completed | Kaleido Biosciences | 4 | 2019-01-06 | 2020-01-10 |
| Neuroimaging and Neuropsychological Outcomes in Urea Cycle Disorders MRI, Behavioral - Urea Cycle Disorders | Unknown | Children's National Research Institute | 56 | 2016-08-01 | 2024-06-25 | |
| PCORI Urea Cycle Disorder Study No Intervention Given - Urea Cycle Disorders | Completed | Children's National Research Institute | 187 | 2016-03-04 | 2021-01-26 | |
| 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 |
| Pilot Study: Urea Cycle Disorders Practice Patterns and Outcomes Assessment Urea Cycle Disorders | Completed | University of South Florida | 87 | 2014-10-01 | 2018-03-07 | |
| Nitric Oxide Supplementation in Argininosuccinic Aciduria Nitric oxide supplement, Placebo - Argininosuccinic Aciduria, Argininosuccinate Lyase Deficiency | Not applicable | Completed | Baylor College of Medicine | 12 | 2014-09-01 | 2020-01-18 |
| Long-term Safety Follow-up Study of Patients Having Received HepaStem (SAF001) Urea Cycle Disorders, Crigler Najjar Syndrome | Completed | Cellaion SA | 9 | 2013-03-01 | 2019-05-28 | |
| 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 |
| Oxidative Stress, Inflammation and Acute Decompensation in Urea Cycle Disorders Urea Cycle Disorders | Terminated | Mark Batshaw | 10 | 2012-02-01 | 2015-10-02 | |
| Study in Healthy Subjects, Patients With Urea Cycle Disorders (UCD) and Carriers of UCD Mutations to Evaluate Urea Cycle Function oral administration of Sodium [1,2-13C]-Acetate - Urea Cycle Disorders | Not applicable | Completed | Cytonet GmbH & Co. KG | 37 | 2012-01-01 | 2013-06-26 |
| Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs) HPN-100 - Urea Cycle Disorders | Phase 3 | Completed | Amgen | 23 | 2011-07-01 | 2024-07-11 |
| 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 |
| To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs) HPN-100 - Urea Cycle Disorders | Phase 4 | Completed | Amgen | 88 | 2010-10-04 | 2024-08-22 |
| 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 |
| Study of the Safety of HPN (Hyperion)-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD) HPN-100 - Urea Cycle Disorders | Phase 3 | Completed | Amgen | 60 | 2009-11-01 | 2024-07-10 |
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Urea Cycle Disorders, Inborn (28) · Hyperammonemia (6) · Argininosuccinic Aciduria (4) · Ornithine Carbamoyltransferase Deficiency Disease (3) · Amino Acid Metabolism, Inborn Errors (2) · Citrullinemia (2) · Crigler-Najjar Syndrome (2) · Adult-onset citrullinemia type 2 (1) · Brain Diseases (1) · Brain Diseases, Metabolic, Inborn (1) · Brain Edema (1) · Carbamoyl-Phosphate Synthase I Deficiency Disease (1) · Coma (1) · Hepatic Encephalopathy (1) · Hyperargininemia (1)
Brain Diseases, Metabolic (28) · Central Nervous System Diseases (28) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (28) · Genetic Diseases, Inborn (28) · Metabolism, Inborn Errors (28) · Nervous System Diseases (28) · Nutritional and Metabolic Diseases (28) · Brain Diseases (27) · Brain Diseases, Metabolic, Inborn (27) · Metabolic Diseases (27) · Amino Acid Metabolism, Inborn Errors (26) · Pathologic Processes (6) · Pathological Conditions, Signs and Symptoms (6) · Genetic Diseases, X-Linked (3) · Hyperbilirubinemia, Hereditary (2)
glycerol phenylbutyrate (8) · 4-phenylbutyric acid (3) · Acetates (1) · Arginine (1) · Continuous Renal Replacement Therapy (1) · Cool-Down Exercise (1) · Cryotherapy (1) · Dialysis (1) · Hypothermia, Induced (1) · Neuropsychological Tests (1) · Renal Dialysis (1) · Sodium (1)
Amgen (7) · Cytonet GmbH & Co. KG (4) · Cellaion SA (3) · Children's National Research Institute (2) · Andrea Gropman (1) · Baylor College of Medicine (1) · Brendan Lee (1) · Kaleido Biosciences (1) · Korro Bio, Inc. (1) · Mark Batshaw (1) · Rebecca Ahrens-Nicklas (1) · Sequitur Health Corp. (1) · Tongji Hospital (1) · University Children's Hospital, Zurich (1) · University of South Florida (1)
United States (17) · Canada (7) · Germany (4) · Belgium (3) · Switzerland (3) · France (2) · Australia (1) · China (1) · Israel (1) · Italy (1) · Poland (1) · Spain (1) · Ukraine (1) · United Kingdom (1)
Andrea Gropman, MD (1) · Andrea L. Gropman, M.D. (1) · Brendan Lee, MD, PhD (1) · Doris Neuenhofer, MD (1) · Dries Dobbelaere, MD/Prof (1) · Georg F Hoffmann, Prof Dr med (1) · Georg Hoffmann, Prof. (1) · George Diaz (1) · Giuliano Torre, MD (1) · Hanna Mandel, MD (1) · Jeffrey Krischer, PhD (1)
PMID 24144944, PMID 22961727, PMID 20188616, PMID 12736767, PMID 18562231, PMID 18662894, PMID 19287347, PMID 20004862, PMID 20488904, PMID 20512995, PMID 21482610, PMID 22110002, PMID 22901741, PMID 23324524, PMID 24084106, PMID 24485820, PMID 24881970, PMID 25135652, PMID 25712381, PMID 26067829, PMID 27132782, PMID 27215558, PMID 32028991
U54HD061221 (3), R44HD112243 (1), U54RR019453 (1)