2 completed, 2 recruiting. 4 Phase 1, 3 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 4 | Completed 2, Recruiting 2 |
| Phase 2 | 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 |
| 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 |
| A Study of Glyceryl Tri-(4-phenylbutyrate) Administered Orally as a Single Dose, and Twice Daily for Seven Consecutive Days to Subjects With Hepatic Impairment With Cirrhosis and to a Control Group HPN-100 - Hepatic Encephalopathy, Urea Cycle Disorders | Phase 1 | Completed | Amgen | 32 | 2006-09-01 | 2024-06-20 |
Urea Cycle Disorders, Inborn (4) · Adult-onset citrullinemia type 2 (1) · Argininosuccinic Aciduria (1) · Crigler-Najjar Syndrome (1) · Hepatic Encephalopathy (1) · Hyperargininemia (1) · N-acetyl glutamate synthetase deficiency (1) · Ornithine Carbamoyltransferase Deficiency Disease (1)
Amino Acid Metabolism, Inborn Errors (4) · Brain Diseases (4) · Brain Diseases, Metabolic (4) · Brain Diseases, Metabolic, Inborn (4) · Central Nervous System Diseases (4) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (4) · Genetic Diseases, Inborn (4) · Metabolic Diseases (4) · Metabolism, Inborn Errors (4) · Nervous System Diseases (4) · Nutritional and Metabolic Diseases (4) · Digestive System Diseases (1) · Genetic Diseases, X-Linked (1) · Hepatic Insufficiency (1) · Hyperbilirubinemia, Hereditary (1)
Amgen (1) · Cellaion SA (1) · Korro Bio, Inc. (1) · Rebecca Ahrens-Nicklas (1)
Australia (1) · Belgium (1) · France (1) · Israel (1) · Italy (1) · Ukraine (1) · United Kingdom (1) · United States (1)
Rebecca Ahrens-Nicklas, M.D., Ph.D. (1)