7 completed, 5 recruiting, 2 active not recruiting, 2 terminated. 3 Phase 2, 3 Phase 3, 2 Not applicable, 2 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 3 | Completed 2, Recruiting 1 |
| Phase 3 | 3 | Completed 2, Terminated 1 |
| Not applicable | 2 | Completed 1, Recruiting 1 |
| Phase 1 | 2 | Completed 1, Recruiting 1 |
Follow Hyperargininemia: new trials by email Literature Review of Hyperargininemia trials Research Report on Hyperargininemia
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 |
| Evaluation of the Safety of Loargys Arginine Test System in Loargys-treated Patients Loargys Arginine Test System - ARG1 Deficiency | Not applicable | Recruiting | Immedica Pharma US Inc | 100 | 2026-04-17 | 2026-05-07 |
| Pegzilarginase in Subjects <24 Months Old With Arginase 1 Deficiency Pegzilarginase - Arginase 1 Deficiency | Phase 3 | Completed | Immedica Pharma AB | 3 | 2024-08-30 | 2026-05-22 |
| A Study of Safety of Weekly Subcutaneous Pegzilarginase in Subjects With Arginase 1 Deficiency Pegzilarginase - Arginase I Deficiency, Hyperargininemia | Phase 3 | Terminated | Aeglea Biotherapeutics | 3 | 2023-04-04 | 2023-07-27 |
| Triple Recipient Strategy Using Split, Domino, and Auxiliary Techniques Hepatocellular Carcinoma (HCC) | Completed | Second Affiliated Hospital, Zhejiang University, School of Medicine | 3 | 2023-04-02 | 2024-12-05 | |
| Improving Quality of Life and Daily Life Activities With Bioarginine in Patients With COPD Arginine, Placebo - COPD, Dyspnea | Not applicable | Completed | University of Milan | 153 | 2023-01-18 | 2026-05-04 |
| Selective Screening of Children for Hereditary Metabolic Diseases by Tandem Mass Spectrometry in Kazakhstan Obtaining Dry Blood Spots From Healthy Newborns (Aged 1-7 Days), Obtaining Dry Blood Spots From Healthy Children Aged 8 Days - 7 Years, Obtaining Dry Blood Spots From Healthy Children Aged 8-18 Years - Propionic/Methylmalonic Acidemias, Maple Syrup Urine Disease | Unknown | West Kazakhstan Medical University | 2250 | 2022-10-03 | 2023-06-18 | |
| Baby Detect : Genomic Newborn Screening Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1 | Completed | Centre Hospitalier Universitaire de Liege | 6824 | 2022-09-01 | 2025-08-12 | |
| Hepatic Histopathology in Urea Cycle Disorders Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency | Recruiting | Baylor College of Medicine | 70 | 2022-02-24 | 2025-07-20 | |
| Liver Disease in Urea Cycle Disorders Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency | Active not recruiting | Baylor College of Medicine | 62 | 2021-11-04 | 2026-04-27 | |
| Systemic Biomarkers of Brain Injury From Hyperammonemia Urea Cycle Disorder, Organic Acidemia | Recruiting | Children's National Research Institute | 24 | 2020-07-09 | 2024-02-07 | |
| Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency Pegzilarginase, Placebo - Arginase I Deficiency, Hyperargininemia | Phase 3 | Completed | Immedica Pharma AB | 32 | 2019-05-01 | 2026-08-21 |
| Early Check: Expanded Screening in Newborns Confirmatory Testing - Spinal Muscular Atrophy, Fragile X Syndrome | Active not recruiting | RTI International | 30000 | 2018-10-15 | 2026-07-01 | |
| A Study of AEB1102 (Pegzilarginase) in Patients With Arginase 1 Deficiency AEB1102 - Arginase I Deficiency, Hyperargininemia | Phase 2 | Completed | Immedica Pharma AB | 14 | 2017-12-07 | 2026-09-24 |
| A Phase 1/2 Study of AEB1102 in Patients With Arginase I Deficiency AEB1102 - Arginase I Deficiency, Hyperargininemia | Phase 1 / Phase 2 | Completed | Aeglea Biotherapeutics | 16 | 2016-06-01 | 2019-06-12 |
| The NIH UNI Study: Urea Cycle Disorders, Nutrition and Immunity Argininosuccinic Aciduria, Carbamoyl-Phosphate Synthase I Deficiency | Terminated | National Human Genome Research Institute (NHGRI) | 4 | 2011-08-08 | 2019-12-09 | |
| Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford Rare Disorders, Undiagnosed Disorders | Recruiting | Sanford Health | 20000 | 2010-07-01 | 2026-10-28 |
Argininosuccinic Aciduria (8) · Ornithine Carbamoyltransferase Deficiency Disease (8) · Citrullinemia (7) · N-acetyl glutamate synthetase deficiency (6) · Carbamoyl-Phosphate Synthase I Deficiency Disease (5) · Urea Cycle Disorders, Inborn (5) · Carnitine-Acylcarnitine Translocase Deficiency (4) · Glutaric Acidemia I (4) · Medium chain acyl CoA dehydrogenase deficiency (4) · Trifunctional Protein Deficiency With Myopathy And Neuropathy (4) · 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency (3) · Acidemia, isovaleric (3) · Ataxia with vitamin E deficiency (3) · Beta ketothiolase deficiency (3)
Amino Acid Metabolism, Inborn Errors (17) · Brain Diseases (17) · Brain Diseases, Metabolic (17) · Brain Diseases, Metabolic, Inborn (17) · Central Nervous System Diseases (17) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (17) · Genetic Diseases, Inborn (17) · Metabolic Diseases (17) · Nervous System Diseases (17) · Nutritional and Metabolic Diseases (17) · Metabolism, Inborn Errors (16) · Urea Cycle Disorders, Inborn (12) · Genetic Diseases, X-Linked (9) · Digestive System Diseases (6) · Pathological Conditions, Signs and Symptoms (6)
Immedica Pharma AB (3) · Aeglea Biotherapeutics (2) · Baylor College of Medicine (2) · Centre Hospitalier Universitaire de Liege (1) · Children's National Research Institute (1) · Immedica Pharma US Inc (1) · Korro Bio, Inc. (1) · National Human Genome Research Institute (NHGRI) (1) · RTI International (1) · Sanford Health (1) · Second Affiliated Hospital, Zhejiang University, School of Medicine (1) · University of Milan (1) · West Kazakhstan Medical University (1)
United States (10) · Canada (4) · United Kingdom (4) · Portugal (3) · Austria (2) · Italy (2) · Australia (1) · Belgium (1) · China (1) · France (1) · Germany (1) · Kazakhstan (1)
Mattias Rudebeck, PhD MSc BMedSc (3) · Lindsay Burrage, MD, PhD (2) · Cortney Caudill (1) · Curt Scharfe, MD, PhD (1) · Gulmira M. Zharmakhanova, MD, PhD (1) · Josie Gayton (1) · Laurent Servais (1) · Ljubica Caldovic, PhD (1) · Lyazzat M. Syrlybayeva, MD (1) · Mattias Rudebeck, PhD (1) · Nicholas Ah Mew, MD (1) · Peter J McGuire, M.D. (1) · Victoria I. Kononets, MD, MS (1)
PMID 33325055, PMID 31315600, PMID 35142618, PMID 38283151, PMID 38292042
R21TR003166 (1)