5 completed, 3 recruiting, 2 active not recruiting, 1 terminated. 3 Phase 2, 2 Not applicable, 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 3 | Completed 2, Recruiting 1 |
| Not applicable | 2 | Completed 2 |
| Phase 1 | 1 | Recruiting 1 |
Follow Argininosuccinic Aciduria: new trials by email Literature Review of Argininosuccinic Aciduria trials Research Report on Argininosuccinic Aciduria
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 |
| 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 | |
| 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 | |
| Nitric Oxide Supplementation on Neurocognitive Functions in Patients With ASLD Neo-ASA, Placebo - Argininosuccinate Lyase Deficiency, Urea Cycle Disorder | Not applicable | Completed | Baylor College of Medicine | 16 | 2017-09-15 | 2023-02-27 |
| 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 |
| 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 | |
| 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 |
| 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 |
Ornithine Carbamoyltransferase Deficiency Disease (9) · Urea Cycle Disorders, Inborn (9) · Citrullinemia (8) · Hyperargininemia (8) · Carbamoyl-Phosphate Synthase I Deficiency Disease (6) · N-acetyl glutamate synthetase deficiency (6) · 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) · Beta ketothiolase deficiency (3) · Biotinidase Deficiency (3)
Brain Diseases, Metabolic (12) · Brain Diseases, Metabolic, Inborn (12) · Central Nervous System Diseases (12) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (12) · Genetic Diseases, Inborn (12) · Metabolic Diseases (12) · Nervous System Diseases (12) · Nutritional and Metabolic Diseases (12) · Amino Acid Metabolism, Inborn Errors (11) · Brain Diseases (11) · Metabolism, Inborn Errors (11) · Genetic Diseases, X-Linked (9) · Mitochondrial Diseases (6) · Digestive System Diseases (4) · Pathological Conditions, Signs and Symptoms (4)
4-phenylbutyric acid (1) · Aging (1) · Arginine (1) · Continuous Renal Replacement Therapy (1) · Cool-Down Exercise (1) · Cryotherapy (1) · Dialysis (1) · Hypothermia, Induced (1) · Renal Dialysis (1)
Baylor College of Medicine (4) · Brendan Lee (1) · Centre Hospitalier Universitaire de Liege (1) · Children's National Research Institute (1) · Korro Bio, Inc. (1) · National Human Genome Research Institute (NHGRI) (1) · RTI International (1) · Uta Lichter-Konecki (1) · West Kazakhstan Medical University (1)
United States (9) · Australia (1) · Belgium (1) · Kazakhstan (1)
Lindsay Burrage, MD, PhD (2) · Brendan Lee, M.D., PhD (1) · Brendan Lee, MD, PhD (1) · Curt Scharfe, MD, PhD (1) · Gulmira M. Zharmakhanova, MD, PhD (1) · Laurent Servais (1) · Ljubica Caldovic, PhD (1) · Lyazzat M. Syrlybayeva, MD (1) · Nicholas Ah Mew, MD (1) · Peter J McGuire, M.D. (1) · Sandesh C Nagamani, M.D. (1) · Sandesh CS Nagamani, MD, FACMG (1) · Uta Lichter-Konecki, MD, PhD (1) · Victoria I. Kononets, MD, MS (1)
PMID 31315600, PMID 35142618, PMID 38283151
R21TR003166 (1), U54HD061221 (1)