2 completed, 1 recruiting. 2 Not applicable, 1 Phase 1, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Not applicable | 2 | Completed 2 |
| Phase 1 | 1 | Recruiting 1 |
| Phase 2 | 1 | Recruiting 1 |
Follow Argininosuccinate Lyase Deficiency: new trials by email Literature Review of Argininosuccinate Lyase Deficiency trials Research Report on Argininosuccinate Lyase Deficiency
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 |
| 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 |
Argininosuccinic Aciduria (3) · Urea Cycle Disorders, Inborn (3) · Adult-onset citrullinemia type 2 (1) · Hyperargininemia (1) · N-acetyl glutamate synthetase deficiency (1) · Ornithine Carbamoyltransferase Deficiency Disease (1)
Amino Acid Metabolism, Inborn Errors (3) · Brain Diseases (3) · Brain Diseases, Metabolic (3) · Brain Diseases, Metabolic, Inborn (3) · Central Nervous System Diseases (3) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (3) · Genetic Diseases, Inborn (3) · Metabolic Diseases (3) · Metabolism, Inborn Errors (3) · Nervous System Diseases (3) · Nutritional and Metabolic Diseases (3) · Genetic Diseases, X-Linked (1)
Baylor College of Medicine (2) · Korro Bio, Inc. (1)
United States (2) · Australia (1)
Brendan Lee, M.D., PhD (1) · Sandesh C Nagamani, M.D. (1) · Sandesh CS Nagamani, MD, FACMG (1)