16 completed, 2 recruiting, 2 terminated, 2 withdrawn. 7 Phase 2, 5 Phase 3, 4 Phase 1, 2 Phase 4, 1 Not applicable.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 7 | Completed 5, Unknown 1, Withdrawn 1 |
| Phase 3 | 5 | Completed 3, Active not recruiting 1, Unknown 1 |
| Phase 1 | 4 | Completed 3, Terminated 1 |
| Phase 4 | 2 | Completed 2 |
| Not applicable | 1 | Completed 1 |
Follow Hyperammonemia: new trials by email Literature Review of Hyperammonemia trials Research Report on Hyperammonemia
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 |
|---|---|---|---|---|---|---|
| Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP) No Intervention - Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease | Recruiting | iECURE, Inc. | 13 | 2024-12-23 | 2026-11-05 | |
| An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency ECUR-506 - Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease | Phase 3 | Active not recruiting | iECURE, Inc. | 20 | 2024-04-08 | 2026-09-30 |
| Non-Cirrhotic Hyperammonemia Versus Hyperlactatemia in Septic Patients and Impact of Treatment Sepsis | Completed | Menoufia University | 60 | 2023-07-01 | 2024-12-06 | |
| An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics Carglumic Acid - Hyperammonemia, Methylmalonic Acidemia | Recruiting | RECORDATI GROUP | 20 | 2022-06-30 | 2025-04-15 | |
| A Healthy Volunteer Study to Compare Fezagepras (PBI-4050) With Sodium Phenylbutyrate Fezagepras, Sodium phenylbutyrate - Hyperammonemia | Phase 1 | Terminated | Liminal BioSciences Ltd. | 8 | 2022-05-13 | 2022-08-01 |
| PK Profile and Preliminary Efficacy of TNP-2092 Capsules in Liver Cirrhosis Patients With Hyperammonemia TNP-2092 capsules, Placebo - Hyperammonemia | Phase 1 / Phase 2 | Completed | TenNor Therapeutics (Suzhou) Limited | 36 | 2020-08-27 | 2025-04-25 |
| Monitoring the Hyperammonaemia:TOXicity of Drugs (AmmoTOX) Drugs inducing hyperammonemia - Hyperammonemia, Metabolic Disease | Completed | Groupe Hospitalier Pitie-Salpetriere | 2700 | 2019-05-01 | 2023-04-05 | |
| Vitamin E Supplementation in Hyperinsulinism/Hyperammonemia Syndrome Vitamin E - Hyperinsulinism-Hyperammonemia Syndrome | Not applicable | Completed | Elizabeth A Rosenfeld | 14 | 2019-04-15 | 2022-11-25 |
| A Safety and Efficacy Study of Infusions of HepaStem in Urea Cycle Disorders Pediatric Patients HepaStem Infusion - Urea Cycle Disorder | Phase 2 | Withdrawn | HLB Cell Co., Ltd. | 2018-07-12 | 2020-11-06 | |
| International Registry of Congenital Portosystemic Shunt (IRCPSS) Shunt Closure - Congenital Portosystemic Shunt, CPSS (Congenital Portosystemic Shunt) | Enrolling by invitation | Prof. Valérie Mc Lin | 500 | 2018-04-26 | 2025-06-18 | |
| A Phase 1 Study to Evaluate PK Profile and Food Effects on PK Parameters of TNP-2092 Capsules TNP-2092 capsules, TNP-2092 placebo capsules - Hyperammonemia | Phase 1 | Completed | TenNor Therapeutics (Suzhou) Limited | 58 | 2016-06-06 | 2025-04-25 |
| A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders RAVICTI - Urea Cycle Disorder | Phase 4 | Completed | Amgen | 27 | 2014-12-31 | 2024-07-01 |
| Phase 2B Efficacy/Safety of Ornithine Phenylacetate in Hospitalized Cirrhotic Patients With Hepatic Encephalopathy (STOP-HE) ornithine phenylacetate, placebo intravenous infusion - Acute Episode of Overt Hepatic Encephalopathy, Hepatic Encephalopathy | Phase 2 | Completed | Ocera Therapeutics, Inc. | 231 | 2014-01-07 | 2021-09-16 |
| Ammonia Levels and Cognitive Impairment Dementia, Cognitive Decline | Terminated | Duke University | 17 | 2014-01-01 | 2015-03-17 | |
| Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia Carbaglu, Placebo - Propionic Acidemia, Type I and/or Type II, Methylmalonic Acidemia | Phase 2 | Completed | Mendel Tuchman | 35 | 2012-09-01 | 2021-02-15 |
| 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 |
| 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 |
| Investigation of Brain Nitrogen in Partial Ornithine Transcarbamylase Deficiency (OTCD) Using 1 H MRS, DTI, and fMRI MRI scanning, Cognitive testing - Ornithine Transcarbamylase Deficiency | Completed | Andrea Gropman | 49 | 2010-09-01 | 2024-03-08 | |
| 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 |
Showing 20 of 25. Sign in to see all of them.
Urea Cycle Disorders, Inborn (10) · Ornithine Carbamoyltransferase Deficiency Disease (5) · Propionic Acidemia (4) · Brain Diseases (3) · Hypoglycemia (3) · Metabolic Diseases (3) · Metabolism, Inborn Errors (3) · Methylmalonic acidemia (3) · Amino Acid Metabolism, Inborn Errors (2) · Brain Diseases, Metabolic (2) · Brain Diseases, Metabolic, Inborn (2) · Carbamoyl-Phosphate Synthase I Deficiency Disease (2) · Central Nervous System Diseases (2) · Genetic Diseases, Inborn (2)
Pathologic Processes (25) · Pathological Conditions, Signs and Symptoms (25) · Nutritional and Metabolic Diseases (20) · Metabolic Diseases (17) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (16) · Nervous System Diseases (15) · Central Nervous System Diseases (13) · Genetic Diseases, Inborn (13) · Amino Acid Metabolism, Inborn Errors (12) · Brain Diseases (12) · Brain Diseases, Metabolic (12) · Metabolism, Inborn Errors (12) · Brain Diseases, Metabolic, Inborn (10) · Digestive System Diseases (4) · Neurologic Manifestations (4)
glycerol phenylbutyrate (6) · carglumic acid (3) · 4-phenylbutyric acid (2) · TNP-2092 (2) · Continuous Renal Replacement Therapy (1) · Cool-Down Exercise (1) · Cryotherapy (1) · Dialysis (1) · Glutamic Acid (1) · Glutamine (1) · Hypothermia, Induced (1) · N-carbamylglutamate (1) · Neuropsychological Tests (1) · Renal Dialysis (1) · Sodium Citrate (1)
Amgen (6) · Mendel Tuchman (2) · TenNor Therapeutics (Suzhou) Limited (2) · iECURE, Inc. (2) · Andrea Gropman (1) · Children's Mercy Hospital Kansas City (1) · Duke University (1) · Elizabeth A Rosenfeld (1) · Groupe Hospitalier Pitie-Salpetriere (1) · HLB Cell Co., Ltd. (1) · Liminal BioSciences Ltd. (1) · Menoufia University (1) · Nicola Longo (1) · Ocera Therapeutics, Inc. (1) · Prof. Valérie Mc Lin (1)
United States (18) · Canada (6) · France (4) · United Kingdom (4) · Australia (3) · Spain (3) · Belgium (2) · China (2) · Germany (2) · Hungary (2) · Israel (2) · Italy (2) · Netherlands (2) · Austria (1) · Bulgaria (1)
George Diaz, M.D., Ph.D (2) · Mendel Tuchman, MD (2) · Yanhua Ding, MD (2) · Andrea L Gropman, M.D. (1) · Chetanbabu M Patel, MD (1) · Elizabeth Rosenfeld, MD (1) · Nicholas Ah Mew, MD (1) · Nicola Longo, MD, PhD (1) · Sami Omar (1) · Sanghoon Lee, MD. Ph.D (1) · Uta Lichter-Konecki, MD, PhD (1)
PMID 24144944, PMID 22961727, PMID 23324524, PMID 28712602, PMID 28916119, PMID 30217721, PMID 33006765, PMID 33069881, PMID 34786649, PMID 35238476
2016 (1), 2018 (1), R01HD058567 (1), R21DK077415 (1), T32DK063688 (1)