62 completed, 17 recruiting, 14 terminated, 7 active not recruiting. 24 Phase 2, 21 Not applicable, 19 Phase 1, 10 Phase 3, 4 Phase 4.
Follow Urea Cycle Disorders, Inborn: new trials by email Literature Review of Urea Cycle Disorders, Inborn trials Research Report on Urea Cycle Disorders, Inborn
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 |
|---|---|---|---|---|---|---|
| Outside the Cage (OTC) Robotic Esophagectomy OTC MIE Esophagectomy - Esophageal Cancer | Phase 1 | Not yet recruiting | Centre hospitalier de l'Université de Montréal (CHUM) | 31 | 2027-01-01 | 2026-03-18 |
| 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 |
| Functional Evaluation in Patients With Urea Cycle Disorders (UCD) During a Driving Task Urea Cycle Disorder | Recruiting | St. Jude Children's Research Hospital | 60 | 2026-07-20 | 2026-07-31 | |
| 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 |
| Looking for VUS to Confirm Dominant Wolfram-like Syndrome Instead of Recessive Wolfram Syndrome Optical Coherence Tomography (OCT) of the Retina, Mutation | Active not recruiting | Hôpital Necker-Enfants Malades | 45 | 2026-03-08 | 2026-03-20 | |
| Epiretinal Membrane in Patients With DR. OCT - Epiretinal Membrane | Not yet recruiting | Assiut University | 225 | 2026-01-01 | 2025-11-21 | |
| A Clinical Study of Glycerol Phenylbutyrate in Chinese Patients With Urea Cycle Disorders Glycerol phenylbutyrate Oral Liquid - Urea Cycle Disorders | Recruiting | Tongji Hospital | 40 | 2025-12-09 | 2026-04-27 | |
| At Home Ammonia Monitoring of Inborn Errors of Ammonia Metabolism Ammonia Study Device - Urea Cycle Disorders, Organic Acidemias | Not applicable | Recruiting | Sequitur Health Corp. | 30 | 2025-09-23 | 2026-05-27 |
| Role of OCT & OCT Angiography in Patients With Posterior at the Uveitis Clinic of Assiut University Hospital. Uveitis, Posterior | Not yet recruiting | Assiut University | 25 | 2025-05-01 | 2025-04-21 | |
| Visual (Path)Ways in Multiple Sclerosis - Part II Measurement of retinal vascular density and visual cognition - Multiple Sclerosis, Optic Neuritis, Demyelinating Disease | Not applicable | Completed | University Hospital, Lille | 64 | 2025-01-24 | 2026-05-15 |
| 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 | 2026-08-31 | |
| MyRareDiet A Novel Diet Tracking Tool MDR diet assessment, 24 hour recall - Urea Cycle Disorder, Propionic Aciduria | Not applicable | Not yet recruiting | Oregon Health and Science University | 60 | 2024-11-15 | 2024-10-30 |
| A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD ARCT-810 - OTC Deficiency, Ornithine Transcarbamylase Deficiency | Phase 2 | Recruiting | Arcturus Therapeutics, Inc. | 9 | 2024-11-04 | 2025-03-17 |
| 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 |
| 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 |
| Study of CMP-CPS-001 in Healthy Volunteers and Participants With Abnormal Heterozygous OTC Genotype CMP-CPS-001, Placebo - Healthy Volunteers, OTC Deficiency | Phase 1 | Active not recruiting | CAMP4 Therapeutics Corporation | 120 | 2024-02-05 | 2026-04-22 |
| Effect of Fixed Combination Citicoline Homotaurine and Pyrroloquinoline Quinone on Pattern-electroretinogram in Glaucoma Citicoline 500 mg plus Homotaurine 50 mg plus Pyrroloquinoline quinone (Neuprozin Mito® ) - Glaucoma, Neuroprotection | Phase 3 | Completed | Fondazione IRCCS Policlinico San Matteo di Pavia | 40 | 2024-01-27 | 2025-05-01 |
| Chuvashia Inappropriate Prescribing Study Multimorbidity, Cardiovascular Diseases | Completed | National Medical Research Center for Therapy and Preventive Medicine | 300 | 2023-12-12 | 2025-06-10 | |
| Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEn AAVLK03hOTC - Ornithine Transcarbamylase Deficiency | Phase 1 / Phase 2 | Recruiting | University College, London | 12 | 2023-11-01 | 2023-11-07 |
Showing 20 of 114. Sign in to see all of them.
Ornithine Carbamoyltransferase Deficiency Disease (59) · Hyperargininemia (17) · Argininosuccinic Aciduria (12) · Citrullinemia (12) · Hyperammonemia (12) · Metabolism, Inborn Errors (11) · Carbamoyl-Phosphate Synthase I Deficiency Disease (9) · Amino Acid Metabolism, Inborn Errors (7) · Methylmalonic acidemia (7) · Propionic Acidemia (7) · N-acetyl glutamate synthetase deficiency (6) · Crigler-Najjar Syndrome (5) · Maple Syrup Urine Disease (5) · Rare Diseases (5)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (114) · Nutritional and Metabolic Diseases (114) · Brain Diseases, Metabolic (112) · Central Nervous System Diseases (112) · Nervous System Diseases (112) · Brain Diseases (111) · Genetic Diseases, Inborn (111) · Brain Diseases, Metabolic, Inborn (110) · Metabolic Diseases (110) · Amino Acid Metabolism, Inborn Errors (107) · Metabolism, Inborn Errors (103) · Genetic Diseases, X-Linked (58) · Pathological Conditions, Signs and Symptoms (26) · Pathologic Processes (23)
glycerol phenylbutyrate (10) · 4-phenylbutyric acid (6) · Adrenal Cortex Hormones (2) · Arginine (2) · Neuropsychological Tests (2) · Sodium Benzoate (2) · carglumic acid (2) · Acetaminophen (1) · Acetates (1) · Acupuncture Therapy (1) · Aging (1) · Amino Acids (1) · Angiography (1) · Angioplasty (1) · Bentonite (1)
Amgen (10) · Baylor College of Medicine (7) · Ultragenyx Pharmaceutical Inc (6) · Arcturus Therapeutics, Inc. (4) · Cellaion SA (4) · Cytonet GmbH & Co. KG (4) · Andrea Gropman (3) · Assiut University (3) · Children's National Research Institute (3) · Immedica Pharma AB (3) · Abbott Medical Devices (2) · Aeglea Biotherapeutics (2) · Kaleido Biosciences (2) · National Center for Research Resources (NCRR) (2) · Nicholas Ah Mew (2)
United States (60) · Canada (18) · United Kingdom (15) · France (13) · Spain (12) · Italy (11) · Belgium (9) · Germany (9) · Switzerland (8) · Australia (6) · Austria (4) · China (4) · Netherlands (4) · Portugal (4) · Brazil (3)
Nicholas Ah Mew, MD (4) · Mattias Rudebeck, PhD MSc BMedSc (3) · Andrea Gropman, MD (2) · Brendan Lee, MD, PhD (2) · George Diaz, M.D., Ph.D (2) · Lindsay Burrage, MD, PhD (2) · Mendel Tuchman, MD (2) · Sandesh CS Nagamani, MD, FACMG (2) · Adam Wax, Ph.D. (1) · Ahmed Abdel-Galeel, MD (1) · Andrea Gropman, M.D. (1) · Andrea L Gropman, M.D. (1) · Andrea L. Gropman, M.D. (1)
PMID 24144944, PMID 22961727, PMID 20188616, PMID 33325055, PMID 12050336, PMID 12736767, PMID 14523139, PMID 14724301, PMID 17135281, PMID 17145250, PMID 17280537, PMID 17296823, PMID 17296824, PMID 18307999, PMID 18343349, PMID 18414145, PMID 18562231, PMID 18662894, PMID 18765162, PMID 19287347, PMID 19660971, PMID 19709765, PMID 19737697, PMID 19737719, PMID 19758877, PMID 19889649, PMID 20004862, PMID 20338795, PMID 20488904, PMID 20512995, PMID 20554978, PMID 20566609, PMID 21459430, PMID 21482610, PMID 21629569, PMID 22110002, PMID 22789058, PMID 22901741, PMID 23324524, PMID 23996244, PMID 24084106, PMID 24485820, PMID 24758510, PMID 24881970, PMID 25135652, PMID 25712381, PMID 26067829, PMID 26242713, PMID 27132782, PMID 27215558
U54HD061221 (3), 2016 (2), 2018 (2), U54RR019453 (2), 2015 (1), 2025 (1), R01HD058567 (1), R21TR003166 (1), R44HD107766 (1), R44HD112243 (1)