11 completed, 4 terminated, 3 recruiting, 2 not yet recruiting. 4 Not applicable, 3 Phase 2, 2 Phase 1, 2 Phase 4.
| Phase | Trials | Status |
|---|---|---|
| Not applicable | 4 | Completed 3, Not yet recruiting 1 |
| Phase 2 | 3 | Terminated 2, Withdrawn 1 |
| Phase 1 | 2 | Completed 1, Terminated 1 |
| Phase 4 | 2 | Completed 2 |
Follow Urea Cycle Disorder: new trials by email Literature Review of Urea Cycle Disorder trials Research Report on Urea Cycle Disorder
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 |
|---|---|---|---|---|---|---|
| MENA/Levant Epidemiological, Registry of Immunological & Clinical Traits (MERIT) Antiphospholipid (aPL)-Positive, Rheumatic Arthritis | Not yet recruiting | Axiom Bioregistry | 5000 | 2026-10-18 | 2026-10-23 | |
| 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 | |
| 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 |
| Dysregulated Urea-synthesis at Terminal Uremia Aminoacid, alanine-infusion - Urea Cycle Disorder, Uremia | Not applicable | Completed | University of Aarhus | 10 | 2022-03-01 | 2026-05-04 |
| Hepatic Histopathology in Urea Cycle Disorders Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency | Recruiting | Baylor College of Medicine | 70 | 2022-02-24 | 2025-07-20 | |
| Characterization of Intestinal Microbiota in Children With Inborn Errors of Metabolism (IEM) Inborn Errors of Metabolism, Propionic Aciduria | Completed | Emma Marie Caroline Slack | 22 | 2021-11-30 | 2024-03-01 | |
| 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 | |
| Th1, Th2, Th17 Phenotype in Urea Cycle Disorders Urea Cycle Disorder, Lysinuric Protein Intolerance | Completed | Istanbul University - Cerrahpasa | 65 | 2021-04-06 | 2023-09-28 | |
| 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 | |
| UNLOCKED: A Phase 2, Open-label Trial With KB195 in Subjects With a Urea Cycle Disorder KB195 - Urea Cycle Disorder | Phase 2 | Terminated | Kaleido Biosciences | 13 | 2019-09-17 | 2022-01-25 |
| Patient and Observer Reported Outcome Measurements in Inborn Errors of Metabolism Survey - Inborn Errors of Metabolism, Urea Cycle Disorder | Completed | University Children's Hospital, Zurich | 69 | 2019-08-01 | 2020-11-04 | |
| 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 | |
| HepaStem Long-Term Safety Registry Urea Cycle Disorder, Crigler-Najjar Syndrome | Terminated | Cellaion SA | 44 | 2018-04-04 | 2024-05-29 | |
| Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs) RAVICTI, NaPBA - Urea Cycle Disorder | Phase 4 | Completed | Amgen | 16 | 2018-02-20 | 2024-07-01 |
| Chronic Liver Disease in Urea Cycle Disorders Diagnostic Ultrasound - Urea Cycle Disorder | Completed | Baylor College of Medicine | 28 | 2017-11-29 | 2023-02-10 | |
| 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 |
| Safety and Tolerability of SYNB1020-CP-001 Placebo, SYNB1020 - Healthy Volunteer, Urea Cycle Disorder | Phase 1 | Completed | Synlogic | 52 | 2017-06-16 | 2021-05-12 |
| Manipulating the Gut Microbiome Study Acetohydroxamic Acid Oral Tablet, No treatment - Urea Cycle Disorder | Phase 1 / Phase 2 | Terminated | Nicholas Ah Mew | 4 | 2017-03-24 | 2021-12-15 |
| 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 |
| Observational Study That Will Collect Information on Patients With Urea Cycle Disorders (UCDs) Urea Cycle Disorder | Terminated | Amgen | 203 | 2013-09-25 | 2024-07-01 |
Showing 20 of 22. Sign in to see all of them.
Urea Cycle Disorders, Inborn (22) · Ornithine Carbamoyltransferase Deficiency Disease (8) · Argininosuccinic Aciduria (4) · Citrullinemia (3) · Hyperargininemia (3) · Maple Syrup Urine Disease (3) · Metabolism, Inborn Errors (3) · Methylmalonic acidemia (3) · Carbamoyl-Phosphate Synthase I Deficiency Disease (2) · Hyperammonemia (2) · Liver Diseases (2) · N-acetyl glutamate synthetase deficiency (2) · Propionic Acidemia (2) · Acute-On-Chronic Liver Failure (1) · Amino Acid Metabolism, Inborn Errors (1)
Brain Diseases (22) · Brain Diseases, Metabolic (22) · Central Nervous System Diseases (22) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (22) · Genetic Diseases, Inborn (22) · Metabolic Diseases (22) · Nervous System Diseases (22) · Nutritional and Metabolic Diseases (22) · Amino Acid Metabolism, Inborn Errors (21) · Brain Diseases, Metabolic, Inborn (21) · Metabolism, Inborn Errors (19) · Genetic Diseases, X-Linked (8) · Digestive System Diseases (4) · Pathological Conditions, Signs and Symptoms (3) · Female Urogenital Diseases (2)
glycerol phenylbutyrate (2) · 4-phenylbutyric acid (1) · Amino Acids (1) · High-Energy Shock Waves (1) · Surveys and Questionnaires (1) · acetohydroxamic acid (1)
Baylor College of Medicine (4) · Amgen (3) · Andrea Gropman (1) · Axiom Bioregistry (1) · Cellaion SA (1) · Children's National Research Institute (1) · Emma Marie Caroline Slack (1) · HLB Cell Co., Ltd. (1) · Istanbul University - Cerrahpasa (1) · Kaleido Biosciences (1) · Nicholas Ah Mew (1) · Nutricia Research (1) · Oregon Health and Science University (1) · St. Jude Children's Research Hospital (1) · Synlogic (1)
United States (14) · Switzerland (4) · Spain (3) · Belgium (2) · Turkey (Türkiye) (2) · Bulgaria (1) · Canada (1) · Denmark (1) · France (1) · Germany (1) · Italy (1) · Jordan (1) · Poland (1) · South Korea (1) · United Kingdom (1)
Lindsay Burrage, MD, PhD (2) · Nicholas Ah Mew, MD (2) · Andrea Gropman, M.D. (1) · Andrea Gropman, MD (1) · Ayca Kiykim (1) · Brendan Lee, M.D., PhD (1) · Dershung Yang, PhD (1) · Ljubica Caldovic, PhD (1) · Mark Wingertzahn, PhD (1) · Martina Huemer (1) · Sandesh C Nagamani, M.D. (1) · Sanghoon Lee, MD. Ph.D (1)
PMID 28916119, PMID 30217721, PMID 31990680, PMID 33846069
2015 (1), 2016 (1), R21TR003166 (1), R44HD107766 (1), U54RR019453 (1)