19 unknown. 19 Phase 4.
| Phase | Trials | Status |
|---|---|---|
| Phase 4 | 19 | Unknown 19 |
Follow Metabolism, Inborn Errors: new trials by email Literature Review of Metabolism, Inborn Errors trials Research Report on Metabolism, Inborn Errors
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 |
|---|---|---|---|---|---|---|
| Analysis of the Effect of Iron Supplements on Iron Deficiency Anemia in Pregnancy Niferex - Iron Deficiency Anemia of Pregnancy, Iron Storage Disease | Phase 4 | Unknown | Qianfoshan Hospital | 452 | 2023-03-28 | 2023-04-07 |
| Gandouling in the Treatment of Wilson's Disease Gandouling - Wilson's Disease | Phase 4 | Unknown | Jun Li | 240 | 2023-01-01 | 2024-02-09 |
| Comparisons of Different Forms of Glucocorticoid on the Recovery of Reproductive Function in Patients With 21α-hydroxylase Deficiency Hydrocortisone, Prednisone, Dexamethasone - Congenital Adrenal Hyperplasia | Phase 4 | Unknown | Shanghai Jiao Tong University School of Medicine | 120 | 2020-10-01 | 2020-09-02 |
| BH4 Responsiveness in PAH Deficiency PKU Patients BH4 - Pku Phenylketonuria, PAH Deficiency | Phase 4 | Unknown | Taipei Veterans General Hospital, Taiwan | 40 | 2020-06-01 | 2020-01-13 |
| Safety and Efficacy of Aspirin in Stroke Patients With Glucose-6-phosphate Dehydrogenase Deficiency (SAST) Aspirin, Clopidogrel - G6PD Deficiency, Stroke | Phase 4 | Unknown | First Affiliated Hospital, Sun Yat-Sen University | 440 | 2020-01-22 | 2022-03-16 |
| Effects of Evolocumab Versus Placebo Added to Standard Lipid-lowering Therapy on Fasting and Post Fat Load Lipids in Patients With Familial Dysbetalipoproteinemia Evolocumab Auto-Injector [Repatha], Placebos - Familial Dysbetalipoproteinemia, Hyperlipoproteinemia Type III | Phase 4 | Unknown | UMC Utrecht | 30 | 2019-08-01 | 2019-01-22 |
| Chronic Hepatitis C Treatment in Egyptian Children With Gaucher Disease. Ledipasvir/Sofosbuvir - Gaucher Disease, HCV | Phase 4 | Unknown | Mansoura University Children Hospital | 10 | 2018-04-03 | 2018-10-30 |
| Burosumab in Children and Adolescents With X-linked Hypophosphatemia Burosumab Injection - Rare Diseases, X-linked Hypophosphatemia | Phase 4 | Unknown | Bicetre Hospital | 57 | 2018-03-18 | 2020-06-05 |
| The Impact of Urate-lowering Therapy on Kidney Function (IMPULsKF) Febuxostat 80 MG Oral Tablet or Allopurinol 300 MG Oral Tablet - Gout, CKD Stage 1-4 | Phase 4 | Unknown | Medical Practice Prof D. Ivanov | 2 | 2017-10-30 | 2022-08-24 |
| Using Different Doses of Active Vitamin D Combined With Neutral Phosphate in Children With X-linked Hypophosphatemia Calcitriol - X-linked Hypophosphatemia | Phase 4 | Unknown | Peking Union Medical College Hospital | 100 | 2017-01-01 | 2019-01-30 |
| Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine trientine dihydrochloride - Wilson Disease | Phase 4 | Unknown | Univar BV | 90 | 2016-01-01 | 2017-08-23 |
| Feasibility Study of the Intensive Systolic Blood Pressure Control Standard BP control, Moderate BP control, Intensive BP control - Hypertension, Hyperhomocysteinemia | Phase 4 | Unknown | Second Affiliated Hospital of Nanchang University | 100 | 2015-12-01 | 2016-10-14 |
| Immune Modulation Therapy for Pompe Disease Rituximab, intravenous immune globulin, Bortezomib - Pompe Disease | Phase 4 | Unknown | National Taiwan University Hospital | 8 | 2015-08-01 | 2016-04-15 |
| Efficacy of Yinzhihuang Oral Liquid on Indirect Bilirubin of Neonates With Glucose-6-phosphate Dehydrogenase Deficiency Yinzhihuang Oral Liquid - Neonatal Hyperbilirubinemia | Phase 4 | Unknown | Southern Medical University, China | 300 | 2015-07-01 | 2015-11-03 |
| CPAP for Infantile Pompe Disease continuous positive airway pressure - Pompe Disease | Phase 4 | Unknown | National Taiwan University Hospital | 3 | 2014-06-01 | 2015-04-01 |
| Autologous Cell Suspension Grafting Using ReCell in Vitiligo and Piebaldism Patients ReCell epidermal cell suspension grafting, CO2 laser abrasion + UV-therapy - Piebaldism, Segmental Vitiligo | Phase 4 | Unknown | Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) | 10 | 2012-06-01 | 2013-08-05 |
| Punchgrafting Techniques for Vitiligo punchgrafting - Non-segmental Vitiligo, Segmental Vitiligo | Phase 4 | Unknown | Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) | 35 | 2011-06-01 | 2011-06-21 |
| Triglyceride Lowering Study Omega-3-Acid Ethyl Esters - Hypertriglyceridemia | Phase 4 | Unknown | Jewish Hospital, Cincinnati, Ohio | 25 | 2009-07-01 | 2009-07-08 |
| Evaluation of Efficacy and Safety of Agalsidase Beta in Heterozygous Females for Fabry Disease recombinant alpha-galactosidase A - Fabry Disease | Phase 4 | Unknown | Assistance Publique - Hôpitaux de Paris | 34 | 2005-06-01 | 2007-06-18 |
Familial Hypophosphatemic Rickets (2) · Glucosephosphate Dehydrogenase Deficiency (2) · Glycogen Storage Disease Type II (2) · Hepatolenticular Degeneration (2) · Piebaldism (2) · Adrenal Hyperplasia, Congenital (1) · Cardiomyopathies (1) · Fabry Disease (1) · Gaucher Disease (1) · Gout (1) · Hemochromatosis (1) · Hemolysis (1) · Hyperbilirubinemia, Neonatal (1) · Hyperhomocysteinemia (1) · Hyperlipoproteinemia Type III (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (19) · Genetic Diseases, Inborn (19) · Metabolic Diseases (19) · Nutritional and Metabolic Diseases (19) · Brain Diseases (8) · Central Nervous System Diseases (8) · Nervous System Diseases (8) · Brain Diseases, Metabolic (7) · Brain Diseases, Metabolic, Inborn (7) · Metal Metabolism, Inborn Errors (5) · Amino Acid Metabolism, Inborn Errors (4) · Carbohydrate Metabolism, Inborn Errors (4) · Lipid Metabolism Disorders (4) · Lipid Metabolism, Inborn Errors (4)
Allopurinol (1) · Aspirin (1) · Bortezomib (1) · Calcitriol (1) · Clopidogrel (1) · Continuous Positive Airway Pressure (1) · Dexamethasone (1) · Febuxostat (1) · Hydrochlorothiazide (1) · Hydrocortisone (1) · Immunoglobulins, Intravenous (1) · Methotrexate (1) · Niferex (1) · Omacor (1) · Prednisone (1)
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) (2) · National Taiwan University Hospital (2) · Assistance Publique - Hôpitaux de Paris (1) · Bicetre Hospital (1) · First Affiliated Hospital, Sun Yat-Sen University (1) · Jewish Hospital, Cincinnati, Ohio (1) · Jun Li (1) · Mansoura University Children Hospital (1) · Medical Practice Prof D. Ivanov (1) · Peking Union Medical College Hospital (1) · Qianfoshan Hospital (1) · Second Affiliated Hospital of Nanchang University (1) · Shanghai Jiao Tong University School of Medicine (1) · Southern Medical University, China (1) · Taipei Veterans General Hospital, Taiwan (1)
China (7) · France (2) · Netherlands (2) · Taiwan (2) · Egypt (1) · Germany (1) · Greece (1) · Italy (1) · Ukraine (1) · United Kingdom (1) · United States (1)
Charles Glueck, MD (1) · Frank LJ Visseren, prof (1) · Jinsheng Zeng, MD,PhD (1) · Karl-Heinz Weiss, MD (1) · Weimin Huang, Ph.D (1) · Xiaoshu Cheng, MD (1) · Yin-Hsiu Chien (1) · Yin-Hsiu Chien, MD, PhD (1) · Zhang Zhiwei, PH.D (1)
PMID 32527283, PMID 35482910, PMID 38715621, PMID 40104015, PMID 40538652