Clinical Trials on Carbamoyl-Phosphate Synthase I Deficiency Disease
Total 152 results
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Children's Hospital of PhiladelphiaEunice Kennedy Shriver National Institute of Child Health and Human Development...WithdrawnUrea Cycle Disorders, Inborn | Inborn Errors of Metabolism | Propionic Acidemia | Methylmalonic Acidemia | Carbamyl Phosphate Synthetase Deficiency
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Baylor College of MedicineChildren's National Research InstituteRecruitingOrnithine Transcarbamylase Deficiency | Urea Cycle Disorder | Carbamyl Phosphate Synthetase Deficiency | Argininosuccinic Aciduria | Hyperargininemia | Citrullinemia 1 | ARGI Deficiency | ASL Deficiency | ASS Deficiency | NAGS DeficiencyUnited States
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Cytonet GmbH & Co. KGCompletedOrnithine Transcarbamylase Deficiency | Urea Cycle Disorders | Citrullinemia | Carbamoylphosphate Synthetase I DeficiencyGermany
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West Kazakhstan Medical UniversityRecruitingOrnithine Transcarbamylase Deficiency | Biotinidase Deficiency | Citrullinemia | Glutaric Acidemia Type II | Argininosuccinic Aciduria | Maple Syrup Urine Disease | Primary Carnitine Deficiency | Homocystinuria | Carnitine Palmitoyltransferase II Deficiency | Arginase Deficiency | Very Long-chain Acyl-CoA... and other conditionsKazakhstan
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National Human Genome Research Institute (NHGRI)TerminatedCitrullinemia | Argininosuccinic Aciduria | Hyperargininemia | Ornithine Carbamoyltransferase Deficiency | Carbamoyl-Phosphate Synthase I Deficiency | N-Acetylglutamate Synthase DeficiencyUnited States
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Mendel TuchmanChildren's Hospital of Philadelphia; University of California, Los Angeles; Icahn... and other collaboratorsCompletedMethylmalonic Acidemia | Carbamoyl-Phosphate Synthase I Deficiency Disease | Propionic Acidemia, Type I and/or Type II | Ornithine Carbamoyltransferase DeficiencyUnited States
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MedicureRecruitingPyridox(am)Ine 5'-Phosphate Oxidase DeficiencyUnited States, Australia
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DDC Clinic - Center for Special Needs ChildrenUnknownGM3 Synthase Deficiency
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University of Mississippi, OxfordCompletedMalaria | Glucose 6 Phosphate Dehydrogenase DeficiencyUnited States
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PATHUniversity of Oxford; Mahidol UniversityCompletedGlucose-6 Phosphate Dehydrogenase DeficiencyThailand
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PATHEijkman Institute for Molecular BiologyCompletedGlucose-6-Phosphate Dehydrogenase DeficiencyIndonesia
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Ain Shams UniversityUnknownGlucose-6-Phosphate Dehydrogenase DeficiencyEgypt
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University Hospital, Strasbourg, FranceUnknownThe Glutathione Synthetase DeficiencyFrance
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Nicholas Ah MewIcahn School of Medicine at Mount Sinai; Boston Children's HospitalRecruitingN-acetylglutamate Synthase (NAGS) DeficiencyUnited States
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Jens Rikardt AndersenSt. Olavs HospitalCompletedStarvation | Refeeding Syndrome | Phosphate Deficiency
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The First Hospital of Jilin UniversityNational Natural Science Foundation of ChinaUnknownGenetic Diseases, Inborn | Asparagine Synthetase DeficiencyChina
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Children's Memorial Health Institute, PolandDepartment of Internal Medicine, Hypertension and Vascular Diseases, The...Not yet recruitingGlucose 6 Phosphatase DeficiencyPoland
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Centre Hospitalier Régional de la CitadelleSanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Universitaire... and other collaboratorsRecruitingCongenital Adrenal Hyperplasia | Hemophilia A | Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II | Cystic Fibrosis | Alpha 1-Antitrypsin Deficiency | Sickle Cell Disease | Fanconi Anemia | Chronic Granulomatous Disease | Wilson Disease | Severe Congenital Neutropenia | Ornithine Transcarbamylase... and other conditionsBelgium
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Cliniques universitaires Saint-Luc- Université...UnknownGlucose 6 Phosphatase Deficiency | Glycogen Storage Disease Type IBelgium
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RTI InternationalEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationPrimary Hyperoxaluria Type 3 | Diabetes Mellitus | Hemophilia A | Hemophilia B | Hereditary Fructose Intolerance | Cystic Fibrosis | Factor VII Deficiency | Phenylketonurias | Sickle Cell Disease | Dravet Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Chronic Granulomatous Disease and other conditionsUnited States
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Catalyst BiosciencesTerminatedPrevious Diagnosis With a Complement-mediated Disease and/or With Clinical Manifestations Reasonably Associated With Complement Factor I DeficiencyUnited States
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University of SevilleRecruitingInsulin Growth Factor I Deficiency | IGF1 Deficiency | Executive Function DisorderSpain
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Ankara City Hospital BilkentRecruitingCovid19 | Mortality | Phosphate DeficiencyTurkey
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Prometic Biotherapeutics, Inc.CompletedHypoplasminogenemia | Type I Plasminogen DeficiencyUnited States
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Southern Medical University, ChinaUnknownNeonatal HyperbilirubinemiaChina
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First Affiliated Hospital, Sun Yat-Sen UniversityRecruitingStroke | G6PD DeficiencyChina
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National Institute of Neurological Disorders and...Completed
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Rocket Pharmaceuticals Inc.California Institute for Regenerative Medicine (CIRM)CompletedLeukocyte Adhesion Defect - Type ISpain, United States, United Kingdom
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Rocket Pharmaceuticals Inc.WithdrawnLeukocyte Adhesion Defect - Type ISpain
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Pharvaris Netherlands B.V.Active, not recruitingHereditary Angioedema | Hereditary Angioedema Type I | Hereditary Angioedema Type II | Hereditary Angioedema Types I and II | Hereditary Angioedema Attack | Hereditary Angioedema With C1 Esterase Inhibitor Deficiency | Hereditary Angioedema - Type 1 | Hereditary Angioedema - Type 2 | C1 Esterase Inhibitor... and other conditionsUnited States, Poland, Germany, Austria, Bulgaria, Canada, Ireland, Italy, United Kingdom
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Pharvaris Netherlands B.V.RecruitingHereditary Angioedema | Hereditary Angioedema Type I | Hereditary Angioedema Type II | Hereditary Angioedema Types I and II | Hereditary Angioedema Attack | Hereditary Angioedema With C1 Esterase Inhibitor Deficiency | Hereditary Angioedema - Type 1 | Hereditary Angioedema - Type 2 | C1 Esterase Inhibitor... and other conditionsUnited States
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Aeglea BiotherapeuticsTerminatedHomocystinuria Due to Cystathionine Beta-Synthase DeficiencyUnited States, United Kingdom, Australia
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Pharvaris Netherlands B.V.CompletedHereditary Angioedema | Hereditary Angioedema Type I | Hereditary Angioedema Type II | Hereditary Angioedema Types I and II | Hereditary Angioedema Attack | Hereditary Angioedema With C1 Esterase Inhibitor Deficiency | Hereditary Angioedema - Type 1 | Hereditary Angioedema - Type 2 | C1 Esterase Inhibitor... and other conditionsBulgaria, United States, Spain, Israel, Germany, Canada, Czechia, France, Hungary, Italy, Netherlands, Poland, United Kingdom
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Pharvaris Netherlands B.V.RecruitingHereditary Angioedema | Hereditary Angioedema Type I | Hereditary Angioedema Type II | Hereditary Angioedema Types I and II | Hereditary Angioedema Attack | Hereditary Angioedema With C1 Esterase Inhibitor Deficiency | Hereditary Angioedema - Type 1 | Hereditary Angioedema - Type 2 | C1 Esterase Inhibitor... and other conditionsUnited States, Bulgaria, Czechia, Hungary, Spain, France, Germany, Poland, Canada, Israel
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CENTOGENE GmbH RostockCompletedHereditary Angioedema | Hereditary Angioedema Type I | Hereditary Angioedema Type II | C1 Esterase Inhibitor Deficiency | HAE | Angio Edema | C4 Deficiency | Hereditary Angioedema Type IIITurkey, Armenia, Georgia, India, Peru, Poland, Romania
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Rigshospitalet, DenmarkUnknownCarbohydrate Metabolism, Inborn Errors | Metabolism, Inborn Errors | Lipid Metabolism, Inborn Errors | Glycogen Storage Disease Type II | Glycogen Storage Disease Type V | VLCAD Deficiency | Glycogen Storage Disease Type III | Phosphoglycerate Kinase Deficiency | Neutral Lipid Storage Disease | Carnitine... and other conditionsDenmark
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Universidade Metodista de PiracicabaCompletedHTLV-I InfectionsBrazil
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Great Ormond Street Hospital for Children NHS Foundation...National Institute for Health Research, United Kingdom; The Freya FoundationRecruitingPyruvate Dehydrogenase Complex Deficiency | Pyruvate Dehydrogenase E1 Alpha Deficiency | Pyruvate Dehydrogenase E1-Beta Deficiency | Pyruvate Dehydrogenase E2 Deficiency | Pyruvate Dehydrogenase Phosphatase DeficiencyUnited Kingdom
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Amsterdam Molecular TherapeuticsThe Clinical Trial CompanyCompletedFamilial Lipoprotein Lipase DeficiencyCanada
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Amsterdam Molecular TherapeuticsInternational Antiviral Therapy Evaluation CenterUnknownFamilial Lipoprotein Lipase DeficiencyCanada
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KalVista Pharmaceuticals, Ltd.TerminatedAngioedema, Hereditary, Types I and IIUnited States, Czechia, Germany, Hungary, Italy, North Macedonia, United Kingdom, Australia, Bulgaria, Canada, France, New Zealand, Puerto Rico
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HAE Global Registry FoundationRecruitingHereditary Angioedema Type I and IIItaly
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KalVista Pharmaceuticals, Ltd.CompletedHereditary AngioedemaUnited States, Austria, Czechia, Germany, Hungary, Italy, Netherlands, North Macedonia, Poland, United Kingdom
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Prothya BiosolutionsCompletedHereditary Angioedema Type I | Angioneurotic EdemaNetherlands
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University Hospital Center of MartiniqueLaboratoire CerbaCompletedHTLV-I InfectionsMartinique
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Travere Therapeutics, Inc.Recruiting
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MP Biomedicals, LLCMP Biomedicals Asia Pacific Pte. Ltd.CompletedHTLV-I Infections | HTLV-II Infections | Human T-lymphotropic Virus 1 | Human T-lymphotropic Virus 2 | HTLV I Associated T Cell Leukemia Lymphoma | HTLV I Associated MyelopathiesUnited States
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MP Biomedicals, LLCVital Systems Inc.UnknownHTLV-I Infections | HTLV-II Infections | Human T-lymphotropic Virus 1 | Human T-lymphotropic Virus 2 | HTLV I Associated T Cell Leukemia Lymphoma | HTLV I Associated MyelopathiesUnited States
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MP Biomedicals, LLCMP Biomedicals Asia Pacific Pte. Ltd.CompletedHTLV-I Infections | HTLV-II Infections | Human T-lymphotropic Virus 1 | Human T-lymphotropic Virus 2 | HTLV I Associated T Cell Leukemia Lymphoma | HTLV I Associated MyelopathiesUnited States
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National Cancer Institute (NCI)Completed