Clinical Trials on GTP+Cyclohydrolase+I+Deficiency
Total 140 results
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University of UtahBioMarin PharmaceuticalCompletedGTP Cyclohydrolase DeficiencyUnited 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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Ultragenyx Pharmaceutical IncCompletedCarnitine Palmitoyltransferase (CPT I or CPT II) Deficiency | Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency | Long-chain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency | Trifunctional Protein (TFP) Deficiency | Carnitine-acylcarnitine Translocase (CACT) DeficiencyUnited States, United Kingdom
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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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Prometic Biotherapeutics, Inc.CompletedHypoplasminogenemia | Type I Plasminogen DeficiencyUnited States
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Cytonet GmbH & Co. KGCompletedOrnithine Transcarbamylase Deficiency | Urea Cycle Disorders | Citrullinemia | Carbamoylphosphate Synthetase I DeficiencyGermany
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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.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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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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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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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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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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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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Universidade Metodista de PiracicabaCompletedHTLV-I InfectionsBrazil
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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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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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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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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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National Cancer Institute (NCI)Completed
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University Hospital Center of MartiniqueLaboratoire CerbaCompletedHTLV-I InfectionsMartinique
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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, 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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Inozyme PharmaGACI GlobalNot yet recruitingATP-Binding Cassette Subfamily C Member 6 Deficiency | Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 Deficiency
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Aeglea BiotherapeuticsTerminatedArginase I Deficiency | HyperargininemiaCanada
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Aeglea BiotherapeuticsCompletedArginase I Deficiency | HyperargininemiaUnited States, Italy, Canada, France, United Kingdom, Austria, Germany
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UniQure Biopharma B.V.Chiesi Farmaceutici S.p.A.Withdrawn
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Université de SherbrookeInstitut de Recherches Cliniques de MontrealRecruitingLipoprotein Lipase DeficiencyCanada
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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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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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University of Colorado, DenverCompletedLipoprotein Lipase Deficiency
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University of CatanzaroUnknownAndrogen Receptor Abnormal | Insulin-Like Growth Factor I Deficiency | Insulin Receptor, Defect inItaly
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Masonic Cancer Center, University of MinnesotaCompletedMucopolysaccharidosis I | Mucopolysaccharidosis VI | Mannosidosis | Mucolipidosis Type II (I-cell Disease)United States
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National Institute of Neurological Disorders and...Recruiting
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Genzyme, a Sanofi CompanyCompletedCerebroside Lipidosis Syndrome | Glucosylceramide Beta-Glucosidase Deficiency Disease | Gaucher Disease Type I | Clucocerebrosidase Deficiency Disease | Gaucher Disease, Non-Neuronopathic FormUnited States
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Amsterdam Molecular TherapeuticsThe Clinical Trial CompanyUnknown
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Catabasis PharmaceuticalsCompletedHypertriglyceridemia | Hyperlipoproteinemia Type I | Lipoprotein Lipase Deficiency, Familial | Chylomicronemia, FamilialCanada
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Dr. Loges & Co. GmbHBioTeSys GmbHCompletedEndothelial Dysfunction | Hyperhomocysteinemia | Hypertension Grade I, Subgroup "Borderline" (WHO)Germany
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University of Alabama at BirminghamNational Institutes of Health (NIH); Mereo BioPharmaCompletedEmphysema or COPD | Alpha-1 Antitrypsin Deficiency (AATD) | Pi*ZZ, Pi*SZ, Pi*Null, Another Rare Phenotype/Genotype Known to be Associated With Either Low or Functionally Impaired AAT Including F or I MutationsUnited States
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Inozyme PharmaNot yet recruitingGeneralized Arterial Calcification of Infancy | Autosomal Recessive Hypophosphatemic Rickets | Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency | ATP-Binding Cassette Subfamily C Member 6 Deficiency
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National Institute of Neurological Disorders and...CompletedTropical Spastic Paraparesis | Spinal Cord Disease | HTLV-I InfectionUnited States
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NYU Langone HealthDyax Corp.WithdrawnHereditary Angioedema Types I and IIUnited States
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CSL BehringParexelCompletedHereditary Angioedema Types I and IIUnited States, Germany
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CSL BehringCompletedHereditary Angioedema Types I and IIUnited States, Spain, Germany, Australia, Canada, Czechia, Hungary, Israel, Italy, Romania, United Kingdom