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Clinical Trials on GTP Cyclohydrolase I Deficiency in United States
Total 84 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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Prometic Biotherapeutics, Inc.CompletedHypoplasminogenemia | Type I Plasminogen DeficiencyUnited States
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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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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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National Cancer Institute (NCI)Completed
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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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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.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, Puerto Rico
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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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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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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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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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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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Masonic Cancer Center, University of MinnesotaCompletedMucopolysaccharidosis I | Mucopolysaccharidosis VI | Mannosidosis | Mucolipidosis Type II (I-cell Disease)United 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 Institute of Neurological Disorders and...Recruiting
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UniQure Biopharma B.V.Chiesi Farmaceutici S.p.A.Withdrawn
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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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KalVista Pharmaceuticals, Ltd.CompletedHereditary AngioedemaUnited States, Austria, Czechia, Germany, Hungary, Italy, Netherlands, North Macedonia, Poland, United Kingdom
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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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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 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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University of WashingtonNational Cancer Institute (NCI)CompletedPain | Arthralgia | Stage I Breast Cancer | Stage II Breast Cancer | Stage IIIA Breast Cancer | Stage IIIB Breast Cancer | Stage IIIC Breast Cancer | Recurrent Breast Cancer | Musculoskeletal ComplicationsUnited States
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Aeglea BiotherapeuticsCompletedArginase I Deficiency | HyperargininemiaUnited States, Italy, Canada, France, United Kingdom, Austria, Germany
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Fred Hutchinson Cancer CenterUniversity of WashingtonCompleted
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ViiV HealthcareGlaxoSmithKlineWithdrawnInfection, Human Immunodeficiency Virus IUnited States
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Nova Southeastern UniversityNational Institute of Mental Health (NIMH)CompletedHuman Immunodeficiency Virus I InfectionUnited States
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CSL BehringParexelCompletedHereditary Angioedema Types I and IIUnited States, Germany
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Vir Biotechnology, Inc.Bill and Melinda Gates FoundationCompletedHIV I InfectionUnited States
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University of Texas Southwestern Medical CenterCure SPG50RecruitingMicrocephaly | Spasticity, Muscle | Growth Retardation | Intellectual Deficiency | SPG50 | Spastic ParaplegiaUnited States
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Aeglea BiotherapeuticsCompletedArginase I Deficiency | HyperargininemiaUnited States, Canada, United Kingdom, Portugal
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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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Aeglea BiotherapeuticsCompletedArginase I Deficiency | HyperargininemiaUnited States, Canada, United Kingdom, Portugal
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National Institute of Neurological Disorders and...CompletedMultiple Sclerosis | Vasculitis | Tropical Spastic Paraparesis | HTLV-I Infection | Herpesviridae InfectionUnited States
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George Washington UniversityCompletedInfluenza, Human | Human Immunodeficiency Virus I InfectionUnited States
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CSL BehringCompletedHereditary Angioedema Types I and IIUnited States, Spain, Germany, Australia, Canada, Czechia, Hungary, Israel, Italy, Romania, United Kingdom
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University of PittsburghRecruitingLysosomal Storage Diseases | Leukodystrophy | Niemann-Pick Diseases | Batten Disease | Gaucher Disease | Krabbe Disease | Alpha-Mannosidosis | Osteopetrosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | GM1 Gangliosidoses | MPS I | MPS II | Vanishing White Matter Disease | Multiple Sulfatase Deficiency... and other conditionsUnited States
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CSL BehringCompletedHereditary Angioedema Types I and IIUnited States, Spain, Australia, Canada, Czechia, Hungary, Israel, Italy, Romania, United Kingdom
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St. Jude Children's Research HospitalRecruitingMucopolysaccharidosis I | Severe Combined Immunodeficiency | Neuroblastoma | Neoplasm | Malignancy, HematologicUnited States
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Stealth BioTherapeutics Inc.Active, not recruitingMitochondrial Diseases | Mitochondrial Myopathies | Mitochondrial Complex I Deficiency | Mitochondrial Pathology | Mitochondrial DNA Depletion | Mitochondrial DNA Mutation | Mitochondrial DNA Deletion | Mitochondrial Metabolism DefectSpain, United States, Italy, Netherlands, Australia, Germany, Hungary, New Zealand, Norway, United Kingdom
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ViiV HealthcareCompletedHIV Infection | Infection, Human Immunodeficiency Virus IUnited States, Italy
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Inozyme PharmaIQVIA BiotechActive, not recruitingPseudoxanthoma Elasticum | Generalized Arterial Calcification of Infancy | ATP-Binding Cassette Subfamily C Member 6 DeficiencyUnited States, United Kingdom
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Fred Hutchinson Cancer CenterNational Cancer Institute (NCI); National Heart, Lung, and Blood Institute...WithdrawnStage III Adult Hodgkin Lymphoma | Stage IV Adult Hodgkin Lymphoma | Human Immunodeficiency Virus 1 Positive | Stage I Adult Hodgkin Lymphoma | Stage II Adult Hodgkin Lymphoma | Stage I Adult Non-Hodgkin Lymphoma | Stage II Adult Non-Hodgkin Lymphoma | Stage III Adult Non-Hodgkin Lymphoma | Stage IV...United States
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MacroGenicsNational Institute of Allergy and Infectious Diseases (NIAID); National Institutes... and other collaboratorsActive, not recruitingHuman Immunodeficiency Virus I Infection | Immunodeficiency Virus Type 1, Human | Human Immunodeficiency Virus Type 1United States
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Akcea TherapeuticsIonis Pharmaceuticals, Inc.CompletedFamilial Chylomicronemia Syndrome | Lipoprotein Lipase Deficiency | Hyperlipoproteinemia Type 1United States, Canada, Germany, Israel, Italy, South Africa, Spain, United Kingdom, Brazil, France, Netherlands
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ViiV HealthcareCompletedHIV Infection | Infection, Human Immunodeficiency Virus IUnited States