Clinical Trials on Aromatic L-Amino Acid Decarboxylase Deficiency in United States
Total 51 results
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Krzysztof BankiewiczNational Institute of Neurological Disorders and Stroke (NINDS); University...Recruiting
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Origin BiosciencesCompletedMolybdenum Cofactor Deficiency | Isolated Sulfite Oxidase DeficiencySpain, Israel, United States, United Kingdom, Canada, Netherlands, Germany, Saudi Arabia, Tunisia, Japan, Italy, Malaysia, Poland, Turkey
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Baylor Research InstituteNational Institutes of Health (NIH)CompletedHyperhomocysteinemiaUnited States
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University of West LondonCompletedVitamin B12 DeficiencyUnited Kingdom
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National Institute of Neurological Disorders and...CompletedSuccinic Semialdehyde Dehydrogenase Deficiency | SSADH DeficiencyUnited States
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Ultragenyx Pharmaceutical IncCompletedOrnithine Transcarbamylase DeficiencyUnited States
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National Institute of Neurological Disorders and...CompletedSuccinic Semialdehyde | Dehydrogenase Deficiency | DiseasesUnited States
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Ultragenyx Pharmaceutical IncTerminatedOrnithine Transcarbamylase DeficiencyUnited States
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Homology Medicines, IncTerminatedPhenylketonuria | Phenylketonurias | PAH DeficiencyUnited States
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Homology Medicines, IncEnrolling by invitationPhenylketonuria | PAH DeficiencyUnited States
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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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Arcturus Therapeutics, Inc.Active, not recruitingOrnithine Transcarbamylase DeficiencyUnited States
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Ultragenyx Pharmaceutical IncActive, not recruitingOrnithine Transcarbamylase (OTC) DeficiencyCanada, United States, France, Spain, United Kingdom
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PTC TherapeuticsCompletedBH4 Deficiency | HyperphenylalaninemiaUnited States
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Pamlab, Inc.Clionsky Neuro Systems Inc.UnknownMild Cognitive ImpairmentUnited States
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Baylor College of MedicineCompletedCitrullinemiaUnited 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 College, LondonRecruitingOrnithine Transcarbamylase DeficiencyUnited Kingdom
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Arcturus Therapeutics, Inc.RecruitingOTC Deficiency | Ornithine Transcarbamylase Deficiency | OTCDSpain, Belgium, United Kingdom, Sweden
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Andrea GropmanNational Center for Research Resources (NCRR); Rare Diseases Clinical Research... and other collaboratorsCompletedBrain Diseases, Metabolic, Inborn | Ornithine Transcarbamylase Deficiency | Urea Cycle DisorderUnited States
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Aeglea BiotherapeuticsCompletedArginase I Deficiency | HyperargininemiaUnited States, Italy, Canada, France, United Kingdom, Austria, Germany
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Ultragenyx Pharmaceutical IncRecruitingOTC DeficiencyUnited States, Portugal, Australia, Italy, Spain, United Kingdom, Japan, Canada, Germany, France, Argentina, Netherlands, Brazil
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Baylor College of MedicineCompletedUrea Cycle Disorders | Argininosuccinic Aciduria | Argininosuccinate Lyase DeficiencyUnited States
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University of Massachusetts, AmherstWithdrawnOxidative Stress | COPD | Tetrahydrobiopterin DeficiencyUnited States
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Homology Medicines, IncTerminated
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Aeglea BiotherapeuticsCompletedArginase I Deficiency | HyperargininemiaUnited States, Canada, United Kingdom, Portugal
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Travere Therapeutics, Inc.Recruiting
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Baylor College of MedicineChildren's Hospital of Philadelphia; Children's Hospital Colorado; Seattle Children... and other collaboratorsRecruitingOrnithine Transcarbamylase Deficiency | Urea Cycle Disorder | Argininosuccinic Aciduria | Hyperargininemia | Citrullinemia 1 | ARGI Deficiency | ASL Deficiency | ASS DeficiencyUnited States
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metaX Institut fuer Diatetik GmbHBirmingham Children's HospitalCompletedPhenylketonurias | Hyperphenylalaninaemia | Tetrahydrobiopterin DeficiencyUnited Kingdom
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Aeglea BiotherapeuticsCompletedArginase I Deficiency | HyperargininemiaUnited States, Canada, United Kingdom, Portugal
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iECURE, Inc.RecruitingUrea Cycle Disorders, Inborn | Ornithine Transcarbamylase Deficiency | Ornithine Transcarbamylase Deficiency Disease | Ornithine Carbamoyltransferase Deficiency (Disorder)United Kingdom
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Baylor College of MedicineRare Diseases Clinical Research Network; Neogenis LaboratoriesCompletedUrea Cycle Disorders, Inborn | Urea Cycle Disorder | Argininosuccinic Aciduria | Argininosuccinate Lyase DeficiencyUnited States
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BioMarin PharmaceuticalCompletedTetrahydrobiopterin Deficiencies | Hyperphenylalaninemia, Non-PhenylketonuricGermany, United States
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Aeglea BiotherapeuticsTerminatedHomocystinuria Due to Cystathionine Beta-Synthase DeficiencyUnited States, United Kingdom, Australia
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Travere Therapeutics, Inc.RecruitingNatural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)Homocystinuria Due to CBS DeficiencyIreland, Qatar, United States, United Kingdom
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Georgetown UniversityCompletedEnd Stage Renal Disease | HyperhomocysteinemiaUnited States
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Ultragenyx Pharmaceutical IncTerminatedWilson Disease | Ornithine Transcarbamylase Deficiency | Glycogen Storage Disease Type IASpain, United Kingdom, Canada, United States, Brazil
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National Human Genome Research Institute (NHGRI)RecruitingHermansky-Pudlak Syndrome (HPS)United States
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National Human Genome Research Institute (NHGRI)RecruitingPulmonary Fibrosis | Healthy Volunteers | Hermansky-Pudlak Syndrome (HPS)United States
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William Gahl, M.D.National Human Genome Research Institute (NHGRI)CompletedPulmonary Fibrosis | Inborn Errors of Metabolism | Albinism | Oculocutaneous Albinism | Platelet Storage Pool DeficiencyUnited States
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National Institute of Allergy and Infectious Diseases...WithdrawnColitis | Cytokines | Hermanski-Pudlak Syndrome | Lymphocytes | Drug EvaluationUnited States
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National Human Genome Research Institute (NHGRI)TerminatedPulmonary Fibrosis | Metabolic Disease | Oculocutaneous Albinism | Hermansky-Pudlak Syndrome (HPS) | Platelet Storage Pool DeficiencyUnited States
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Jesse RomanGenentech, Inc.UnknownInterstitial Lung Disease | Hermansky Pudlak SyndromeUnited States, Puerto Rico
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Vanderbilt UniversityNational Heart, Lung, and Blood Institute (NHLBI); University of South Florida and other collaboratorsCompletedHermansky Pudlak SyndromeUnited States
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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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Mayo ClinicRecruitingCystinuria | Hyperoxaluria | Dent Disease | Lowe Syndrome | Adenine Phosphoribosyltransferase DeficiencyUnited States, Canada, Iceland, Israel
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National Human Genome Research Institute (NHGRI)CompletedCancer | Myelofibrosis | Pulmonary Fibrosis | Gaucher Disease | Hermansky-Pudlak Syndrome (HPS)United States
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Sanford HealthNational Ataxia Foundation; Beyond Batten Disease Foundation; Pitt Hopkins Research... and other collaboratorsRecruitingMitochondrial Diseases | Retinitis Pigmentosa | Myasthenia Gravis | Eosinophilic Gastroenteritis | Multiple System Atrophy | Leiomyosarcoma | Leukodystrophy | Anal Fistula | Spinocerebellar Ataxia Type 3 | Friedreich Ataxia | Kennedy Disease | Lyme Disease | Hemophagocytic Lymphohistiocytosis | Spinocerebellar Ataxia... and other conditionsUnited States, Australia