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Clinical Trials on Aromatic L-Amino Acid Decarboxylase Deficiency
Total 112 results
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Translate Bio, Inc.WithdrawnOrnithine Transcarbamylase Deficiency
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Tufts UniversityNational Heart, Lung, and Blood Institute (NHLBI)CompletedHeart Diseases | Cardiovascular Diseases | Hyperhomocysteinemia
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Bangkok Metropolitan Administration Medical College...CompletedCardiovascular Disease | Hyperhomocysteinemia
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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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PTC TherapeuticsCompletedBH4 Deficiency | HyperphenylalaninemiaUnited States
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Homology Medicines, IncTerminatedPhenylketonuria | Phenylketonurias | PAH DeficiencyUnited States
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Homology Medicines, IncTerminated
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Taipei Veterans General Hospital, TaiwanBioMarin PharmaceuticalUnknownPAH Deficiency | Pku Phenylketonuria
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metaX Institut fuer Diatetik GmbHBirmingham Children's HospitalCompletedPhenylketonurias | Hyperphenylalaninaemia | Tetrahydrobiopterin DeficiencyUnited Kingdom
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Homology Medicines, IncEnrolling by invitationPhenylketonuria | PAH DeficiencyUnited States
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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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Ultragenyx Pharmaceutical IncTerminatedOrnithine Transcarbamylase DeficiencyUnited States
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Cytonet GmbH & Co. KGCompletedOrnithine Transcarbamylase Deficiency | Urea Cycle Disorders | Citrullinemia | Carbamoylphosphate Synthetase I DeficiencyGermany
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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 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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Aeglea BiotherapeuticsCompletedArginase I Deficiency | HyperargininemiaUnited States, Canada, United Kingdom, Portugal
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Aeglea BiotherapeuticsCompletedArginase I Deficiency | HyperargininemiaUnited States, Canada, United Kingdom, Portugal
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Orphanetics Pharma Entwicklungs GmbHTerminatedPhenylalanine Hydroxylase Deficiencies
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Dr. Loges & Co. GmbHBioTeSys GmbHCompletedEndothelial Dysfunction | Hyperhomocysteinemia | Hypertension Grade I, Subgroup "Borderline" (WHO)Germany
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Pamlab, Inc.Clionsky Neuro Systems Inc.UnknownMild Cognitive ImpairmentUnited States
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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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BioMarin PharmaceuticalCompletedTetrahydrobiopterin Deficiencies | Hyperphenylalaninemia, Non-PhenylketonuricGermany, United States
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Baylor College of MedicineCompletedCitrullinemiaUnited States
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Andrea GropmanChildren's National Research InstituteCompletedOrnithine Transcarbamylase Deficiency
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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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BioMarin PharmaceuticalCompletedHyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) DeficiencySweden, France, Italy, Spain, Germany, Netherlands, Austria, Slovakia, Portugal
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Travere Therapeutics, Inc.Recruiting
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Hospital de Clinicas de Porto AlegreFundação de Amparo à Pesquisa do Estado do Rio Grande do Sul, BrazilSuspendedCBS DeficiencyBrazil
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Baylor College of MedicineCompletedUrea Cycle Disorders | Argininosuccinic Aciduria | Argininosuccinate Lyase DeficiencyUnited States
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Ludwig-Maximilians - University of MunichCompletedPhenylketonuria | Nutritional DeficienciesGermany
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Assiut UniversityCompletedCognitive Dysfunction | Hypercholesterolemia | HyperhomocysteinemiaEgypt
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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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University of Massachusetts, AmherstWithdrawnOxidative Stress | COPD | Tetrahydrobiopterin DeficiencyUnited States
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Central Hospital, Nancy, FranceNot yet recruitingAlzheimer Disease | Homocystinemia
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Universidade Estadual de LondrinaCompletedCardiovascular Disease | Hemodialysis | Chronic Renal Failure | Uremia | HyperhomocysteinemiaBrazil
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Hospital Universitario Dr. Jose E. GonzalezLaboratorios Valdecasas S.A.CompletedDiabetic Nephropathies | HyperhomocysteinemiaMexico
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Aeglea BiotherapeuticsTerminatedHomocystinuria Due to Cystathionine Beta-Synthase DeficiencyUnited States, United Kingdom, Australia
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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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Georgetown UniversityCompletedEnd Stage Renal Disease | HyperhomocysteinemiaUnited States
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Istanbul Medeniyet UniversityCompletedEpilepsy | Ischemic Stroke | Hyperhomocysteinemia, Thrombotic, Cbs-RelatedTurkey
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Shenzhen Ausa Pharmed Co.,LtdNanfang Hospital of Southern Medical UniversityWithdrawn
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University Children's Hospital, ZurichCompletedPhenylketonurias | OTC Deficiency | Inborn Errors of Metabolism | Urea Cycle Disorder | Maple Syrup Urine Disease | Methylmalonicacidemia | Aminoacidopathy | Patient Reported Outcome MeasurementsSwitzerland
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Bezmialem Vakif UniversityCompleted
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University of WashingtonNational Heart, Lung, and Blood Institute (NHLBI)CompletedMyocardial Infarction | Heart Diseases | Cardiovascular Diseases | Coronary Disease | Cerebrovascular Accident | Hyperhomocysteinemia
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National Heart, Lung, and Blood Institute (NHLBI)CompletedMyocardial Infarction | Heart Diseases | Cardiovascular Diseases | Cerebrovascular Disorders | Peripheral Vascular Diseases | Atherosclerosis | Hyperhomocysteinemia
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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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Ultragenyx Pharmaceutical IncTerminatedWilson Disease | Ornithine Transcarbamylase Deficiency | Glycogen Storage Disease Type IASpain, United Kingdom, Canada, United States, Brazil
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Centre Hospitalier Universitaire de LiegeSanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Régional... 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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National Human Genome Research Institute (NHGRI)RecruitingHermansky-Pudlak Syndrome (HPS)United States
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Vanderbilt UniversityNational Heart, Lung, and Blood Institute (NHLBI); University of South Florida and other collaboratorsCompletedHermansky Pudlak SyndromeUnited States