- ICH GCP
- US Clinical Trials Registry
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Clinical Trials on Brain Diseases, Metabolic
Total 380587 results
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University Children's Hospital, ZurichRecruitingUrea Cycle DisordersSwitzerland
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University Hospital, ToursCompletedAdult Phenylketonuria Non Treated PatientsFrance
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Swedish Orphan BiovitrumCompletedHereditary Tyrosinemia, Type IBelgium, Denmark, France, Germany, Austria, Czechia, Finland, Hungary, Ireland, Italy, Netherlands, Norway, Poland, Portugal, Spain, Sweden, United Kingdom
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Cycle Pharmaceuticals Ltd.ParexelCompletedHereditary Tyrosinemia, Type ISouth Africa
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CENTOGENE GmbH RostockWithdrawnMetabolic Disorders | Tyrosinosis | Hepatorenal Tyrosinemia | Fumarylacetoacetase Deficiency | Fah DeficiencyGermany, Sri Lanka, India
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CENTOGENE GmbH RostockCompletedLysosomal Storage Diseases | Sphingolipidoses | Gaucher DiseaseAlbania, Greece, India, Israel, Morocco, Pakistan, Spain
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Vitaflo International, LtdActive, not recruiting
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University of South FloridaNational Institutes of Health (NIH)CompletedUrea Cycle DisordersUnited States
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Cycle Pharmaceuticals Ltd.ParexelCompletedHereditary Tyrosinemia, Type ISouth Africa
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Swedish Orphan BiovitrumCompletedEfficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1 (HT-1)Hereditary Tyrosinemia, Type IBelgium, Denmark, France, Germany, Sweden
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Cytonet GmbH & Co. KGCompletedUrea Cycle DisordersGermany
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Wan-Jin ChenChang Gung Memorial Hospital; Taipei Veterans General Hospital, TaiwanRecruiting
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National Institute of Neurological Disorders and...CompletedGaucher's DiseaseUnited 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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Eunice Kennedy Shriver National Institute of Child...Completed
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The Hospital for Sick ChildrenCompletedGangliosidoses, GM2 | Tay-Sachs Disease | Sandhoff DiseaseCanada
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National Center for Research Resources (NCRR)National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); University...Completed
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University Hospital, Clermont-FerrandUnknown
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Baylor College of MedicineCompletedUrea Cycle Disorders, InbornUnited States
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Children's Hospital of PhiladelphiaCompletedMaple Syrup Urine DiseaseUnited States
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Promethera TherapeuticsCompletedUrea Cycle DisordersBelgium, Spain, France, Poland
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Kaleido BiosciencesCompletedUrea Cycle DisordersSwitzerland
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He HuangLingyi Biotech Co., Ltd.Recruiting
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CANbridge (Suzhou) Bio-pharma Co., Ltd.RecruitingGaucher Disease, Type 1 | Gaucher Disease, Type 3China
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Cambridge University Hospitals NHS Foundation TrustMedical Research Council; National Institute for Health Research, United KingdomRecruitingGaucher Disease, Type III | Gaucher Disease, Type IUnited Kingdom
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SanofiCompleted
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Shaare Zedek Medical CenterCompleted
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Amicus TherapeuticsCompletedGaucher Disease | Gaucher Disease, Type 1 | Type 1 Gaucher DiseaseUnited States
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Amicus TherapeuticsCompletedGaucher Disease | Gaucher Disease, Type 1 | Type 1 Gaucher DiseaseUnited Kingdom, Israel, South Africa, United States
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SanofiRecruitingGlycogen Storage Disease Type IIBelgium, United States, China, Germany, Italy, Netherlands, Spain, Taiwan, United Kingdom
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Taipei Veterans General Hospital, TaiwanUnknownGlycogen Storage Disease Type IITaiwan
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Freeline TherapeuticsRecruitingGaucher Disease, Type 1Spain, Israel, United States, United Kingdom, Brazil, Germany, Paraguay
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Taipei Veterans General Hospital, TaiwanNot yet recruitingGlycogen Storage Disease Type II
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LYSOGENETerminatedGM1 GangliosidosisUnited Kingdom, United States, France
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LYSOGENECasimir, LLC; Cure GM1 FoundationCompleted
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Arcturus Therapeutics, Inc.CompletedOrnithine Transcarbamylase DeficiencyNew Zealand
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Translate Bio, Inc.WithdrawnOrnithine Transcarbamylase Deficiency
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Horizon Therapeutics, LLCTerminatedUrea Cycle DisorderUnited States
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Horizon Pharma Ireland, Ltd., Dublin IrelandCompletedUrea Cycle DisordersUnited States, Canada
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Mark BatshawEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsTerminatedUrea Cycle DisordersUnited States, Canada
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Lysosomal and Rare Disorders Research and Treatment...RecruitingBone Diseases | Gaucher DiseaseUnited States
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Lysosomal and Rare Disorders Research and Treatment...UnknownGaucher Disease Type 1United States
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Brendan LeeEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsCompletedAmino Acid Metabolism, Inborn Errors | Urea Cycle Disorders | Argininosuccinic AciduriaUnited States
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University of GlasgowCompletedPhenylketonuria (PKU)United Kingdom
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University of WashingtonCompleted
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National Center for Research Resources (NCRR)University of MichiganCompletedTyrosinemia, Type I
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Centre Hospitalier de CayenneCompleted
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Massachusetts General HospitalActive, not recruiting
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SanofiCompletedGaucher DiseaseUnited States