- ICH GCP
- US Clinical Trials Registry
- Search trials
Clinical Trials on Brain Diseases, Metabolic in United States
Total 147767 results
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Travere Therapeutics, Inc.Enrolling by invitation
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Tulane University School of MedicineBioMarin PharmaceuticalCompletedPhenylketonuria | Behavior and Behavior Mechanisms | PAH Gene ExpressionUnited States
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Terence FlotteMassachusetts General Hospital; University of Massachusetts, WorcesterActive, not recruitingTay-Sachs Disease | Sandhoff DiseaseUnited States
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Duke UniversitySanofiRecruiting
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Duke UniversityGenzyme, a Sanofi CompanyRecruitingPompe DiseaseUnited States
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University of Massachusetts, AmherstWithdrawnOxidative Stress | COPD | Tetrahydrobiopterin DeficiencyUnited States
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University of California, IrvineGenzyme, a Sanofi CompanyCompletedPompe DiseaseUnited States
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Amicus TherapeuticsTerminated
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University of FloridaTerminated
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O & O Alpan LLCCompleted
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Washington University School of MedicineUniversity of Missouri-Columbia; Northwestern University; Oregon Health and Science... and other collaboratorsTerminated
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University Research Foundation for Lysosomal Storage...University of PittsburghCompletedGaucher DiseaseUnited States
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Asklepios Biopharmaceutical, Inc.Active, not recruiting
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Amicus TherapeuticsCompletedGaucher Disease | Gaucher Disease, Type 1 | Type 1 Gaucher DiseaseUnited Kingdom, United States
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Prevail TherapeuticsEli Lilly and CompanyActive, not recruitingGaucher Disease, Type 2United States, United Kingdom
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Baylor College of MedicineSeattle Children's Hospital; University of California, San FranciscoCompleted
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SynlogicCompletedHealthy | PhenylketonuriaUnited States
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Columbia UniversityEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationMitochondrial Disease | Mitochondrial Disorders | Melas | Kearns Sayer | NARP | MNGIE | LHON | Mitochondrial Depletion Syndrome | Leigh's DiseaseUnited States
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University of PittsburghNational Institute of Neurological Disorders and Stroke (NINDS)Active, not recruiting
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BioMarin PharmaceuticalCompleted
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Ultragenyx Pharmaceutical IncTerminatedOrnithine Transcarbamylase DeficiencyUnited States
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Valerion Therapeutics, LLCTerminatedPompe DiseaseUnited States, United Kingdom
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SynlogicCompletedHomocystinuriaUnited States
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University of MinnesotaRecruitingInflammation | Oxidative Stress | Gaucher Disease Type IUnited States
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National Institute of Neurological Disorders and...Completed
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Serhat Gumrukcu, MD PhDFrida Therapeutics LLCRecruiting
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Duke UniversityCompleted
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Duke UniversityGenzyme, a Sanofi CompanyCompleted
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Duke UniversityCompletedPompe DiseaseUnited States
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Duke UniversityCompleted
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Dwight Koeberl, M.D., Ph.D.Completed
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SynlogicCompletedHealthy Volunteer | Urea Cycle DisorderUnited States
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Baylor College of MedicineCompletedUrea Cycle Disorders | Argininosuccinic Aciduria | Argininosuccinate Lyase DeficiencyUnited States
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National Center for Research Resources (NCRR)Baylor College of MedicineUnknownAmino Acid Metabolism, Inborn ErrorsUnited States
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PTC TherapeuticsAxio Research. LLCCompleted
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Forge Biologics, IncNot yet recruitingKrabbe DiseaseUnited States
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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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PTC TherapeuticsCompletedBH4 Deficiency | HyperphenylalaninemiaUnited States
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Genzyme, a Sanofi CompanyCompleted
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II | Pompe DiseaseUnited States
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National Institute of Neurological Disorders and...Completed
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Children's Hospital Medical Center, CincinnatiCompletedOsteopenia | Gaucher's DiseaseIsrael, United States
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BioMarin PharmaceuticalTerminatedLate-onset Pompe DiseaseUnited States, United Kingdom
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Duke UniversityCompleted
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State University of New York at BuffaloEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsRecruiting
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsTerminatedTay-Sachs Disease | Sandhoff Disease | GM1 Gangliosidoses | GM2 GangliosidosesUnited States
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Children's National Research InstitutePatient-Centered Outcomes Research Institute; Eunice Kennedy Shriver National... and other collaboratorsCompleted
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Eunice Kennedy Shriver National Institute of Child...Completed