- ICH GCP
- US Clinical Trials Registry
- Search trials
Clinical Trials on Inborn Errors of Metabolism
Total 6431 results
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Boston Children's HospitalBioMarin PharmaceuticalCompleted
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NestléWithdrawn
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Universidade do PortoBioMarin Pharmaceutical; Centro Hospitalar do PortoCompleted
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University of British ColumbiaVitaflo International, LtdUnknown
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BioMarin PharmaceuticalCompletedPhenylketonuriaUnited States
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Nutricia UK LtdWithdrawn
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BioMarin PharmaceuticalCompletedPhenylketonuriaUnited States
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BioMarin PharmaceuticalCompletedPhenylketonuriaUnited States
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Academisch Ziekenhuis MaastrichtCompleted
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University of Wisconsin, MadisonBoston Children's HospitalCompletedPhenylketonuriaUnited States
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BioMarin PharmaceuticalTerminatedPhenylketonuriaItaly, Switzerland
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Emory UniversityBioMarin Pharmaceutical; Atlanta Clinical and Translational Science InstituteCompletedPhenylketonuriaUnited States
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Children's National Research InstituteGeorgetown UniversityWithdrawn
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Oregon Health and Science UniversityTerminated
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Merck KGaA, Darmstadt, GermanySmerud Medical Research International AS; Merck Serono NorwayCompleted
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BioMarin PharmaceuticalCompletedStudy of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006PhenylketonuriaUnited States
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The Kennedy Institute-National Eye ClinicSygekassernes HelsefondCompleted
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Johnson & Johnson Vision Care, Inc.CompletedCorrection of Refraction Error | Attenuation of Bright LightUnited States
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Vanderbilt University Medical CenterRecruitingPseudohypoparathyroidism Type 1a | Albright Hereditary Osteodystrophy | PseudohypoparathyroidismUnited States
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Ashley ShoemakerEnrolling by invitationPseudohypoparathyroidism Type 1a | Albright Hereditary Osteodystrophy | PseudohypoparathyroidismUnited States
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Vanderbilt University Medical CenterMassachusetts General HospitalCompletedPseudohypoparathyroidism Type 1a | Albright Hereditary OsteodystrophyUnited States
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University Children's Hospital, ZurichRecruitingUrea Cycle DisordersSwitzerland
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Brain-Gut Research GroupRecruitingFructose Intolerance | Functional Gastrointestinal Disorders | Lactose IntoleranceSwitzerland
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Agios Pharmaceuticals, Inc.CompletedPyruvate Kinase DeficiencyUnited States, Italy, Netherlands
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CENTOGENE GmbH RostockCompletedPyruvate Kinase DeficiencyUnited States
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Agios Pharmaceuticals, Inc.CompletedAnemia, Hemolytic | Pyruvate Kinase DeficiencyUnited States, Spain, Switzerland, Turkey, Germany, France, United Kingdom, Japan, Netherlands, Brazil, Canada, Czechia, Denmark, Italy, Korea, Republic of, Thailand
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Ultragenyx Pharmaceutical IncTerminatedGlycogen Storage Disease Type IIIUnited States, Spain, Italy
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Chiesi Farmaceutici S.p.A.Completed
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Zymenex A/SCompleted
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Eunice Kennedy Shriver National Institute of Child...Completed
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Zymenex A/SEuropean CommissionCompletedAlpha-MannosidosisBelgium, Denmark, Spain, United Kingdom
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HemoShear TherapeuticsPrometrika, LLC; AllStripes Research Inc.; Genome MedicalTerminatedPropionic Acidemia | Methylmalonic AcidemiaUnited States, Canada, United Kingdom
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Rigshospitalet, DenmarkUniversity College, LondonCompletedMcArdle DiseaseDenmark, United Kingdom
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Reneo Pharma LtdCompletedMcArdle DiseaseSpain, United Kingdom
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IRCCS Eugenio MedeaUniversity of Pisa; University of MessinaCompleted
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Rigshospitalet, DenmarkCompleted
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Children's National Research InstituteBoston Children's HospitalRecruitingUrea Cycle DisordersUnited States
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Cytonet GmbH & Co. KGCRS Clinical Research Services Mannheim GmbHCompleted
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NGGT (Suzhou) Biotechnology Co., Ltd.Not yet recruitingPhenylketonurias
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ModernaTX, Inc.RecruitingPhenylketonuriaUnited States, Australia
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APR Applied Pharma Research s.a.RecruitingPhenylketonuriasUnited Kingdom
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PTC TherapeuticsRecruitingPhenylketonuriaUnited States, Germany, Spain, Australia, Brazil, United Kingdom, Denmark, Mexico, Portugal, Turkey, Canada, Georgia, Italy, Netherlands, Japan
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Nutricia UK LtdNot yet recruitingPhenylketonurias | Hyperphenylalaninaemia
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Ajinomoto Co., Inc.Great Ormond Street Hospital for Children NHS Foundation Trust; Birmingham...Active, not recruiting
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Vitaflo International, LtdOregon Health and Science UniversityCompleted
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Vitaflo International, LtdArla FoodsCompletedPhenylketonuriasUnited Kingdom, Denmark
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Vitaflo International, LtdActive, not recruiting
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PTC TherapeuticsCompletedPhenylketonuriaSpain, United States, Australia, Germany, United Kingdom, Brazil, Denmark, Canada, France, Georgia, Italy, Mexico, Netherlands, Portugal, Turkey