Clinical Trials on Metabolism, Inborn Errors in United States
Total 28242 results
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Oregon Health and Science UniversityTerminated
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Sheffield Teaching Hospitals NHS Foundation TrustCompleted
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Medical Research Laboratories InternationalTerminatedFamilial HypercholesterolemiaNetherlands, United States, Norway
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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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Washington University School of MedicineUniversity of Missouri-Columbia; BioMarin PharmaceuticalCompleted
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Shoji YanoBioMarin PharmaceuticalCompletedPhenylketonuriaUnited States
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Vitaflo International, LtdOregon Health and Science UniversityCompleted
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Children's Hospital Medical Center, CincinnatiBioMarin PharmaceuticalCompleted
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University of MiamiCompletedPhenylketonuriaUnited States
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Children's Hospital of PhiladelphiaCompleted
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Eunice Kennedy Shriver National Institute of Child...Completed
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National Human Genome Research Institute (NHGRI)RecruitingPropionic Acidemia | Metabolic Disease | Organic AcidemiaUnited States
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Vitaflo International, LtdCompleted
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PTC TherapeuticsAxio Research. LLCCompleted
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Rocket Pharmaceuticals Inc.Active, not recruiting
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University of Southern CaliforniaNational PKU AllianceActive, not recruitingPhenylketonuriasUnited States
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Emory UniversityBioMarin Pharmaceutical; Atlanta Clinical and Translational Science InstituteCompletedPhenylketonuriaUnited States
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National Human Genome Research Institute (NHGRI)Recruiting
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Vitaflo International, LtdBirmingham Women's and Children's NHS Foundation TrustCompletedPhenylketonuria | Inborn Errors of MetabolismUnited Kingdom
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CENTOGENE GmbH RostockCompletedPyruvate Kinase DeficiencyUnited States
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University of South FloridaNational Institutes of Health (NIH)CompletedUrea Cycle DisordersUnited States
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PTC TherapeuticsCompletedLeigh SyndromeUnited States
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Alexion Pharmaceuticals, Inc.RecruitingHypophosphatasiaUnited States, Japan, Germany, Turkey, Italy, India, Australia, France, United Kingdom, Argentina, Canada
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Applied Therapeutics, Inc.Active, not recruiting
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BioMarin PharmaceuticalCompletedPhenylketonuriaUnited States
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Emory UniversityNational PKU AllianceCompleted
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Essentia HealthSt. Luke's Hospital of Duluth; WHITESIDE Institute for Clinical ResearchRecruitingFamilial Hypercholesterolemia | Genetic Disease | Cholesterol, ElevatedUnited States
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National Heart, Lung, and Blood Institute (NHLBI)Completed
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TakedaTerminatedHypercholesterolemiaFrance, United Kingdom, United States, Israel, Poland, Canada
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National Human Genome Research Institute (NHGRI)RecruitingArterial Calcification Due to Deficiency of CD73United States
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HemoShear TherapeuticsPrometrika, LLC; AllStripes Research Inc.; Genome MedicalTerminatedPropionic Acidemia | Methylmalonic AcidemiaUnited States, Canada, United Kingdom
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Ultragenyx Pharmaceutical IncCompletedGSD1Netherlands, United States, Canada, Spain
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Alexion PharmaceuticalsCompletedHypophosphatasia (HPP)United States, Canada, United Arab Emirates, United Kingdom
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University of Missouri-ColumbiaBioMarin PharmaceuticalEnrolling by invitationPhenylketonuriasUnited States
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University of Wisconsin, MadisonBoston Children's HospitalCompletedPhenylketonuriaUnited States
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Children's National Research InstituteGeorgetown UniversityWithdrawn
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Origin BiosciencesCompletedMolybdenum Cofactor Deficiency, Type AUnited States, Australia, Netherlands, United Kingdom, Tunisia
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HDL TherapeuticsMedStar Heart and Vascular InstituteUnknownHomozygous Familial Hypercholesterolemia | HoFHUnited States
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The Rogosin InstituteRecruitingHomozygous Familial HypercholesterolemiaUnited States
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National Heart, Lung, and Blood Institute (NHLBI)Completed
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Areeg El-GharbawyUltragenyx Pharmaceutical IncCompletedGlycogen Storage Disease Type IUnited States
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Alexion PharmaceuticalsCompletedHypophosphatasia (HPP)United States, Taiwan, United Kingdom, Australia, Canada, Germany, Spain
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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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Alexion PharmaceuticalsCompletedHypophosphatasiaUnited States, Spain, Australia, United Kingdom, Italy, France, Canada, Germany, Japan, Russian Federation, Saudi Arabia, Turkey
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LogicBio Therapeutics, IncAlexion Pharmaceuticals, Inc.TerminatedMethylmalonic AcidemiaUnited 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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Children's National Research InstituteBoston Children's HospitalRecruitingUrea Cycle DisordersUnited States