- ICH GCP
- US Clinical Trials Registry
- Search trials
Clinical Trials on Inborn Errors of Metabolism in United States
Total 3211 results
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University of Texas Southwestern Medical CenterWithdrawnFamilial HypercholesterolemiaUnited States
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Emory UniversityPatient-Centered Outcomes Research InstituteCompleted
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Homology Medicines, IncEnrolling by invitationPhenylketonuria | PAH DeficiencyUnited States
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Protagonist Therapeutics, Inc.CompletedHereditary HemochromatosisUnited States, Canada
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The Medicines CompanyCompletedHomozygous Familial HypercholesterolemiaUnited States, Netherlands, South Africa
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Ardea Biosciences, Inc.Completed
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Vanderbilt University Medical CenterTerminatedAlbright Hereditary Osteodystrophy | Pseudohypoparathyroidism and Pseudopseudohypoparathyroidism | Pseudohypoparathyroidism Type IaUnited States
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Lysosomal and Rare Disorders Research and Treatment...UnknownGaucher DiseaseUnited States
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Aegerion Pharmaceuticals, Inc.CompletedFamilial HypercholesterolemiaUnited States, Canada, Italy, South Africa
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Purdue UniversityRecruitingLactose IntoleranceUnited States
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Alexion PharmaceuticalsCompletedHypophosphatasia (HPP)United States, France, United Kingdom, Canada, Netherlands, Russian Federation, Turkey, Australia
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Connecticut Children's Medical CenterJohns Hopkins University; Eunice Kennedy Shriver National Institute of Child... and other collaboratorsRecruitingAlbright Hereditary Osteodystrophy | Pseudopseudohypoparathyroidism | Pseudohypoparathyroidism Type 1AUnited States
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ModernaTX, Inc.RecruitingPropionic AcidemiaUnited States, Spain, Netherlands, France, United Kingdom, Australia, Canada, Italy
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SynlogicCompletedHealthy | PhenylketonuriaUnited States
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BioMarin PharmaceuticalActive, not recruiting
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University of MinnesotaRecruitingGaucher DiseaseUnited States
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TakedaCompletedGaucher DiseaseUnited States
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Genzyme, a Sanofi CompanyCompleted
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Brigham and Women's HospitalGenzyme, a Sanofi CompanyUnknown
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Applied Therapeutics, Inc.CompletedClassic GalactosemiaUnited States
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Rutgers, The State University of New JerseyCompleted
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Mayo ClinicNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Orphan...CompletedHyperoxaluriaUnited States
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University of Massachusetts, WorcesterTakedaCompleted
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Indiana UniversityCompletedChronic Kidney DiseaseUnited States
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Mirum Pharmaceuticals, Inc.Completed
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BioMarin PharmaceuticalCompleted
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TakedaCompleted
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Ritter Pharmaceuticals, Inc.CompletedLactose IntoleranceUnited States
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Origin BiosciencesCompletedMolybdenum Cofactor Deficiency, Type AUnited States, Spain, Israel, Norway, Turkey, United Kingdom
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Cerenis Therapeutics, SACompletedHomozygous Familial HypercholesterolemiaUnited States, Canada, Italy, Netherlands, United Kingdom
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PTC TherapeuticsGenzyme, a Sanofi CompanyTerminatedAmino Acid Metabolism, Inborn ErrorsFrance, United Kingdom, Italy, Belgium, Germany, Switzerland
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Regeneron PharmaceuticalsCompletedHomozygous Familial HypercholesterolemiaUnited States, Netherlands, Canada
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University of Missouri-ColumbiaCompleted
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Kastle Therapeutics, LLCIonis Pharmaceuticals, Inc.CompletedHypercholesterolemia, FamilialUnited States, Netherlands
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Oregon Health and Science UniversityUniversity of Oregon; Oregon State UniversityRecruitingCarbohydrate IntoleranceUnited States
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OxTheraCompletedPrimary HyperoxaluriaUnited States
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Amicus TherapeuticsActive, not recruiting
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Massachusetts General HospitalAlexion PharmaceuticalsUnknownCholesterol Ester Storage Disease | Lysosomal Acid Lipase DeficiencyUnited States
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Duke UniversityRecruiting
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Alexion Pharmaceuticals, Inc.RecruitingHypophosphatasiaItaly, Spain, Israel, Korea, Republic of, Belgium, Germany, United States, China, France, Taiwan, Austria, Japan, India, Australia, Argentina, Poland, Turkey, Brazil, Canada, Thailand, United Kingdom, Slovakia, Czechia
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Duke UniversityRecruitingAdult Polyglucosan Body Disease | Glycogen Storage Disease Type IV | GSD4 | GSD IV | APBDUnited States
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Lundquist Institute for Biomedical Innovation at...Swedish Orphan Biovitrum; Cure Sanfilippo FoundationCompleted
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Mayo ClinicCompleted
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Mayo ClinicNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Oxalosis...Completed
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BioMarin PharmaceuticalCompleted
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BioMarin PharmaceuticalCompletedMucopolysaccharidosis VIUnited States
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BioMarin PharmaceuticalCompletedMucopolysaccharidosis VIUnited States
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BioMarin PharmaceuticalCompletedMucopolysaccharidosis VIUnited States
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Amicus TherapeuticsActive, not recruitingBatten Disease | CLN6United States