- 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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Taysha Gene Therapies, Inc.Withdrawn
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Poudre Valley Health SystemEnrolling by invitationFamilial HyperlipidemiaUnited States
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University of PennsylvaniaNorthwestern University; Family Heart FoundationEnrolling by invitationFamilial HypercholesterolemiaUnited States
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Poudre Valley Health SystemActive, not recruiting
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VA Office of Research and DevelopmentCompletedGout | Health Related Quality of Life | Low Medication AdherenceUnited States
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LogicBio Therapeutics, IncAlexion Pharmaceuticals, Inc.Active, not recruitingMethylmalonic AcidemiaUnited States
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Bellicum PharmaceuticalsNo longer availableHurler Syndrome | Inborn Errors of Metabolism | Metachromatic Leukodystrophy | Inherited Metabolic Disorder | Lysosomal Storage DisorderUnited States
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US Department of Veterans AffairsWithdrawn
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Children's Mercy Hospital Kansas CityRecruiting
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Northwestern UniversityUniversity of Pennsylvania; Family Heart FoundationEnrolling by invitationFamilial HypercholesterolemiaUnited States
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Columbia UniversityAmgenTerminatedFamilial HypercholesterolemiaUnited States
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University of PennsylvaniaCompletedHypercholesterolemia, FamilialUnited States
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Emory UniversityKaneka Medical America LLCCompletedFamilial HypercholesterolemiaUnited States
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Wellstat TherapeuticsCompletedHereditary Orotic AciduriaUnited States
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Jerry Vockley, MD, PhDUltragenyx Pharmaceutical IncRecruitingMedium-chain Acyl-CoA Dehydrogenase DeficiencyUnited States
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Geisinger ClinicNational Heart, Lung, and Blood Institute (NHLBI)Enrolling by invitationFamilial HypercholesterolemiaUnited States
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ModernaTX, Inc.RecruitingPropionic AcidemiaUnited States, Canada, United Kingdom
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Alexion PharmaceuticalsCompletedHypophosphatasiaUnited States, Canada
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Vanderbilt University Medical CenterNational Heart, Lung, and Blood Institute (NHLBI)RecruitingFamilial HypercholesterolemiaUnited States
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National Heart, Lung, and Blood Institute (NHLBI)CompletedDyslipidemia | Hypercholesterolemia | Hyperlipidemia | HypolipoproteinemiaUnited States
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Northwestern UniversityUniversity of Pennsylvania; Family Heart FoundationCompletedFamilial HypercholesterolemiaUnited States
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University of Texas Southwestern Medical CenterCompletedHypertriglyceridemia | Hyperlipoproteinemia Type IUnited States
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Alexion PharmaceuticalsCompletedHypophosphatasia | Bone DiseaseUnited States
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Alexion PharmaceuticalsApproved for marketingHypophosphatasiaUnited States, France
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Selecta Biosciences, Inc.National Human Genome Research Institute (NHGRI)SuspendedMethylmalonic Acidemia (MMA)United States
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Alexion PharmaceuticalsCompletedHypophosphatasia (HPP)United States, Canada
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Alexion PharmaceuticalsCompletedHypophosphatasia (HPP)United States, Canada
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Origin BiosciencesCompletedMolybdenum Cofactor DeficiencyUnited States, Australia, United Kingdom, Germany, Netherlands, Turkey
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The Familial Hypercholesterolemia FoundationRecruitingHypercholesterolemiaUnited States
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University of PennsylvaniaLancaster General Hospital; The Familial Hypercholesterolemia FoundationCompleted
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In His ImageKos PharmaceuticalsCompletedDyslipidemia | Hyperlipidemia | Mixed HyperlipidemiaUnited States
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Alexion PharmaceuticalsCompletedHypophosphatasiaUnited States, United Arab Emirates, United Kingdom
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University of Wisconsin, MadisonJohns Hopkins University; National Institute of Diabetes and Digestive and... and other collaboratorsEnrolling by invitationIron Overload | Hemosiderosis | HemochromatosisUnited States
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Boston Children's HospitalWellth Inc.; MMAS-8 with permission from Dr Donald Morisky. as written belowRecruitingAdherence, Medication | Familial HypercholesterolemiaUnited States
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National Center for Research Resources (NCRR)Oregon Health and Science UniversityUnknownCerebrotendinous XanthomatosisUnited States
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Alexion PharmaceuticalsCompletedHypophosphatasia (HPP)United States, Canada
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National Human Genome Research Institute (NHGRI)CompletedUndiagnosed Disease | Healthy Volunteer (Adult With Typically Developing Child) | Inherited Neurodegenerative Disorders | Inherited Metabolic Disorders (Inborn Errors of Metabolism) | Alzheimer's Disease and Related DementiasUnited States
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University of Wisconsin, MadisonNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Stanford...Completed
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University of PennsylvaniaRegeneron PharmaceuticalsCompleted
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National Human Genome Research Institute (NHGRI)RecruitingMitochondrial Disorders | Leigh Disease | Oxidative Phosphorylation Deficiencies | Electron Transport Chain Disorders, MitochondrialUnited States
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Vanderbilt University Medical CenterHarvard UniversityRecruitingAlbright Hereditary Osteodystrophy | PseudohypoparathyroidismUnited States
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Mayo ClinicCompletedHemochromatosisUnited States
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Augusta UniversityCompletedGlucose Intolerance | Fructose Intolerance | Lactose Intolerance | Small Intestinal Bacterial OvergrowthUnited States
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AlexionRecruiting
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Sheffield Teaching Hospitals NHS Foundation TrustNational Institute for Health Research, United Kingdom; Alexion Pharmaceuticals...Completed
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Sheffield Teaching Hospitals NHS Foundation TrustUniversity of SheffieldActive, not recruiting
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Brigham and Women's HospitalActive, not recruitingASCVD | Hyperlipemia, MixedUnited States
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Lumos DiagnosticsAptatek BiosciencesRecruiting
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Emory UniversityBioMarin PharmaceuticalRecruitingPhenylketonuriasUnited States
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Rubius TherapeuticsTerminated