Clinical Trials on Inborn Errors of Metabolism in United States
Total 3211 results
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Horizon Pharma Ireland, Ltd., Dublin IrelandCompletedUrea Cycle DisordersUnited 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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SynlogicCompletedHealthy Volunteer | Urea Cycle DisorderUnited States
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Ultragenyx Pharmaceutical IncCompletedOrnithine Transcarbamylase DeficiencyUnited 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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LIB Therapeutics LLCMedpace, Inc.Enrolling by invitationFamilial Hypercholesterolemia | Elevated Cholesterol | Cardiovascular Disease With Mention of ArteriosclerosisUnited States, Israel, Norway, South Africa, Turkey, India
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University of PennsylvaniaUniversity of Amsterdam; University of Cape Town; University of Witwatersrand...RecruitingHomozygous Familial HypercholesterolemiaUnited States, Netherlands, South Africa
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Regeneron PharmaceuticalsSanofiCompletedHypercholesterolemiaFrance, United States
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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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Ultragenyx Pharmaceutical IncCompletedGlycogen Storage Disease Type IIIUnited States
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Ultragenyx Pharmaceutical IncTerminatedGlycogen Storage Disease Type IIIUnited States, Spain, Italy
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PTC TherapeuticsCompletedBH4 Deficiency | HyperphenylalaninemiaUnited States
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CoA Therapeutics, Inc., a BridgeBio companyTerminatedHealthy Volunteers | Propionic Acidemia | Methylmalonic Acidemia | Organic AcidemiaUnited States
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University of California, Los AngelesEnrolling by invitationTophaceous GoutUnited States
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Alexion Pharmaceuticals, Inc.Enrolling by invitationHypophosphatasia (HPP)United States, Italy, Russian Federation, Spain, United Kingdom, Germany, Canada, France, Saudi Arabia, Poland, Australia
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Imperial College London Diabetes CentreUnknownFamilial HypercholesterolemiaUnited Arab Emirates
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Horizon Therapeutics, LLCTerminatedUrea Cycle DisorderUnited States
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ModernaTX, Inc.RecruitingPropionic AcidemiaUnited States, Canada, United Kingdom
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Tulane University School of MedicineBioMarin PharmaceuticalCompletedPhenylketonuria | Behavior and Behavior Mechanisms | PAH Gene ExpressionUnited States
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Genzyme, a Sanofi CompanyCompletedGaucher DiseaseUnited States
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University of NottinghamUniversity College, London; Newcastle University; University of ManchesterUnknownFamilial HypercholesterolemiaUnited Kingdom
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Johnson & Johnson Vision Care, Inc.CompletedCorrection of Refraction Error | Attenuation of Bright LightUnited States
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La Jolla Pharmaceutical CompanyPRA Health SciencesCompletedHereditary HemochromatosisUnited States, Australia, France, United Kingdom
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ModernaTX, Inc.RecruitingMethylmalonic AcidemiaUnited States, Spain, Canada, Netherlands, France, Australia, United Kingdom
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Target PharmaSolutions, Inc.Recordati Rare DiseasesEnrolling by invitationPropionic Acidemia | Methylmalonic Acidemia | HyperammonemiaUnited States
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University of Alabama at BirminghamKaiser Permanente; University of NebraskaCompleted
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Cerenis Therapeutics, SATerminatedFamilial HypoalphalipoproteinemiaFrance, United States, Belgium, Canada, Israel, Italy, Netherlands
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Novartis PharmaceuticalsCompletedIron Overload | Hereditary HemochromatosisUnited States, Germany, Italy, Australia, Canada, France
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University of Texas Southwestern Medical CenterCompletedInsulin Resistance | Partial Lipodystrophy | Generalized Lipodystrophy | HypoleptinemiaUnited States
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Inozyme PharmaRecruitingGeneralized Arterial Calcification of Infancy | Autosomal Recessive Hypophosphatemic Rickets | Ectonucleotide Pyrophosphatase/phosphodiesterase1 DeficiencyUnited States, United Kingdom, Canada, France, Germany
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Kastle Therapeutics, LLCIonis Pharmaceuticals, Inc.CompletedHypercholesterolemia, FamilialUnited States, Netherlands
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Children's Hospital of PhiladelphiaTerminatedAlbright Hereditary Osteodystrophy | Pseudohypoparathyroidism Type 1AUnited States
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Alexion PharmaceuticalsTerminated
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BioMarin PharmaceuticalCompletedPhenylketonuriaUnited States
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University of PennsylvaniaRecruitingLCAT DeficiencyUnited States
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University of MinnesotaRecruitingGaucher Disease, Type 3United States
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PfizerWithdrawnType 1 Gaucher DiseaseUnited States
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National Institute of Neurological Disorders and...CompletedType 2 Gaucher DiseaseUnited States
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National Institute of Neurological Disorders and...CompletedGaucher DiseaseUnited States
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National Institute of Neurological Disorders and...Completed
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University of California, San FranciscoCompletedSickle Cell Disease | Hemoglobinopathies | Thalassemia | Hematologic Malignancies | Immunodeficiencies | Fanconi's Anemia | Nonmalignant Diseases | Genetic Inborn Errors of MetabolismUnited States
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Sanguine BiosciencesTerminatedGlycogen Storage Disease Type IBUnited States
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AstraZenecaCompletedFamilial HypercholesterolemiaUnited States, Spain, Netherlands, Norway, Canada
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Neurogene Inc.RecruitingCeroid Lipofuscinosis, Neuronal 5United States
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Arrowhead PharmaceuticalsActive, not recruitingHomozygous Familial HypercholesterolemiaAustralia, Canada, United States, South Africa
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Ardea Biosciences, Inc.Completed
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Emily de los ReyesRecruitingBatten Disease | CLN6United States
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StemCells, Inc.WithdrawnNeuronal Ceroid LipofuscinosisUnited States