- 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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Alexion PharmaceuticalsCompletedHypophosphatasia (HPP)United States, Canada, United Arab Emirates, United Kingdom
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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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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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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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LogicBio Therapeutics, IncAlexion Pharmaceuticals, Inc.TerminatedMethylmalonic AcidemiaUnited States
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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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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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Rocket Pharmaceuticals Inc.Not yet recruitingPyruvate Kinase DeficiencySpain, United States
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Brendan LeeCompleted
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BioMarin PharmaceuticalRecruitingPhenylketonuria (PKU)United States
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University of Southern CaliforniaBioMarin PharmaceuticalCompleted
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Children's National Research InstituteBoston Children's HospitalRecruitingUrea Cycle DisordersUnited States
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SanofiRecruiting
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Baylor College of MedicineCompletedCitrullinemiaUnited States
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Medical Research Laboratories InternationalTerminatedFamilial HypercholesterolemiaIsrael, Netherlands, United States, Canada, Norway
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AldagenTerminatedMucopolysaccharidosis | Inborn Errors of Metabolism | Lysosomal Storage Disorders | Inherited Metabolic Diseases | Peroxisomal Storage DiseasesUnited States
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University of Alabama at BirminghamUniversity of Auckland, New ZealandCompleted
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Baylor College of MedicineUniversity of Alabama at BirminghamCompleted
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University of Alabama at BirminghamUniversity of NebraskaCompleted
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Ritter Pharmaceuticals, Inc.CompletedLactose IntoleranceUnited States
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NeuroBo Pharmaceuticals Inc.CompletedHypercholesteremiaUnited States, Canada, Israel
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Daiichi Sankyo, Inc.CompletedHypercholesterolemia | Hyperlipidemia, Familial CombinedUnited States
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Homology Medicines, IncTerminatedPhenylketonuria | Phenylketonurias | PAH DeficiencyUnited States
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Agios Pharmaceuticals, Inc.CompletedPyruvate Kinase DeficiencyUnited States, Italy, Netherlands
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Dr. Linda RandolphBioMarin PharmaceuticalTerminated
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PfizerCompletedHypercholesterolemia, FamilialCanada, United States, South Africa
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University of Alabama at BirminghamVanderbilt University Medical CenterRecruiting
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Indiana UniversityCompletedLactose IntoleranceUnited States
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Stanford UniversityCompleted
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ModernaTX, Inc.RecruitingPhenylketonuriaUnited States, Australia
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Beyond Batten Disease FoundationTheranexusActive, not recruitingBatten DiseaseUnited States
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jasvinder singhNovartis; Takeda; Regeneron Pharmaceuticals; Birmingham, Alabama VA Medical CenterWithdrawn
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University of South FloridaCompleted
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University of California, Los AngelesNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)Not yet recruiting
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Purdue UniversityRecruiting
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Vivante HealthDuke UniversityCompletedLactose IntoleranceUnited States
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Ritter Pharmaceuticals, Inc.CompletedLactose IntoleranceUnited States
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Alexion Pharmaceuticals, Inc.Not yet recruitingHypophosphatasiaUnited States, Taiwan, Thailand, Vietnam, United Kingdom, Korea, Republic of, Israel, Italy, Australia, Turkey, France, Poland, Germany, Spain, Belgium, China, Canada, Brazil, Argentina, Mexico
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Baylor College of MedicineCompletedUrea Cycle Disorders, InbornUnited States
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Applied Therapeutics, Inc.Active, not recruitingClassic GalactosemiaUnited States
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Ultragenyx Pharmaceutical IncTerminatedGlycogen Storage Disease Type IIIUnited States, Netherlands
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Geisinger ClinicCompletedHypercholesterolemia | Hypercholesterolemia, Familial | Hypercholesteremia in Children | Hyperlipidemia in ChildrenUnited States