- ICH GCP
- US Clinical Trials Registry
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Clinical Trials on Metabolism, Inborn Errors
Total 66148 results
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Rubius TherapeuticsTerminated
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Ludwig-Maximilians - University of MunichEuropean UnionCompletedPhenylketonuriaGermany, Italy, Spain, United Kingdom
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BioMarin PharmaceuticalCompletedPhenylketonuriaUnited States
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SynlogicCompletedHealthy | PhenylketonuriaUnited States
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Assiut UniversityUnknown
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Boston Children's HospitalBrigham and Women's HospitalCompletedPhenylketonuriaUnited States
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Washington University School of MedicineUniversity of Missouri-Columbia; BioMarin PharmaceuticalCompleted
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Boston Children's HospitalBeth Israel Deaconess Medical Center; Brigham and Women's HospitalCompletedPhenylketonuriaUnited States
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University of Missouri-ColumbiaBioMarin PharmaceuticalCompletedPhenylketonuriaUnited States
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BioMarin PharmaceuticalCompleted
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BioMarin PharmaceuticalCompletedPhenylketonuriaUnited States, Canada
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Emory UniversityAtlanta Clinical and Translational Science InstituteCompleted
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The University of Texas Health Science Center,...BioMarin PharmaceuticalTerminatedPhenylketonuria | HyperphenylalaninemiaUnited States
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Boston Children's HospitalBeth Israel Deaconess Medical Center; Brigham and Women's HospitalCompletedPhenylketonuriaUnited States
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The University of Texas Health Science Center,...BioMarin PharmaceuticalCompleted
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Emory UniversityTerminated
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Merck KGaA, Darmstadt, GermanyCompleted
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BioMarin PharmaceuticalCompleted
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Shoji YanoBioMarin PharmaceuticalCompletedPhenylketonuriaUnited States
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BioMarin PharmaceuticalCompletedPhenylketonuriasUnited States
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BioMarin PharmaceuticalApproved for marketing
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BioMarin PharmaceuticalCompletedPhenylketonuriasUnited States
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Rocket Pharmaceuticals Inc.Not yet recruitingPyruvate Kinase DeficiencySpain, United States
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Agios Pharmaceuticals, Inc.Active, not recruitingPyruvate Kinase DeficiencyUnited States, Spain, Japan, Canada, United Kingdom, Korea, Republic of, Ireland, Thailand, Italy, Turkey, Denmark, Brazil, France, Germany, Netherlands, Switzerland
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Russian Cardiology Research and Production CenterPfizer; AstraZeneca; AmgenRecruitingFamilial HypercholesterolemiaRussian Federation
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Amryt PharmaRecruitingHomozygous Familial HypercholesterolemiaItaly, United States, Argentina, Canada, France, Greece, Netherlands, United Kingdom
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LIB Therapeutics LLCMedpace, Inc.CompletedHeterozygous Familial HypercholesterolemiaUnited States, Israel, Norway, South Africa, Turkey
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Innovent Biologics (Suzhou) Co. Ltd.CompletedHomozygous Familial HypercholesterolemiaChina
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Regeneron PharmaceuticalsCompletedHomozygous Familial HypercholesterolemiaUnited States, Australia, Austria, Netherlands, Taiwan, Ukraine
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Novartis PharmaceuticalsCompletedA Study of Inclisiran in Participants With Homozygous Familial Hypercholesterolemia (HoFH) (ORION-5)Homozygous Familial HypercholesterolemiaHong Kong, Israel, Russian Federation, Serbia, South Africa, Taiwan, Turkey, Ukraine
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AmgenCompletedSevere Familial HypercholesterolemiaItaly, Czechia, United States, Israel, New Zealand, South Africa, France, Spain, Australia, Belgium, Canada, Netherlands, Hong Kong, Brazil, United Kingdom, Greece, Japan, Lebanon
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Organon and CoTerminatedLipid Metabolism, Inborn Errors | Hypercholesterolemia, Familial
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Korea United Pharm. Inc.Active, not recruiting
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Pfizer's Upjohn has merged with Mylan to form Viatris...CompletedPediatric Heterozygous HypercholesterolemiaGreece, Norway, Canada
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AmgenCompletedHomozygous Familial Hypercholesterolemia HoFHIndia
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Regeneron PharmaceuticalsSanofiCompletedHypercholesterolemia | Heterozygous Familial HypercholesterolemiaUnited States, Canada
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Regeneron PharmaceuticalsSanofiCompletedHeterozygous Familial HypercholesterolemiaUnited States, Germany
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Laval UniversityAmgenCompleted
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Akcea TherapeuticsIonis Pharmaceuticals, Inc.WithdrawnHomozygous Familial HypercholesterolemiaCanada
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AmgenCompletedHomozygous Familial HypercholesterolemiaNew Zealand, South Africa, France, United States, Spain, Belgium, Czechia, Italy, Netherlands, Hong Kong, Canada, Lebanon
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Aegerion Pharmaceuticals, Inc.CompletedFamilial Hypercholesterolemia - HomozygousJapan
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Aegerion Pharmaceuticals, Inc.FDA Office of Orphan Products DevelopmentCompletedHomozygous Familial HypercholesterolemiaUnited States, Canada, Italy, South Africa
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PfizerCompletedHeterozygous Familial HypercholesterolemiaCanada, Netherlands, United Kingdom, United States, Spain, Poland, Finland, Norway, South Africa, Italy, Bulgaria
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Laval UniversityCompletedHomozygous Familial HypercholesterolemiaCanada
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Regeneron PharmaceuticalsSanofiCompletedHeterozygous Familial HypercholesterolemiaUnited Kingdom, Netherlands, Czech Republic, Norway
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Cerenis Therapeutics, SACompletedHomozygous Familial HypercholesterolemiaUnited States, Canada, Italy, Netherlands, United Kingdom
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Aegerion Pharmaceuticals, Inc.University of Pennsylvania; Doris Duke Charitable FoundationCompletedHomozygous Familial HypercholesterolemiaUnited States
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The University of Western AustraliaNational Health and Medical Research Council, AustraliaUnknownFamilial Hypercholesterolaemia | HypertriglyceridaemiaAustralia
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HemoShear TherapeuticsTerminatedPropionic Acidemia | Methylmalonic AcidemiaUnited States, Saudi Arabia, Australia