- ICH GCP
- US Clinical Trials Registry
- Search trials
Clinical Trials on Metabolism, Inborn Errors
Total 66343 results
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Fresenius Medical Care Deutschland GmbHCompletedCardiovascular Diseases | Dyslipidemias | Hypercholesterolemia, FamilialGermany
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The Medicines CompanyCompletedElevated Cholesterol | Heterozygous Familial HypercholesterolemiaUnited States, Canada, Czechia, Denmark, Netherlands, South Africa, Spain, Sweden
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AstraZenecaCompletedHomozygous Familial Hypercholesterolemia (HoFH)Malaysia, Canada, Taiwan, Belgium, Denmark, Israel
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National Heart, Lung, and Blood Institute (NHLBI)CompletedBlood Disease | Hemochromatosis
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University of RochesterNational Heart, Lung, and Blood Institute (NHLBI)CompletedBlood Disease | Hemochromatosis
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Sheba Medical CenterUnknownCerebrotendinous Xanthomatosis (CTX)Israel
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Amsterdam Molecular TherapeuticsInternational Antiviral Therapy Evaluation CenterUnknownFamilial Lipoprotein Lipase DeficiencyCanada
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BioMarin PharmaceuticalRecruitingPhenylketonuria (PKU)United States, Germany, Italy
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Nutricia ResearchCompleted
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BioMarin PharmaceuticalActive, not recruiting
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BioMarin PharmaceuticalCompletedPhenylketonuria (PKU)United States, Turkey, Canada, France, Germany, Italy, United Kingdom
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Sutphin DrugsUnknownHereditary Tyrosinemia, Type IIndia
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Charite University, Berlin, GermanyJohannes Gutenberg University MainzCompletedPhenylketonuria | Endothelial Dysfunction
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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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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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University of BonnRecruitingHypophosphatasiaGermany
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ModernaTX, Inc.CompletedPropionic Acidemia | Methylmalonic AcidemiaUnited States, United Kingdom, Spain, France
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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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Khondrion BVDrug Research Unit Ghent, BelgiumCompletedMitochondrial Disease | MELAS | Leigh Syndrome | LHON | Mitochondrial DNA tRNALeu(UUR) m.3243A<G MutationBelgium
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Regeneron PharmaceuticalsActive, not recruitingGeneralized LipodystrophyUnited States, Peru, Russian Federation, Turkey
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metaX Institut fuer Diatetik GmbHBirmingham Children's HospitalCompletedPhenylketonurias | Hyperphenylalaninaemia | Tetrahydrobiopterin DeficiencyUnited Kingdom
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BioMarin PharmaceuticalActive, not recruitingPhenylketonuria (PKU)United States, Germany
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Homology Medicines, IncEnrolling by invitationPhenylketonuria | PAH DeficiencyUnited States
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BioMarin PharmaceuticalCompletedPhenylketonuria (PKU)United States
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BioMarin PharmaceuticalCompletedPhenylketonuria (PKU)United States
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University of British ColumbiaRare Disease Foundation, Vancouver, CanadaCompleted
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Brendan LeeCompleted
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University of GlasgowUnknownPhenylketonuria (PKU)United Kingdom
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University Hospital, ToursInstitut National de la Santé Et de la Recherche Médicale, FranceCompletedPKU | HyperphenylalaninemiaFrance
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Dr. Linda RandolphBioMarin PharmaceuticalTerminated
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Baylor College of MedicineCompletedCitrullinemiaUnited States
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Shenzhen People's HospitalRecruitingEvaluate the Efficacy and Safety of IBI306 in Subjects With Homozygous Familial HypercholesterolemiaLipid Metabolism Disorders | Familial Hypercholesterolemia - Homozygous | Proprotein Convertase Subtilisin/Kexin 9China
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Laval UniversityUniversité de Montréal; Centre de Recheche du Centre Hospitalier Université...RecruitingCardiovascular Diseases | Familial Hypercholesterolemia | Cholesterol, Elevated | Nutrition, Healthy | Cholesterol; Metabolic DisorderCanada
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Luca RonfaniUniversity of TriesteCompletedUrea Cycle Disorders, Inborn | Other Metabolic DiseasesItaly
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Aegerion Pharmaceuticals, Inc.Active, not recruitingGeneralized LipodystrophyUnited States
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Laval UniversityCentre de Recherche de l'Institut Universitaire de Cardiologie et de Pneumologie... and other collaboratorsActive, not recruitingDiet Habit | Hypercholesterolemia, Familial | Calcification; HeartCanada
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Federal University of Rio Grande do SulCompletedSedentary Lifestyle | Dyslipidemias | Lipid Metabolism Disorders | Carbohydrate Inducible Hyperlipemia
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Aeglea BiotherapeuticsCompletedArginase I Deficiency | HyperargininemiaUnited States, Canada, United Kingdom, Portugal
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Aeglea BiotherapeuticsCompletedArginase I Deficiency | HyperargininemiaUnited States, Canada, United Kingdom, Portugal
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Shanghai Institute Of Biological ProductsThe First Affiliated Hospital of Henan University of Science and Technology; First Affiliated Hospital Bengbu Medical College and other collaboratorsRecruiting
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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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ETH ZurichUniversity of Zurich; Agroscope Liebefeld-Posieux Research Station ALP; Swiss...RecruitingLactose IntoleranceSwitzerland
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Novozymes A/SAnalyze & RealizeNot yet recruiting
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Xinhua Hospital, Shanghai Jiao Tong University...Recruiting
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Purdue UniversityRecruiting
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Chinese University of Hong KongRecruiting
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Assistance Publique - Hôpitaux de ParisCanon Medical Systems, USARecruiting
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Alnylam PharmaceuticalsTerminated
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Eisai Co., Ltd.Completed