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- US Clinical Trials Registry
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Clinical Trials on Inherited Metabolic Storage Diseases in United States
Total 26919 results
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SanofiRecruitingFabry DiseaseChina, Finland, Germany, Norway, United Kingdom, United States, Denmark, Austria, Greece, Romania, Argentina, Brazil, Canada, Japan, Mexico, Poland, Italy, France, Turkey, Australia, Switzerland
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ShireCompletedSanfilippo SyndromeUnited States, Spain, United Kingdom, Argentina, France, Germany, Italy, Netherlands
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State University of New York - Downstate Medical...TerminatedDiabetes Mellitus | End-Stage Renal DiseaseUnited States
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Ege UniversityUniversity of Kentucky; Fresenius Medical Care North AmericaCompletedHemodialysis | End-Stage Renal DiseaseTurkey, United States
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Nemours Children's ClinicEunice Kennedy Shriver National Institute of Child Health and Human Development...RecruitingMucopolysaccharidosis IV Type A | Morquio A Syndrome | MPS IVAUnited States
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BioMarin PharmaceuticalTerminatedMorquio A Syndrome | MPS IVA | Mucopolysaccharidosis IVAUnited Kingdom, United States, Germany
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Northwell HealthStaten Island University HospitalCompleted
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Zymenex A/SEuropean CommissionCompletedAlpha MannosidosisCzechia, Germany, Norway, United Kingdom
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University Hospital Birmingham NHS Foundation TrustNorthern Care Alliance NHS Foundation Trust; Cambridge University Hospitals... and other collaboratorsActive, not recruitingArrhythmia Burden, Risk of Sudden Cardiac Death and Stroke in Patients With Fabry Disease (RaILRoAD)Fabry DiseaseUnited Kingdom, Australia
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University of ManchesterUniversity College, London; Manchester University NHS Foundation Trust; Orchard... and other collaboratorsActive, not recruitingMucopolysaccharidosis Type IIIAUnited Kingdom
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IntraBio IncCompletedNiemann-Pick Disease, Type CUnited States, United Kingdom, Germany, Slovakia, Spain
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Woman'sPfizer; American Cancer Society, Inc.; Our Lady of the Lake Regional Medical...WithdrawnInsulin Resistance | Breast Cancer Stage | Racial BiasUnited States
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National Human Genome Research Institute (NHGRI)RecruitingRare Diseases | Inherited Hematological Diseases | FPDMMUnited States
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Allievex CorporationActive, not recruitingMucopolysaccharidosis Type IIIBUnited States, Germany, Spain, Argentina, Australia, Brazil, Colombia, Taiwan, Turkey
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Eunice Kennedy Shriver National Institute of Child...University of MinnesotaCompleted
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Greenwood Genetic CenterShriners Hospitals for Children; BioMarin PharmaceuticalCompletedMPS IVA | Maroteaux Lamy Syndrome | MPS VI | Morquio Syndrome AUnited States
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Genzyme, a Sanofi CompanyBioMarin/Genzyme LLCCompletedMucopolysaccharidosis I | Hurler-Scheie Syndrome | Hurlers SyndromeUnited States, Canada, Germany
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BioMarin PharmaceuticalCompleted
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University of Texas Southwestern Medical CenterNational Heart, Lung, and Blood Institute (NHLBI)RecruitingLipid Metabolism DisorderUnited States
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Masonic Cancer Center, University of MinnesotaCompletedMucopolysaccharidosis | Hurler Syndrome | Sphingolipidoses | Peroxisomal Disorders | Krabbe Disease | Adrenoleukodystrophy (ALD) | Hunter Syndrome | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Alpha Mannosidosis | Metachromatic Leukodystrophy (MLD) | Maroteaux-Lamy SyndromeUnited States
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JCR Pharmaceuticals Co., Ltd.Enrolling by invitationMucopolysaccharidosis IIUnited States
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REGENXBIO Inc.Enrolling by invitation
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Massachusetts General HospitalNational Cancer Institute (NCI); Boston Medical CenterCompletedGenetic TestingUnited States
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Masonic Cancer Center, University of MinnesotaTerminatedSickle Cell Disease | Thalassemia | Cerebral Adrenoleukodystrophy | Inherited Metabolic Disorders | High Risk Hematologic DisordersUnited States
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National Center for Research Resources (NCRR)University of California, Los AngelesCompletedThrombocytopenia | Inborn Errors of Metabolism | Metachromatic Leukodystrophy | Fanconi's Anemia | Thalassemia Major | Pure Red-Cell AplasiaUnited States
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Masonic Cancer Center, University of MinnesotaRecruitingHurler Syndrome | Sphingolipidoses | Peroxisomal Disorders | Metachromatic Leukodystrophy | Alpha-Mannosidosis | Hunter Syndrome | Mucopolysaccharidosis Disorders | Maroteaux Lamy Syndrome | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Glycoprotein Metabolic Disorders | Recessive Leukodystrophies | Globoid... and other conditionsUnited States
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University of Mississippi Medical CenterIndena S.p.AWithdrawnLipid Metabolism DisordersUnited States
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BioMarin PharmaceuticalTerminatedMorquio A Syndrome | MPS IVA | Mucopolysaccharidosis IVAUnited States, United Kingdom, Canada, Germany
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ShireCompletedMucopolysaccharidosis (MPS)United Kingdom, Netherlands
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Columbia UniversityCompletedMetabolism Disorder, GlucoseUnited States
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Birmingham Women's and Children's NHS Foundation...BioMarin PharmaceuticalWithdrawnMucopolysaccharidosis IV | Morquio A DiseaseUnited Kingdom
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The University of Texas Health Science Center at...National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)CompletedGlucose Metabolism Disorders | Metabolic Diseases | Lipid Metabolism DisordersUnited States
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REGENXBIO Inc.Active, not recruitingHurler Syndrome | Hurler-Scheie Syndrome | Mucopolysaccharidosis Type I (MPS I)United States, Brazil, Israel
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University of ChicagoNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedKrabbe Disease | Mucopolysaccharidosis Type II (MPS II) | Mucopolysaccharidosis Type I (MPS I) | Mucopolysaccharidosis Type III (MPS III) | Mucopolysaccharidosis Type VI (MPS VI)United States
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Alexion PharmaceuticalsTerminatedMucopolysaccharidosis III, Type B (MPS IIIB) | Sanfilippo BUnited Kingdom
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Alexion PharmaceuticalsCompletedMPS IIIB (Sanfilippo B Syndrome)United Kingdom
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BiogenCompletedPrimary Inherited ErythromelalgiaUnited States
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Washington University School of MedicineUnited States Department of DefenseRecruitingGlucose Metabolism DisordersUnited States
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Tasly Pharmaceuticals, Inc.Natural Immune Systems IncUnknownGlucose Metabolism DisordersUnited States
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Washington University School of MedicineCompletedGlucose Metabolism DisordersUnited States
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Victor GordeukWeill Medical College of Cornell UniversityCompleted
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Allievex CorporationCompletedMucopolysaccharidosis Type IIIB | Mucopolysaccharidosis Type 3 B | MPS III B | MPS 3 BUnited States, Spain, Turkey, Taiwan, Australia, Colombia, Germany, United Kingdom
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Albert Einstein College of MedicineCompletedChronic Kidney Disease | End-stage Renal Disease | Metabolic AcidosisUnited States
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Luye Pharma Group Ltd.CompletedLipid Metabolism DisorderUnited States
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Keryx BiopharmaceuticalsCollaborative Study Group (CSG)CompletedEnd-Stage Renal Disease | HyperphosphatemiaUnited States, Puerto Rico
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Fairview University Medical CenterUnknownMucopolysaccharidosis I | Niemann-Pick Disease | Mucopolysaccharidosis VI | Wolman Disease | Adrenoleukodystrophy | Metachromatic Leukodystrophy | Pulmonary Complications | Fucosidosis | Globoid Cell Leukodystrophy | Gaucher's Disease | Mannosidosis | I Cell DiseaseUnited States
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IntraBio IncActive, not recruitingNiemann-Pick Disease, Type CUnited States, Australia, United Kingdom, Germany, Slovakia, Netherlands, Czechia, Switzerland
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ZevraDenmarkActive, not recruitingNiemann-Pick Disease, Type CUnited States, Spain, United Kingdom, Denmark, France, Germany, Italy, Poland, Switzerland
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ShireCompleted