- ICH GCP
- US Clinical Trials Registry
- Search trials
Clinical Trials on Inherited Metabolic Storage Diseases in United States
Total 26820 results
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UniQure Biopharma B.V.RecruitingFabry DiseaseUnited States
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Lysosomal and Rare Disorders Research and Treatment...SanofiUnknown
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Boston Children's HospitalWithdrawn
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National Institute of Neurological Disorders and...Completed
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ShireCompletedGaucher DiseaseUnited States, Spain, United Kingdom, Israel
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National Organization for Rare DisordersFood and Drug Administration (FDA)Active, not recruitingMetachromatic LeukodystrophyUnited States
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University of PittsburghActive, not recruitingMetachromatic LeukodystrophyUnited States
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University of California, San DiegoCalifornia Institute for Regenerative Medicine (CIRM); Cystinosis Research...Active, not recruiting
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedParkinson's Disease | Gaucher's DiseaseUnited States
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Baylor Research InstituteNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsUnknown
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Amicus TherapeuticsRecruiting
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National Institute of Neurological Disorders and...CompletedFabry DiseaseUnited States
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O & O Alpan LLCCompletedFabry DiseaseUnited States
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Ohio State UniversityGenzyme, a Sanofi CompanyCompleted
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National Institute of Neurological Disorders and...Completed
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National Institute of Neurological Disorders and...Completed
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Genzyme, a Sanofi CompanyActive, not recruitingGaucher Disease Type 1 | Gaucher Disease Type 3Germany, United States, Japan, United Kingdom
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Alexion PharmaceuticalsCompletedLysosomal Acid Lipase DeficiencySpain, Germany, Italy, United States, Croatia, Canada, Russian Federation, Denmark, United Kingdom, Belgium, Mexico, Australia, Netherlands, Brazil, Turkey
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Zymenex A/SCompletedInfantile Globoid Cell LeukodystrophyUnited States
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Amicus TherapeuticsCompletedGaucher Disease | Gaucher Disease, Type 1 | Type 1 Gaucher DiseaseUnited Kingdom, Israel, South Africa, United States
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ShireCompletedGaucher DiseaseUnited States, Israel, United Kingdom, Poland, Spain
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Alexion PharmaceuticalsTerminatedLysosomal Acid Lipase DeficiencyUnited States
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National Institute of Neurological Disorders and...National Institute of Neurological Disorders and Stroke@@@Shire Human Genetic...Completed
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University of California, San FranciscoRecruitingMucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VI | Wolman Disease | Pompe Disease Infantile-Onset | Mucopolysaccharidosis IV A | Mucopolysaccharidosis VII | Neuronopathic Gaucher DiseaseUnited States
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Genzyme, a Sanofi CompanyRecruitingGlycogen Storage Disease Type II | Pompe DiseaseHungary, Croatia, Serbia, United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, Chile, China, Colombia, Denmark, France, Germany, Greece, Hong Kong, India, Indonesia, Israel, Italy, Japan, Jordan, Korea, Republic of, Kuwait and more
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New York Stem Cell Foundation Research InstituteSilverstein FoundationRecruitingHealthy | Parkinson Disease | Gaucher Disease | GBA Gene MutationUnited States
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Massachusetts General HospitalGenzyme, a Sanofi Company; Scott and White Hospital & ClinicTerminatedKidney Diseases | Genetic Diseases, X-Linked | End Stage Renal Disease | ESRD | Fabry Disease | Rare DiseasesUnited States
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Neurogene Inc.TerminatedLysosomal Storage Diseases | Aspartylglucosaminuria | Aspartylglucosamidase (AGA) DeficiencyUnited States
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Tarix PharmaceuticalsWithdrawnHematologic Malignancy | Cord Blood Transplant | Inherited Metabolic DiseaseUnited States
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Genzyme, a Sanofi CompanyCompletedGaucher Disease, Type 1United States, United Kingdom, Bulgaria, Canada, Colombia, India, Israel, Lebanon, Mexico, Russian Federation, Serbia, Tunisia
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University of UtahGenzyme, a Sanofi CompanyCompletedFabry DiseaseUnited States
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O & O Alpan LLCUnknownLysosomal Storage DisordersUnited States
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Freeline TherapeuticsRecruitingGaucher Disease, Type 1Spain, Israel, United States, United Kingdom, Brazil, Germany, Paraguay
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ActelionCompletedGaucher Disease Type 1United States, Taiwan, Canada, Spain, Australia, France, Brazil, Hungary, Czechia, Germany, Italy, Netherlands, United Kingdom
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Exsar CorporationThe Hospital for Sick Children; NYU Langone Health; University Hospitals Cleveland...WithdrawnG(M2) Ganglioside | Tay-Sachs Disease Ganglioside | Sandhoff Disease GangliosideUnited States, Canada
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National Institute of Neurological Disorders and...CompletedHealthy | Fabry Disease | Cerebrovascular AccidentUnited States
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Amicus TherapeuticsCompletedGaucher Disease | Gaucher Disease, Type 1 | Type 1 Gaucher DiseaseUnited States
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Cedars-Sinai Medical CenterNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsUnknown
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Azafaros A.G.Active, not recruitingGM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff DiseaseUnited States, Brazil, France, Germany, Italy, United Kingdom
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Lysosomal and Rare Disorders Research and Treatment...UnknownLysosomal Storage DiseasesUnited States
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O & O Alpan LLCUnknownFabry Disease | Niemann-Pick Disease | Gaucher Disease | Pompe Disease | Lysosomal Storage DisordersUnited States
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University of California, San FranciscoRecruitingSphingolipidoses | Enzyme DeficiencyUnited States
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Eunice Kennedy Shriver National Institute of Child...Completed
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Genzyme, a Sanofi CompanyApproved for marketingGlycogen Storage Disease Type II (GSD-II) | Acid Maltase Deficiency Disease | Glycogenosis 2 | Pompe Disease (Late-Onset)United States
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Alexion PharmaceuticalsCompletedLysosomal Acid Lipase DeficiencyFrance, Poland, United Kingdom, Spain, Mexico, Turkey, Japan, Australia, Russian Federation, United States, Germany, Italy, Czechia, Argentina, Croatia
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National Human Genome Research Institute (NHGRI)TerminatedPulmonary Fibrosis | Metabolic Disease | Oculocutaneous Albinism | Hermansky-Pudlak Syndrome (HPS) | Platelet Storage Pool DeficiencyUnited States
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SanofiRecruitingFabry's DiseaseUnited States
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National Institute of Neurological Disorders and...CompletedFabry DiseaseUnited States