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- US Clinical Trials Registry
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Clinical Trials on Lysosome Alteration
Total 1177 results
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University of Erlangen-Nürnberg Medical SchoolRecruitingPompe Disease (Late-onset) | Pompe Disease | Pompe's Disease Juvenile Onset | Pompe Disease Infantile-OnsetGermany
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ShireTakeda Development Center Americas, Inc.Active, not recruitingMetachromatic Leukodystrophy (MLD)United States, Canada, Belgium, Israel, Netherlands, United Kingdom, Germany, Spain, Japan, Brazil, Argentina, France, Greece, Italy
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TakedaWithdrawnMetachromatic Leukodystrophy (MLD)Spain
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Duke UniversityGenzyme, a Sanofi CompanyCompletedNeuropathy | Myopathy | Glycogen Storage Disease Type II (Late-onset Pompe Disease)United States
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ShireCompletedMetachromatic Leukodystrophy (MLD)Denmark
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ShireCompletedLate Infantile Metachromatic LeukodystrophyDenmark
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsTerminatedTay-Sachs Disease | Sandhoff Disease | GM1 Gangliosidoses | GM2 GangliosidosesUnited 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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Assistance Publique - Hôpitaux de ParisEuropean Leukodystrophy AssociationCompletedLate Infantile Metachromatic LeukodystrophyFrance
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Patricia I. Dickson, M.D.National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); University... and other collaboratorsCompletedMucopolysaccharidosis I | Scheie Syndrome | Lysosomal Storage Disease | Cognitive Decline | Hurler-Scheie SyndromeUnited States
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Children's Hospital Medical Center, CincinnatiCompletedOsteopenia | Gaucher's DiseaseIsrael, United States
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedTay Sachs Disease | Sandhoff Disease | Late Onset Tay Sachs DiseaseUnited States
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Université du Québec à Trois-RivièresUniversité de Montréal; Fondation Santé Trois-Rivières; Centre intégré universitaire...Not yet recruitingDiabetic Foot | Physical Inactivity | Cardiopulmonary
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University of PittsburghWithdrawnMetachromatic LeukodystrophyUnited States
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SanofiActive, not recruitingGaucher's Disease Type III | Gaucher's Disease Type ICanada, Argentina, France, Italy, Japan, Russian Federation, Spain, Sweden, Turkey, United Kingdom
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ShireTakeda Development Center Americas, Inc.Active, not recruitingMetachromatic Leukodystrophy (MLD)Denmark, Germany, Japan, Australia, France, Brazil, Czechia
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National Organization for Rare DisordersFood and Drug Administration (FDA)Active, not recruitingMetachromatic LeukodystrophyUnited States
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Institut National de la Santé Et de la Recherche...Assistance Publique - Hôpitaux de Paris; European Leukodystrophy AssociationActive, not recruitingMetachromatic LeukodystrophyFrance
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Shenzhen Geno-Immune Medical InstituteUnknown
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Royal Brompton & Harefield NHS Foundation TrustGenzyme, a Sanofi CompanyUnknownPompe's Disease
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Duke UniversityCompletedHemoglobinopathies | Thalassemia | Lysosomal Storage Disease | Immunodeficiencies | Inborn Errors of Metabolism | Sickle Cell | Non Malignant Disorders | Congenital Marrow FailuresUnited States
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II (GSD-II) | Pompe Disease | Glycogenesis 2 Acid Maltase DeficiencyUnited States, Canada, Australia
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Jordi Perez LopezHospital Vall d'HebronUnknown
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Chiesi Farmaceutici S.p.A.ICON plcNot yet recruiting
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UniQure Biopharma B.V.RecruitingFabry DiseaseUnited States
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Brunel UniversityRoyal Free Hospital NHS Foundation TrustRecruiting
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Institut National de la Santé Et de la Recherche...Not yet recruitingFabry Disease
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsRecruitingGM2 Gangliosidosis | GM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease | Late Onset Tay-Sachs DiseaseUnited States
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ShireActive, not recruiting
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SanofiCompletedFabry DiseaseUnited Arab Emirates
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Amicus TherapeuticsActive, not recruitingFabry DiseaseUnited States, United Kingdom
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Idorsia Pharmaceuticals Ltd.Active, not recruitingFabry DiseaseUnited States, United Kingdom, Norway, Germany, Australia, Canada, Spain, Switzerland, Austria, Belgium, France, Netherlands, Poland
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Chiesi ItaliaCompleted
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ProtalixChiesi Farmaceutici S.p.A.CompletedFabry DiseaseAustralia, Netherlands, United Kingdom, Canada, Czechia, Norway, Slovenia
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Freeline TherapeuticsTerminatedLysosomal Storage Diseases | Fabry DiseaseUnited States, Norway, Germany, Austria, United Kingdom, Italy, Canada
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Manchester University NHS Foundation TrustNorthern Care Alliance NHS Foundation TrustActive, not recruiting
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Amicus TherapeuticsCompletedFabry DiseaseJapan, Taiwan, Turkey, United States, Brazil
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Genzyme, a Sanofi CompanyCompletedGlycogen Storage Disease Type II (GSD-II) | Acid Maltase Deficiency Disease | Pompe DiseaseUnited States
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ShireNo longer available
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CENTOGENE GmbH RostockShire Human Genetic Therapies, Inc.CompletedCerebrovascular AccidentAustria, Belgium, Croatia, Finland, France, Georgia, Germany, Ireland, Italy, Malta, Poland, Portugal, Spain, United Kingdom
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Genzyme, a Sanofi CompanyCompletedFabry DiseaseFrance, Poland, United States, Russian Federation, United Kingdom
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Lysosomal and Rare Disorders Research and Treatment...SanofiUnknown
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Amicus TherapeuticsCompletedOpen-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry DiseaseFabry DiseaseUnited States, Austria, United Kingdom, Argentina, Australia, Belgium, Brazil, Canada, Denmark, Egypt, France, Italy, Japan, Spain, Turkey
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Genzyme, a Sanofi CompanyTerminatedFabry DiseaseAustria, Brazil, France, United States, Argentina, Belgium, Canada, Finland, Hungary, Norway, Poland, Spain, Taiwan, United Kingdom
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Boston Children's HospitalWithdrawn
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University Hospital, BordeauxCompleted
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Groupe Hospitalier Diaconesses Croix Saint-SimonTerminated
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Amicus TherapeuticsCompletedFabry DiseaseFrance, Austria, United States, Belgium, Japan, United Kingdom, Denmark, Brazil, Australia, Italy
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Amicus TherapeuticsCompletedFabry DiseaseUnited States, Italy, Spain, Turkey, Brazil, Canada, United Kingdom, France, Poland, Australia, Egypt, Argentina, Denmark