Clinical Trials on Lysosomal Storage Diseases, Nervous System
Total 193924 results
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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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Johannes Gutenberg University MainzUnknownMucopolysaccharidosis | Fabry DiseaseGermany
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Amicus TherapeuticsEngage Health Inc.CompletedFabry DiseaseUnited States
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Baylor Research InstituteNational Institute of Neurological Disorders and Stroke (NINDS)Completed
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AlexionCompletedWolman Disease | Lysosomal Acid Lipase DeficiencyUnited States, Canada, United Kingdom, Italy, France
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Ankara UniversityAlexion PharmaceuticalsCompleted
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Assistance Publique - Hôpitaux de ParisCystinosis Research FoundationRecruiting
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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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Alexion PharmaceuticalsCompletedWolman Disease | Lysosomal Acid Lipase DeficiencyUnited Kingdom, France, United States, Egypt, Ireland
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SanofiRecruitingAcid SphingoMyelinase Deficiency | Gaucher Disease, SplenomegalyFrance
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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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National Human Genome Research Institute (NHGRI)Withdrawn
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University of MinnesotaGE HealthcareCompletedParkinson Disease | Gaucher Disease | Idiopathic Rapid Eye Movement Sleep DisorderUnited States
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Washington... and other collaboratorsRecruiting
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Rigshospitalet, DenmarkUnknownCarbohydrate Metabolism, Inborn Errors | Metabolism, Inborn Errors | Lipid Metabolism, Inborn Errors | Glycogen Storage Disease Type II | Glycogen Storage Disease Type V | VLCAD Deficiency | Glycogen Storage Disease Type III | Phosphoglycerate Kinase Deficiency | Neutral Lipid Storage Disease | Carnitine... and other conditionsDenmark
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Fondazione Policlinico Universitario Agostino Gemelli...Recruiting
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Francisco Marín OrtuñoHospital General Universitario Elche; Hospital Universitario San Juan de AlicanteUnknownRight Ventricular Hypertrophy | Fabry Disease, Cardiac Variant
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Eline C. B. EskesRecruitingAsmd, Visceral TypeNetherlands
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Chang Gung Memorial HospitalRecruitingFabry DiseaseTaiwan
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Duke UniversitySuspendedLysosomal Storage Diseases | Genetic Disease | Inborn Errors of Metabolism | Glycogen Storage Disease | Storage DiseaseUnited States
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Baylor Research InstituteCompleted
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Centre Hospitalier Universitaire de NiceUnknown
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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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Baylor Research InstituteShireUnknownQuality of Life | Renal Insufficiency | Cardiac EventUnited States
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University Hospital HeidelbergCompletedFabry Disease | Chronic Abdominal Pain | Chronic Pain in the ExtremitiesGermany
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CENTOGENE GmbH RostockWithdrawnSeizures | Mental Retardation | Hepato-splenomegaly | Dysostosis MultiplexEgypt, Germany, India, Sri Lanka
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Shenzhen Second People's HospitalGuangzhou Women and Children's Medical Center; Shenzhen UniversityRecruitingAdrenoleukodystrophy | Metachromatic LeukodystrophyChina
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SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt)RecruitingGangliosidoses | Galactosialidosis | Sialidosis | GM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease | Morquio B Disease | Gm2-Gangliosidosis, Variant B1 | GM2 Activator DeficiencyGermany
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SanofiPulse Infoframe Ltd.RecruitingNiemann-Pick Diseases | Acid Sphingomyelinase DeficiencyUnited States
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National Eye Institute (NEI)CompletedNiemann Pick DiseasesUnited States, United Kingdom
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Sun Yat-sen UniversityRecruitingBreast Cancer | Cognitive Impairment | Brain Metastases | Radiotherapy Side Effect | Gangliosidosis, GM1China
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Mandos LLCTerminatedNiemann-Pick Disease, Type CCosta Rica
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Klinikum Wels-GrieskirchenGenzyme, a Sanofi CompanyCompletedFocus of Study: Prevalence of Fabry Disease in CKD PopulationAustria
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ActelionCompleted
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Vtesse, LLC, a Mallinckrodt Pharmaceuticals CompanyWithdrawnNiemann-Pick Disease, Type C
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Amicus TherapeuticsRecruitingFabry DiseaseSpain, United States, Japan, Australia, France, Portugal
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University Hospital, GhentCompletedBelgian Screening Project for the Detection of Anderson-Fabry Disease in Hypertrophic CardiomyopathyLeft Ventricular HypertrophyBelgium
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AlexionRecruitingWolman Disease | Cholesterol Ester Storage Disease | Lysosomal Acid Lipase Deficiency | Acid Cholesteryl Ester Hydrolase Deficiency, Type 2 | Acid Lipase Deficiency | LIPA Deficiency | LAL-DeficiencyFrance, Belgium, United States, Spain, Germany, Greece, Israel, Italy, Slovenia, United Kingdom, Brazil, Canada, Denmark, Australia, Croatia, Czechia, Ireland, Mexico, Netherlands, Poland, Portugal, Saudi Arabia
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Qilu Hospital of Shandong UniversityRecruitingFabry Disease | Hypertrophic Cardiomyopathy | Left Ventricular HypertrophyChina
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University of ZurichCompletedFabry DiseaseSwitzerland
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University Hospital, MontpellierRecruiting
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University of Roma La SapienzaAlexion PharmaceuticalsRecruitingNon-Alcoholic Fatty Liver DiseaseItaly
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Mandos LLCTerminatedNiemann-Pick Disease, Type CUnited States, Australia, United Kingdom, Turkey, Switzerland, Germany, New Zealand, Singapore, France
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Mandos LLCCompletedNiemann-Pick Disease, Type CUnited States, Australia, United Kingdom, Germany, France, New Zealand, Singapore, Spain, Turkey
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Assistance Publique - Hôpitaux de ParisRecruitingAdrenoleukodystrophy | Adrenomyeloneuropathy | Metachromatic Leukodystrophy | Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented GliaFrance
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University Of PerugiaRecruitingObesity | Ketosis | Lysosomal Acid Lipase Deficiency | Ketogenic DietingItaly
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LMU KlinikumCompletedHealthy | Myotonic Dystrophy Type 2 | Pompe Disease (Late-onset) | Spinal Muscular Atrophy Type 3 | Myotonic Dystrophy Type 1 (DM1) | Facioscapulohumeral Muscular Dystrophy 1 | Inclusion Body Myositis, SporadicGermany
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National Institute of Neurological Disorders and...CompletedBrain Neoplasm | Alzheimer's Disease | Niemann Pick DiseaseUnited States
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Wuerzburg University HospitalAcademisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA); St George... and other collaboratorsRecruitingFabry Disease | Rare Diseases | Hypertrophic Cardiomyopathy | Fabry Disease, Cardiac VariantGermany
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Azafaros A.G.Active, not recruitingNiemann-Pick Disease, Type C | GM2 GangliosidosisBrazil