Clinical Trials on Late Infantile Metachromatic Leukodystrophy
Total 55 results
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ShireTerminatedLate Infantile Metachromatic LeukodystrophyDenmark
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ShireCompletedLate Infantile Metachromatic LeukodystrophyDenmark
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Assistance Publique - Hôpitaux de ParisEuropean Leukodystrophy AssociationCompletedLate Infantile Metachromatic LeukodystrophyFrance
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ShireCompletedMetachromatic Leukodystrophy (MLD)Australia, Denmark, Japan, France, Germany
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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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Shenzhen Geno-Immune Medical InstituteUnknown
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TakedaWithdrawnMetachromatic Leukodystrophy (MLD)Spain
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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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ShireCompletedMetachromatic Leukodystrophy (MLD)Denmark
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ShireTerminatedMetabolic Diseases | Brain Diseases | Central Nervous System Diseases | Nervous System Diseases | Demyelinating Diseases | Genetic Diseases, Inborn | Metabolism, Inborn Errors | Lysosomal Storage Diseases | Lipid Metabolism Disorders | Sphingolipidoses | Hereditary Central Nervous System Demyelinating Diseases and other conditionsUnited States, Denmark, France, Argentina, Belgium, Brazil, Canada, Germany, Japan, Turkey
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The Cooper Health SystemCompletedMetachromatic LeukodystrophyUnited States
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Talaris Therapeutics Inc.Duke UniversityNo longer availableMetachromatic LeukodystrophyUnited States
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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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Institut National de la Santé Et de la Recherche...Assistance Publique - Hôpitaux de Paris; European Leukodystrophy AssociationActive, not recruitingMetachromatic LeukodystrophyFrance
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Orchard TherapeuticsOspedale San RaffaeleActive, not recruitingLysosomal Storage Diseases | Metachromatic LeukodystrophyItaly
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Assistance Publique - Hôpitaux de ParisShire; Zymenex A/S; European Leukodystrophy AssociationCompletedMetachromatic LeukodystrophyFrance
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Orchard TherapeuticsOspedale San RaffaeleActive, not recruitingLysosomal Storage Disease | Metachromatic LeukodystrophyItaly
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Orchard TherapeuticsOspedale San RaffaeleActive, not recruitingLysosomal Storage Disease | Metachromatic LeukodystrophyItaly
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CENTOGENE GmbH RostockWithdrawnMuscle Weakness | Peripheral NeuropathyEgypt, Germany, India, Sri Lanka
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Talaris Therapeutics Inc.Duke UniversityNo longer availableMetachromatic LeukodystrophyUnited 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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Bellicum PharmaceuticalsNo longer availableHurler Syndrome | Inborn Errors of Metabolism | Metachromatic Leukodystrophy | Inherited Metabolic Disorder | Lysosomal Storage DisorderUnited States
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Zymenex A/SCompletedInfantile Globoid Cell LeukodystrophyUnited States
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Shenzhen Second People's HospitalGuangzhou Women and Children's Medical Center; Shenzhen UniversityRecruitingAdrenoleukodystrophy | Metachromatic LeukodystrophyChina
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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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Talaris Therapeutics Inc.Duke UniversityTerminatedNiemann-Pick Disease | Alpha-mannosidosis | Tay Sachs Disease | Sandhoff Disease | Metachromatic Leukodystrophy (MLD) | Hurler-Scheie Syndrome | Hurler Syndrome (MPS I) | Hunter Syndrome (MPS II) | Sanfilippo Syndrome (MPS III) | Krabbe Disease (Globoid Leukodystrophy) | Adrenoleukodystrophy (ALD and AMN) | Pelizaeus...United States
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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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Masonic Cancer Center, University of MinnesotaCompletedNiemann-Pick Disease | Wolman Disease | Adrenoleukodystrophy | Batten Disease | Metachromatic Leukodystrophy | GM1 Gangliosidosis | Fucosidosis | Globoid Cell Leukodystrophy | Gaucher's Disease | Tay Sachs Disease | Sandhoff DiseaseUnited States
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Masonic Cancer Center, University of MinnesotaCompletedWolman Disease | Adrenoleukodystrophy | Metachromatic Leukodystrophy | Sanfilippo Syndrome | GM1 Gangliosidosis | Globoid Cell Leukodystrophy | Tay Sachs Disease | Sandhoffs Disease | I-Cell DiseaseUnited 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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Assistance Publique - Hôpitaux de ParisEuropean Leukodystrophy AssociationWithdrawnDeficiency Disease | Canavan Disease | Infantile | Aspartoacylase | Leukodystrophy, Spongiform
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Amicus TherapeuticsCompletedVariant Late-Infantile Neuronal Ceroid LipofuscinosisUnited States
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BioMarin PharmaceuticalActive, not recruitingLate-Infantile Neuronal Ceroid Lipofuscinosis Type 2United States
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Weill Medical College of Cornell UniversityNational Institute of Neurological Disorders and Stroke (NINDS); National Institutes... and other collaboratorsCompletedBatten Disease | Late Infantile Neuronal Ceroid LipofuscinosisUnited States
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Weill Medical College of Cornell UniversityTerminatedBatten Disease | Late Infantile Neuronal Ceroid LipofuscinosisUnited States
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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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Weill Medical College of Cornell UniversityNathan's Battle FoundationCompletedBatten Disease | Late Infantile Neuronal Ceroid LipofuscinosisUnited States
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Weill Medical College of Cornell UniversityNational Institutes of Health (NIH)CompletedBatten Disease | Late-Infantile Neuronal Ceroid LipofuscinosisUnited States
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Weill Medical College of Cornell UniversityCompletedBatten Disease | Late Infantile Neuronal Ceroid LipofuscinosisUnited States
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REGENXBIO Inc.WithdrawnLate-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)
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BioMarin PharmaceuticalCompletedBatten Disease | CLN2 Disease | Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 | Jansky-Bielschowsky DiseaseUnited States, Italy, Germany, United Kingdom
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BioMarin PharmaceuticalCompletedBatten Disease | CLN2 Disease | Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 | Jansky-Bielschowsky Disease | CLN2 DisorderUnited States, Germany, Italy, United Kingdom
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BioMarin PharmaceuticalCompletedBatten Disease | CLN2 Disease | Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 | Jansky-Bielschowsky Disease | CLN2 DisorderUnited States, Germany, Italy, United Kingdom
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Laylay Mohammed Khalleefah AlhibshiCompletedPregnancy Rate | Endometrial ThicknessEgypt
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Weill Medical College of Cornell UniversityNathan's Battle FoundationCompletedBatten Disease | Late-Infantile Neuronal Ceroid Lipfuscinosis
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Albert Einstein College of MedicineEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationMucopolysaccharidosis II | Fabry Disease | Niemann-Pick Disease, Type C | Mucopolysaccharidosis VI | Lysosomal Acid Lipase Deficiency | Metachromatic Leukodystrophy | Gaucher Disease | Cerebrotendinous Xanthomatosis | Mucopolysaccharidosis IV A | GM1 Gangliosidosis | Mucopolysaccharidosis VII | Acid Sphingomyelinase... and other conditionsUnited States
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New York Medical CollegeCompletedMucopolysaccharidosis I | Myelodysplastic Syndrome | Acute Lymphocytic Leukemia | Acute Myelogenous Leukemia | Niemann-Pick Disease | Mucopolysaccharidosis VI | Wolman Disease | Adrenoleukodystrophy | Batten Disease | Metachromatic Leukodystrophy | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Fucosidosis | Gaucher... and other conditionsUnited States
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Children's Hospital of PhiladelphiaEli Lilly and Company; University of Pennsylvania; Takeda; National Institute of... and other collaboratorsRecruitingMucopolysaccharidoses | Leukoencephalopathies | Leukodystrophy | Adrenoleukodystrophy | Adrenomyeloneuropathy | X-linked Adrenoleukodystrophy | Gangliosidoses | Metachromatic Leukodystrophy | Krabbe Disease | Refsum Disease | Cadasil | Sjogren-Larsson Syndrome | Allan-Herndon-Dudley Syndrome | White Matter Disease | GM2... and other conditionsUnited States
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University of Erlangen-Nürnberg Medical SchoolRecruitingPompe Disease (Late-onset) | Pompe Disease | Pompe's Disease Juvenile Onset | Pompe Disease Infantile-OnsetGermany