Clinical Trials on GM3 Gangliosidosis
Total 80 results
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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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Azafaros A.G.Active, not recruitingGM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff DiseaseUnited States, Brazil, France, Germany, Italy, United Kingdom
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Idorsia Pharmaceuticals Ltd.CompletedGM2 Gangliosidosis | GM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease | Gaucher Disease, Type 2 | AB Variant Gangliosidosis GM2United States, Spain, Germany, Italy, Belgium, Brazil, France, Portugal, Switzerland, United Kingdom
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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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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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The Hospital for Sick ChildrenActelionCompletedGangliosidoses GM2Canada
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National Human Genome Research Institute (NHGRI)Sio Gene TherapiesRecruitingGangliosidosis | Lysosomal Diseases | GM1United States
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Dr. Anupam SehgalTaysha Gene Therapies, Inc.; GlycoNetActive, not recruitingInfantile GM2 Gangliosidosis (Disorder)Canada
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Children's National Research InstituteActelionCompletedSandhoff Disease | GM2 Gangliosidoses | Tay-SachsUnited States
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Gemma BiotherapeuticsActive, not recruitingGM1 Gangliosidosis | GM1 Gangliosidosis, Type I | GM1 Gangliosidosis, Type 2 | Beta-Galactosidase-1 (GLB1) DeficiencyUnited States, United Kingdom, Turkey, Brazil
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IntraBio IncCompletedGM2 Gangliosidosis | Tay-Sachs Disease | Sandhoff DiseaseUnited States, Germany, Spain, United Kingdom
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Tehran University of Medical SciencesMashhad University of Medical Sciences; Kashan University of Medical SciencesRecruitingEffects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD) (EMTISTD)Supportive Care | GM2 GangliosidosisIran, Islamic Republic of
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Terence FlotteActive, not recruitingGM2 Gangliosidosis | Tay Sachs Disease | Sandhoff DiseaseUnited States
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Massachusetts General HospitalActive, not recruiting
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The Hospital for Sick ChildrenCompletedGangliosidoses, GM2 | Tay-Sachs Disease | Sandhoff DiseaseCanada
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University of PennsylvaniaPassage Bio, Inc.RecruitingGM1 GangliosidosisUnited States, Turkey, Brazil, Canada, United Kingdom
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LYSOGENETerminatedGM1 GangliosidosisUnited Kingdom, United States, France
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LYSOGENECasimir, LLC; Cure GM1 FoundationCompleted
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University of British ColumbiaMedical University of Graz; Hospital de Clinicas de Porto AlegreUnknownMorquio B Disease | GM1 Gangliosidosis Type IIICanada
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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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University of PittsburghRecruitingLysosomal Storage Diseases | Leukodystrophy | Niemann-Pick Diseases | Batten Disease | Gaucher Disease | Krabbe Disease | Alpha-Mannosidosis | Osteopetrosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | GM1 Gangliosidoses | MPS I | MPS II | Vanishing White Matter Disease | Multiple Sulfatase Deficiency... and other conditionsUnited States
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Azafaros A.G.Active, not recruitingNiemann-Pick Disease, Type C | GM2 GangliosidosisBrazil
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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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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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Sun Yat-sen UniversityTerminatedBreast Cancer | Cognitive Impairment | Brain Metastases | Radiotherapy Side Effect | Gangliosidosis, GM1China
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Terence FlotteMassachusetts General Hospital; University of Massachusetts, WorcesterCompletedTay-Sachs Disease | Sandhoff DiseaseUnited States
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DDC Clinic - Center for Special Needs ChildrenUnknownGM3 Synthase Deficiency
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Genzyme, a Sanofi CompanyTerminatedTay-Sachs Disease | Sandhoff DiseaseUnited States, Argentina, Czechia, Portugal, Russian Federation, Turkey, United Kingdom, Brazil, France, Germany, Italy, Japan, Spain
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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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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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National Center for Research Resources (NCRR)Icahn School of Medicine at Mount SinaiCompletedMetabolism, Inborn Errors | Leukodystrophy, Globoid Cell | Tay-Sachs Disease | Porphyria, ErythropoieticUnited States
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National Institute of Allergy and Infectious Diseases...Terminated
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McGill University Health Centre/Research Institute...CompletedFamilial Dysautonomia | Tay Sachs Disease | Canavan DiseaseCanada
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xCuresRecruitingAmyotrophic Lateral Sclerosis | Amyloidosis | Myasthenia Gravis | Cystic Fibrosis | Sickle Cell Disease | Duchenne Muscular Dystrophy | Huntington Disease | Rare Diseases | Beta-Thalassemia | Ehlers-Danlos Syndrome | Gaucher Disease | Pompe Disease | Alpha-Thalassemia | GM1 Gangliosidosis | Early-Onset Alzheimer Disease and other conditionsUnited States
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CENTOGENE GmbH RostockWithdrawnSeizures | Mental Retardation | Hepato-splenomegaly | Dysostosis MultiplexEgypt, Germany, India, Sri Lanka
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University Hospital, ToursInstitut National de la Santé Et de la Recherche Médicale, FranceCompletedValidate the French Version of GMS PackageFrance
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Shanghai Chest HospitalUnknownNSCLC | SBRT | GM-CSFChina
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Alexion Pharmaceuticals, Inc.RecruitingGeneralized Myasthenia Gravis (gMG) | Refractory gMGChina
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Regeneron PharmaceuticalsNot yet recruitingLong Term Safety Following GM T Cell Therapy
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Joanne Kurtzberg, MDThe Marcus FoundationRecruitingMucopolysaccharidosis II | Brain Diseases, Metabolic, Inborn | Adrenoleukodystrophy | Batten Disease | Leukodystrophy, Metachromatic | Leukodystrophy, Globoid Cell | Alpha-Mannosidosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | Neimann Pick Disease | Sanfilippo MucopolysaccharidosesUnited 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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Children's Hospital of PhiladelphiaIllumina, Inc.CompletedMucopolysaccharidoses | 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 Gangliosidosis | Zellweger... 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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PATHCompletedMeasles Antibody Seroconversion | Rubella Antibody Seroconversion | Rotavirus Geometric Mean Titer (GMT) | Rotavirus Immunoglobulin A (IgA) SeropositivityBangladesh
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National Cancer Institute (NCI)CompletedUntreated Adult Acute Myeloid Leukemia | Adult Acute Promyelocytic Leukemia (M3) | Childhood Acute Promyelocytic Leukemia (M3) | Adult Acute Myeloid Leukemia With t(15;17)(q22;q12) | Untreated Childhood Acute Myeloid Leukemia and Other Myeloid MalignanciesUnited States
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Children's Oncology GroupNational Cancer Institute (NCI)CompletedAdult Acute Promyelocytic Leukemia (M3) | Childhood Acute Promyelocytic Leukemia (M3) | Adult Acute Myeloid Leukemia With t(15;17)(q22;q12) | Adult Acute Myeloid LeukemiaUnited States
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University of JenaUnknownSevere Sepsis or Septic Shock (ICD-10-GM, R65.0, R65.1)Germany
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Qilu Pharmaceutical Co., Ltd.Not yet recruitingTo Evaluate the Effectiveness of GM1 in the Prevention of Albumin-paclitaxel Therapy Induced Peripheral Neuropathy in Breast Cancer Patients
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National Institute of Allergy and Infectious Diseases...CompletedPGM3 Deficiency | Eosinophilic and/or Atopic Dermatitis | OSMR Deficiency | Primary Localized Cutaneous Amyloidosis | Hereditary Alpha-tryptasemiaUnited States
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Chongbo ZhaoGracell Biotechnologies (Shanghai) Co., Ltd.Not yet recruitingGeneralized Myasthenia Gravis (gMG)China