Gangliosidoses
Gangliosidoses
Explore linked clinical trials. Standard reference: MESH:M000898816 results · Page 1 of 1
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders | Bone Diseases, Metabolic, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyskinesias, GM1 gangliosidosis, GM2 gangliosidosis, Gangliosidoses, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Myoclonus, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, bone disorder, brain disorder, central nervous system disorder, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, mucolipidosis, musculoskeletal system disorder, nervous system disorder, sphingolipidosis | Recruiting | Not stated | 2026-10-02 | NCT00029965 | |
| A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, GM1 gangliosidosis, GM2 gangliosidosis, Gangliosidoses, Hemic and Lymphatic Diseases, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Niemann-Pick disease, Niemann-Pick disease type C, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, histiocytosis, inborn errors of metabolism, lymphatic system disorder, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, non-Langerhans cell histiocytosis, sphingolipidosis | Recruiting | Phase 3 | 2026-09-24 | NCT07054515 | |
| A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, GM1 gangliosidosis, GM2 gangliosidosis, Gangliosidoses, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis | Active, not recruiting | Phase 3 | 2026-09-22 | NCT07082543 | |
| A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Gangliosidoses, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis | Recruiting | Phase 1 / Phase 2 | 2026-09-21 | NCT03952637 | |
| UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, GM2 gangliosidosis, Gangliosidoses, Hemic and Lymphatic Diseases, Hereditary Central Nervous System Demyelinating Diseases, Krabbe disease, Leukoencephalopathies, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Mannosidase Deficiency Diseases, Neurobehavioral Manifestations, Neurologic Manifestations, Neuronal Ceroid-Lipofuscinoses, Niemann-Pick disease, Niemann-Pick disease type A, Nutritional and Metabolic Diseases, Pelizaeus-Merzbacher spectrum disorder, Peroxisomal Disorders, Sandhoff disease, Skin and Connective Tissue Diseases, Sulfatidosis, Tay-Sachs disease, X-linked disease, adrenal gland disorder, adrenocortical insufficiency, adrenoleukodystrophy, alpha-mannosidosis, brain disorder, central nervous system disorder, connective tissue disorder, cutaneous mucinosis, demyelinating disease, endocrine system disorder, hereditary disease, histiocytosis, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, juvenile neuronal ceroid lipofuscinosis, lymphatic system disorder, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, metachromatic leukodystrophy, mucopolysaccharidosis, mucopolysaccharidosis type 2, nervous system disorder, neurodegenerative disease, non-Langerhans cell histiocytosis, sphingolipidosis | Recruiting | Phase 1 | 2026-09-03 | NCT02254863 | |
| Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis Clinical Trial | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, GM1 gangliosidosis, Gangliosidoses, Gangliosidosis, Generalized GM1, Type 1, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis | Not yet recruiting | Phase 1 | 2026-08-31 | NCT07479953 | |
| Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol) | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, GM2 gangliosidosis, Gangliosidoses, Hereditary Central Nervous System Demyelinating Diseases, Krabbe disease, Leukoencephalopathies, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, Sandhoff disease, brain disorder, central nervous system disorder, demyelinating disease, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, neuronal ceroid lipofuscinosis 2, sphingolipidosis | Not yet recruiting | Phase 2 | 2026-07-31 | NCT07740512 | |
| ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Ceroid Lipofuscinosis, Neuronal, 2, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Fabry disease, GM1 gangliosidosis, Gangliosidoses, Gaucher disease, Hemic and Lymphatic Diseases, Hereditary Central Nervous System Demyelinating Diseases, Infant, Newborn, Diseases, Leukoencephalopathies, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Mucopolysaccharidosis III, Neurobehavioral Manifestations, Neurologic Manifestations, Niemann-Pick disease, Niemann-Pick disease type C, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Rare Diseases, Skin and Connective Tissue Diseases, Sulfatidosis, Wolman disease, X-linked disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrotendinous xanthomatosis, cerebrovascular disorder, cholesteryl ester storage disease, connective tissue disorder, cutaneous mucinosis, demyelinating disease, hereditary disease, histiocytosis, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, lymphatic system disorder, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, metachromatic leukodystrophy, mucopolysaccharidosis, mucopolysaccharidosis type 2, mucopolysaccharidosis type 4, mucopolysaccharidosis type 6, mucopolysaccharidosis type 7, nervous system disorder, non-Langerhans cell histiocytosis, sphingolipidosis, vascular disorder, xanthomatosis | Enrolling by invitation | Not stated | 2026-07-28 | NCT05368038 | |
| Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, GM1 gangliosidosis, Gangliosidoses, Gangliosidosis, Generalized GM1, Type 1, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Rare Diseases, beta-Galactosidase Deficiency, brain disorder, central nervous system disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis | Active, not recruiting | Phase 1 / Phase 2 | 2026-06-30 | NCT04713475 | |
| A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, GM2 gangliosidosis, Gangliosidoses, Hemic and Lymphatic Diseases, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Niemann-Pick disease, Niemann-Pick disease type C, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, histiocytosis, inborn errors of metabolism, lymphatic system disorder, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, non-Langerhans cell histiocytosis, sphingolipidosis | Recruiting | Phase 2 | 2026-06-18 | NCT07399704 | |
| Translational Potential of ex Vivo Gene Therapy in GM2 Gangliosidosis | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Gangliosidoses, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis | Not yet recruiting | Not stated | 2026-03-06 | NCT07445490 | |
| A Natural History Study of the Gangliosidoses | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, GM1 gangliosidosis, GM2 gangliosidosis, Gangliosidoses, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, Sandhoff disease, Tay-Sachs Disease, Juvenile, Tay-Sachs disease, brain disorder, central nervous system disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis | Recruiting | Not stated | 2026-03-04 | NCT00668187 | |
| Longitudinal Study of Neurodegenerative Disorders | Bone Diseases, Developmental, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Death, Death, Sudden, GM2 gangliosidosis, Gangliosidoses, Gaucher disease, Hemic and Lymphatic Diseases, Hereditary Central Nervous System Demyelinating Diseases, Infant Death, Krabbe disease, Leukoencephalopathies, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Mannosidase Deficiency Diseases, Mucopolysaccharidosis III, Neuronal Ceroid-Lipofuscinoses, Niemann-Pick disease, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Pelizaeus-Merzbacher spectrum disorder, Sandhoff disease, Skin and Connective Tissue Diseases, Sudden Infant Death, Sulfatidosis, Tay-Sachs disease, X-linked disease, adrenoleukodystrophy, alpha-mannosidosis, bone disorder, brain disorder, central nervous system disorder, connective tissue disorder, cutaneous mucinosis, demyelinating disease, disorder of methionine catabolism, giant axonal neuropathy 1, hereditary disease, histiocytosis, hypermethioninemia with deficiency of S-adenosylhomocysteine hydrolase, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, juvenile neuronal ceroid lipofuscinosis, leukodystrophy, leukoencephalopathy with vanishing white matter 1, lymphatic system disorder, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, metachromatic leukodystrophy, mucopolysaccharidosis, mucopolysaccharidosis type 4, mucosulfatidosis, musculoskeletal system disorder, nervous system disorder, neurodegenerative disease, non-Langerhans cell histiocytosis, osteochondrodysplasia, osteopetrosis, osteosclerosis, pantothenate kinase-associated neurodegeneration, purine nucleoside phosphorylase deficiency, sphingolipidosis | Recruiting | Not stated | 2026-02-09 | NCT03333200 | |
| The Myelin Disorders Biorepository Project | Abnormalities, Multiple, Aicardi-Goutieres syndrome, Alexander disease, Alexanders leukodystrophy, Allan-Herndon-Dudley syndrome, Bone Diseases, Developmental, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Canavan disease, Cerebral Small Vessel Diseases, Charcot-Marie-Tooth disease, Cockayne syndrome, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA repair disease, Digestive System Diseases, Dwarfism, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, GM2 gangliosidosis, Gangliosidoses, Hereditary Central Nervous System Demyelinating Diseases, Infant, Newborn, Diseases, Infarction, Krabbe disease, Leukoencephalopathies, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Male Urogenital Diseases, Necrosis, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Pelizaeus-Merzbacher spectrum disorder, Peroxisomal Disorders, Salla disease, Sjogren syndrome, Sjogren-Larsson syndrome, Skin Abnormalities, Skin and Connective Tissue Diseases, Stomatognathic Diseases, Sulfatidosis, Urogenital Diseases, X-linked disease, Xerostomia, Zellweger spectrum disorders, adrenal gland disorder, adrenocortical insufficiency, adrenoleukodystrophy, adrenomyeloneuropathy, adult Refsum disease, adult-onset autosomal dominant demyelinating leukodystrophy, alpha-gal syndrome, arthritic joint disease, arthropathy, attention deficit hyperactivity disorder, inattentive type, autoimmune disease, bone disorder, brain disorder, brain infarction, brain ischemia, cardiovascular disorder, central nervous system disorder, central nervous system malformation, cerebral arterial disease, cerebral infarction, cerebrotendinous xanthomatosis, cerebrovascular disorder, connective tissue disorder, cutaneous mucinosis, dementia, demyelinating disease, dry eye syndrome, endocrine system disorder, eye disorder, hereditary disease, hereditary motor and sensory neuropathy, hereditary skin disorder, hypomyelination with brain stem and spinal cord involvement and leg spasticity, ichthyosis, immune system disorder, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, intracranial arterial disease, ischemic disease, keratosis, kidney disorder, lacrimal apparatus disorder, leukodystrophy, leukodystrophy, hypomyelinating, 5, leukodystrophy, hypomyelinating, 6, leukodystrophy, hypomyelinating, 7, with or without oligodontia and/or hypogonadotropic hypogonadism, leukoencephalopathy with brain stem and spinal cord involvement-high lactate syndrome, leukoencephalopathy with calcifications and cysts, leukoencephalopathy with vanishing white matter 1, leukoencephalopathy, diffuse hereditary, with spheroids 1, liver disorder, lysosomal lipid storage disorder, lysosomal storage disease, megalencephalic leukoencephalopathy with subcortical cysts, metabolic disease, metachromatic leukodystrophy, mouth disorder, mucopolysaccharidosis, mucosulfatidosis, musculoskeletal system disorder, nervous system disorder, neurodegeneration with brain iron accumulation 5, neurodegenerative disease, neurodevelopmental disorder, neuromuscular disease, null syndrome, oppositional defiant disorder, peripheral nervous system disorder, polyneuropathy, psychiatric disorder, rheumatic disorder, rheumatoid arthritis, salivary gland disorder, skin disorder, sphingolipidosis, stroke disorder, urinary system disorder, vascular dementia, vascular disorder, xanthomatosis | Recruiting | Not stated | 2025-10-23 | NCT03047369 | |
| Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, GM2 gangliosidosis, Gangliosidoses, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, Sandhoff disease, Tay-Sachs disease, brain disorder, central nervous system disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis | Active, not recruiting | Not stated | 2025-10-16 | NCT06614569 | |
| First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, GM2 gangliosidosis, Gangliosidoses, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, Sandhoff disease, Tay-Sachs disease, brain disorder, central nervous system disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis | Active, not recruiting | Phase 1 / Phase 2 | 2023-05-09 | NCT04798235 |