leukoencephalopathy, diffuse hereditary, with spheroids 1
leukoencephalopathy, diffuse hereditary, with spheroids 1
Explore linked clinical trials. Standard reference: MONDO:08000275 results · Page 1 of 1
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| A Study to Assess CSF1R-related Leukoencephalopathy After Stem Cell Transplantation | Leukoencephalopathies, brain disorder, central nervous system disorder, leukoencephalopathy, diffuse hereditary, with spheroids 1, nervous system disorder | Enrolling by invitation | Not stated | 2026-01-08 | NCT04503213 | |
| MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis | Abnormalities, Multiple, Adrenoleukodystrophy, Neonatal, Bone Diseases, Developmental, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hereditary Central Nervous System Demyelinating Diseases, Hurler syndrome, Krabbe disease, Leukoencephalopathies, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Male Urogenital Diseases, Mannosidase Deficiency Diseases, Mucopolysaccharidosis I, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Peroxisomal Disorders, Refsum Disease, Infantile, Skin and Connective Tissue Diseases, Sulfatidosis, Urogenital Diseases, X-linked disease, Zellweger spectrum disorders, alpha-mannosidosis, alpha-methylacyl-CoA racemase deficiency, aspartylglucosaminuria, bone disorder, brain disorder, central nervous system disorder, connective tissue disorder, cutaneous mucinosis, d-bifunctional protein deficiency, demyelinating disease, fucosidosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, kidney disorder, leukoencephalopathy, diffuse hereditary, with spheroids 1, liver disorder, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, metachromatic leukodystrophy, mitochondrial neurogastrointestinal encephalomyopathy, mucopolysaccharidosis, mucopolysaccharidosis type 2, mucopolysaccharidosis type 6, mucopolysaccharidosis type 7, musculoskeletal system disorder, nervous system disorder, osteochondrodysplasia, osteopetrosis, osteosclerosis, peroxisomal acyl-CoA oxidase deficiency, sphingolipidosis, urinary system disorder | Active, not recruiting | Phase 2 | 2026-01-07 | NCT02171104 | |
| Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT | Bone Diseases, Developmental, Bone Marrow Failure Disorders, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Chediak-Higashi syndrome, Chronic Disease, Congenital Abnormalities, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Crohn disease, Cytopenia, DNA repair disease, Death, Death, Sudden, Diamond-Blackfan anemia, Digestive System Diseases, Disease Attributes, Eye Diseases, Hereditary, Gaucher disease, Hemic and Lymphatic Diseases, Hereditary Central Nervous System Demyelinating Diseases, Infant Death, Infant, Newborn, Diseases, Krabbe disease, Leukoencephalopathies, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lymphohistiocytosis, Hemophagocytic, Lysosomal Storage Diseases, Nervous System, Mannosidase Deficiency Diseases, Mucopolysaccharidosis I, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Peroxisomal Disorders, Primary Immunodeficiency Diseases, Skin Abnormalities, Skin and Connective Tissue Diseases, Sudden Infant Death, Sulfatidosis, Wiskott-Aldrich syndrome, X-linked disease, adrenal gland disorder, adrenocortical insufficiency, adrenoleukodystrophy, albinism, alpha-mannosidosis, anemia, aplastic anemia, arthritic joint disease, arthropathy, autoimmune disease, autoimmune lymphoproliferative syndrome, beta thalassemia, blood coagulation disease, bone disorder, bone marrow disorder, brain disorder, central nervous system disorder, chronic granulomatous disease, connective tissue disorder, cutaneous mucinosis, demyelinating disease, digestive system disorder, disease, dyskeratosis congenita, endocrine system disorder, eye disorder, familial hemolytic anemia, gastroenteritis, hematologic disorder, hemolytic anemia, hemorrhagic disease, hereditary disease, hereditary skin disorder, histiocytosis, immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome, immune system disorder, immunoproliferative disorder, inborn carbohydrate metabolic disorder, inborn error of immunity, inborn errors of metabolism, inflammatory bowel disease, inherited aplastic anemia, inherited blood coagulation disorder, inherited hemoglobinopathy, inherited neurodegenerative disorder, intellectual disability, intestinal disorder, juvenile idiopathic arthritis, leukocyte adhesion deficiency 1, leukocyte disorder, leukoencephalopathy, diffuse hereditary, with spheroids 1, leukopenia, lymphatic system disorder, lymphopenia, lymphoproliferative syndrome, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, metachromatic leukodystrophy, mucopolysaccharidosis, mucopolysaccharidosis type 2, musculoskeletal system disorder, nervous system disorder, non-Langerhans cell histiocytosis, osteochondrodysplasia, osteopetrosis, osteosclerosis, phagocyte bactericidal dysfunction, pure red-cell aplasia, rheumatic disorder, severe combined immunodeficiency, sickle cell disease, skin disorder, sphingolipidosis, syndromic disease, thalassemia | Recruiting | Phase 2 | 2025-12-15 | NCT01962415 | |
| 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 | |
| Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hereditary Central Nervous System Demyelinating Diseases, Inflammation, Leukoencephalopathies, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Neurobehavioral Manifestations, Neuroinflammatory Diseases, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Peroxisomal Disorders, Sulfatidosis, X-linked disease, adrenal gland disorder, adrenocortical insufficiency, adrenoleukodystrophy, adrenomyeloneuropathy, brain disorder, central nervous system disorder, demyelinating disease, endocrine system disorder, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, leukoencephalopathy, diffuse hereditary, with spheroids 1, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, metachromatic leukodystrophy, nervous system disorder, sphingolipidosis | Recruiting | Not stated | 2025-09-24 | NCT04925349 |