adrenoleukodystrophy
adrenoleukodystrophy
Explore linked clinical trials. Standard reference: MONDO:001854418 results · Page 1 of 1
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| Home Exercise for Individuals With Neurodegenerative Disease | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyskinesias, Hereditary Central Nervous System Demyelinating Diseases, Leukoencephalopathies, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathological Conditions, Signs and Symptoms, Peroxisomal Disorders, Signs and Symptoms, X-linked disease, adrenal gland disorder, adrenocortical insufficiency, adrenoleukodystrophy, adrenomyeloneuropathy, atactic disorder, non-human animal, brain disorder, central nervous system disorder, cerebellar ataxia, cerebellar disorder, demyelinating disease, endocrine system disorder, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, leukodystrophy, leukoencephalopathy with brain stem and spinal cord involvement-high lactate syndrome, metabolic disease, nervous system disorder, neurodegenerative disease | Recruiting | Not applicable | 2026-09-16 | NCT05443906 | |
| Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hereditary Central Nervous System Demyelinating Diseases, Leukoencephalopathies, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Peroxisomal Disorders, X-linked disease, adrenal gland disorder, adrenocortical insufficiency, adrenoleukodystrophy, brain disorder, central nervous system disorder, demyelinating disease, endocrine system disorder, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, metabolic disease, nervous system disorder | Active, not recruiting | Not stated | 2026-09-14 | NCT02698579 | |
| 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 | |
| IT and IV Lentiviral Gene Therapy for X-ALD | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hereditary Central Nervous System Demyelinating Diseases, Leukoencephalopathies, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Peroxisomal Disorders, X-linked disease, adrenal gland disorder, adrenocortical insufficiency, adrenoleukodystrophy, brain disorder, central nervous system disorder, demyelinating disease, endocrine system disorder, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, metabolic disease, nervous system disorder | Not yet recruiting | Not applicable | 2026-09-01 | NCT03727555 | |
| Quality of Life in Women With X-linked Adrenoleukodystrophy | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hereditary Central Nervous System Demyelinating Diseases, Leukoencephalopathies, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Peroxisomal Disorders, X-linked disease, adrenal gland disorder, adrenocortical insufficiency, adrenoleukodystrophy, brain disorder, central nervous system disorder, demyelinating disease, endocrine system disorder, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, metabolic disease, nervous system disorder | Recruiting | Not stated | 2026-08-27 | NCT04675749 | |
| Treating Leg Symptoms in Women With X-linked Adrenoleukodystrophy | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyssomnias, Hereditary Central Nervous System Demyelinating Diseases, Leukoencephalopathies, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Parasomnias, Peroxisomal Disorders, Sleep Disorders, Intrinsic, X-linked disease, adrenal gland disorder, adrenocortical insufficiency, adrenoleukodystrophy, brain disorder, central nervous system disorder, demyelinating disease, endocrine system disorder, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, metabolic disease, nervous system disorder, psychiatric disorder, restless legs syndrome, sleep-wake disorder | Active, not recruiting | Phase 4 | 2026-07-01 | NCT05003648 | |
| Adrenoleukodystrophy National Registry Study | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hereditary Central Nervous System Demyelinating Diseases, Leukoencephalopathies, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Peroxisomal Disorders, X-linked disease, adrenal gland disorder, adrenocortical insufficiency, adrenoleukodystrophy, brain disorder, central nervous system disorder, demyelinating disease, endocrine system disorder, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, metabolic disease, nervous system disorder | Recruiting | Not stated | 2026-05-12 | NCT03789721 | |
| 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 | |
| Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC | Blood Protein Disorders, Bone Diseases, Developmental, Bone Marrow Failure Disorders, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Chediak-Higashi syndrome, Chronic Disease, 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 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, dysgammaglobulinemia, endocrine system disorder, eye disorder, familial hemolytic anemia, gastroenteritis, hematologic disorder, hemolytic anemia, hemorrhagic disease, hereditary disease, histiocytosis, hyper-IgM syndrome, 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, leukocyte adhesion deficiency 1, leukocyte disorder, leukopenia, lymphatic system disorder, lymphopenia, lymphoproliferative syndrome, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, metachromatic leukodystrophy, mucopolysaccharidosis, musculoskeletal system disorder, nervous system disorder, non-Langerhans cell histiocytosis, osteochondrodysplasia, osteopetrosis, osteosclerosis, phagocyte bactericidal dysfunction, pure red-cell aplasia, rheumatic disorder, rheumatoid arthritis, severe combined immunodeficiency, sickle cell disease, sphingolipidosis, syndromic disease, thalassemia | Recruiting | Not stated | 2026-01-13 | NCT04528355 | |
| 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 | |
| A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hereditary Central Nervous System Demyelinating Diseases, Leukoencephalopathies, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Peroxisomal Disorders, X-linked disease, adrenal gland disorder, adrenocortical insufficiency, adrenoleukodystrophy, brain disorder, central nervous system disorder, demyelinating disease, endocrine system disorder, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, metabolic disease, nervous system disorder | Recruiting | Not stated | 2025-07-02 | NCT06224413 | |
| Epidural Spinal Cord Stimulation for Lower-limb Impairment in Adrenomyeloneuropathy | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Hereditary Central Nervous System Demyelinating Diseases, Leukoencephalopathies, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Peroxisomal Disorders, Rare Diseases, X-linked disease, adrenal gland disorder, adrenocortical insufficiency, adrenoleukodystrophy, brain disorder, central nervous system disorder, demyelinating disease, endocrine system disorder, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, metabolic disease, nervous system disorder | Recruiting | Not applicable | 2025-01-28 | NCT06796920 | |
| Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases. | 2-hydroxyglutaric aciduria, Alexander disease, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cysts, Digestive System Diseases, Disease Attributes, Hereditary Central Nervous System Demyelinating Diseases, Krabbe disease, Leukoencephalopathies, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Anatomical, Pathological Conditions, Signs and Symptoms, Pelizaeus-Merzbacher spectrum disorder, Peroxisomal Disorders, Rare Diseases, Sulfatidosis, Undiagnosed Diseases, Wilson disease, X-linked disease, adrenal gland disorder, adrenocortical insufficiency, adrenoleukodystrophy, basal ganglia disorder, bilateral striopallidodentate calcinosis, brain disorder, central nervous system disorder, cerebrotendinous xanthomatosis, demyelinating disease, endocrine system disorder, hereditary disease, inborn errors of metabolism, inborn metal metabolism disorder, inherited neurodegenerative disorder, intellectual disability, liver disorder, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, metachromatic leukodystrophy, methylmalonic aciduria and homocystinuria, movement disorder, neoplasm, nervous system disorder, neurodegenerative disease, polycystic lipomembranous osteodysplasia with sclerosing leukoencephaly, sphingolipidosis, xanthomatosis | Recruiting | Not stated | 2024-11-19 | NCT04880356 | |
| Disease Progression in Women With X-linked Adrenoleukodystrophy | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hereditary Central Nervous System Demyelinating Diseases, Leukoencephalopathies, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Peroxisomal Disorders, X-linked disease, adrenal gland disorder, adrenocortical insufficiency, adrenoleukodystrophy, brain disorder, central nervous system disorder, demyelinating disease, endocrine system disorder, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, metabolic disease, nervous system disorder | Recruiting | Not stated | 2024-01-31 | NCT06178120 | |
| Registry of X-linked Adrenoleukodystrophy | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hereditary Central Nervous System Demyelinating Diseases, Leukoencephalopathies, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Peroxisomal Disorders, X-linked disease, adrenal gland disorder, adrenocortical insufficiency, adrenoleukodystrophy, brain disorder, central nervous system disorder, demyelinating disease, endocrine system disorder, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, metabolic disease, nervous system disorder | Recruiting | Not stated | 2023-07-11 | NCT05939232 | |
| Validation of a Prognostic Biomarker Using Brain Diffusion MRI in X-linked Adrenoleukodystrophy | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hereditary Central Nervous System Demyelinating Diseases, Leukoencephalopathies, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Peroxisomal Disorders, X-linked disease, adrenal gland disorder, adrenocortical insufficiency, adrenoleukodystrophy, brain disorder, central nervous system disorder, demyelinating disease, endocrine system disorder, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, metabolic disease, nervous system disorder | Not yet recruiting | Not stated | 2023-07-03 | NCT05911919 |