juvenile neuronal ceroid lipofuscinosis
juvenile neuronal ceroid lipofuscinosis
Explore linked clinical trials. Standard reference: MONDO:001926212 results · Page 1 of 1
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| Investigations of Juvenile Neuronal Ceroid Lipofuscinosis | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Neuronal Ceroid-Lipofuscinoses, Nutritional and Metabolic Diseases, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, juvenile neuronal ceroid lipofuscinosis, lysosomal lipid storage disorder, metabolic disease, nervous system disorder, neurodegenerative disease | Recruiting | Not stated | 2026-10-05 | NCT03307304 | |
| 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 | |
| Gene Therapy for Children With CLN3 Batten Disease | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Neuronal Ceroid-Lipofuscinoses, Nutritional and Metabolic Diseases, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, juvenile neuronal ceroid lipofuscinosis, lysosomal lipid storage disorder, metabolic disease, nervous system disorder, neurodegenerative disease | Active, not recruiting | Phase 1 / Phase 2 | 2026-06-10 | NCT03770572 | |
| Inherited Retinal Degenerative Disease Registry | Abnormalities, Multiple, Bardet-Biedl syndrome, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Charcot-Marie-Tooth disease, Chronic Disease, Ciliopathies, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Deaf-Blind Disorders, Deafness, Disease Attributes, Dyslipidemias, Ear Diseases, Eye Diseases, Hereditary, Goldmann-Favre syndrome, Hearing Loss, Sensorineural, Juvenile Macular Degeneration, Kearns-Sayre syndrome, Leber congenital amaurosis, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Neurologic Manifestations, Neuronal Ceroid-Lipofuscinoses, Nutritional and Metabolic Diseases, Ocular Motility Disorders, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Peroxisomal Disorders, Rod-Cone Dystrophy, Sensation Disorders, Signs and Symptoms, Stargardt disease, Usher syndrome, X-linked disease, abetalipoproteinemia, achromatopsia, achromatopsia 3, adult Refsum disease, blindness (disorder), brain disorder, cardiomyopathy, cardiovascular disorder, central areolar choroidal dystrophy, central nervous system disorder, central nervous system malformation, choroideremia, color vision disorder, cone dystrophy, cone-rod dystrophy, congenital stationary night blindness, cranial nerve neuropathy, enhanced S-cone syndrome, eye disorder, fundus albipunctatus, hearing disorder, hearing loss disorder, heart disorder, hereditary disease, hereditary motor and sensory neuropathy, hypobetalipoproteinemia, hypolipoproteinemia, hypothalamic disorder, inborn errors of metabolism, inborn mitochondrial metabolism disorder, inborn mitochondrial myopathy, inherited neurodegenerative disorder, inherited retinal dystrophy, juvenile neuronal ceroid lipofuscinosis, lysosomal lipid storage disorder, macular degeneration, metabolic disease, muscle tissue disorder, musculoskeletal system disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, ophthalmoplegia, optic choroid disorder, ornithine aminotransferase deficiency, otorhinolaryngologic disease, palsy, peripheral nervous system disorder, polyneuropathy, progressive external ophthalmoplegia, retinal degeneration, retinal disorder, retinitis pigmentosa, retinitis punctata albescens, retinoschisis, uveal disorder, vision disorder, vitelliform macular dystrophy | Recruiting | Not stated | 2026-05-19 | NCT02435940 | |
| Gene Therapy Trial for CLN6 Batten Disease | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Neuronal Ceroid-Lipofuscinoses, Nutritional and Metabolic Diseases, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, juvenile neuronal ceroid lipofuscinosis, lysosomal lipid storage disorder, metabolic disease, nervous system disorder, neurodegenerative disease | Not yet recruiting | Phase 1 / Phase 2 | 2026-05-14 | NCT07582484 | |
| 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 | |
| Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Neuronal Ceroid-Lipofuscinoses, Nutritional and Metabolic Diseases, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, juvenile neuronal ceroid lipofuscinosis, lysosomal lipid storage disorder, metabolic disease, nervous system disorder, neurodegenerative disease, neuronal ceroid lipofuscinosis 2 | Enrolling by invitation | Not stated | 2025-10-24 | NCT03862274 | |
| Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Neuronal Ceroid-Lipofuscinoses, Nutritional and Metabolic Diseases, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, juvenile neuronal ceroid lipofuscinosis, lysosomal lipid storage disorder, metabolic disease, nervous system disorder, neurodegenerative disease | Active, not recruiting | Not stated | 2025-10-02 | NCT04273243 | |
| Clinical and Neuropsychological Investigations in Batten Disease | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Neuronal Ceroid-Lipofuscinoses, Nutritional and Metabolic Diseases, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, juvenile neuronal ceroid lipofuscinosis, lysosomal lipid storage disorder, metabolic disease, nervous system disorder, neurodegenerative disease | Recruiting | Not stated | 2025-09-12 | NCT01873924 | |
| Natural History of Neuronal Ceroid Lipofuscinosis, Batten's CLN6 Diseae | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Neuronal Ceroid-Lipofuscinoses, Nutritional and Metabolic Diseases, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, juvenile neuronal ceroid lipofuscinosis, lysosomal lipid storage disorder, metabolic disease, nervous system disorder, neurodegenerative disease | Active, not recruiting | Not stated | 2025-07-30 | NCT03285425 | |
| NYSCF Scientific Discovery Biobank | Adnexal Diseases, Alzheimer Disease, Early Onset, Alzheimer Disease, Late Onset, Alzheimer disease, CNS demyelinating autoimmune disease, Cognition Disorders, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyskinesias, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Frontotemporal Lobar Degeneration, Genital Diseases, Huntington disease, Lewy body dementia, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Neoplasms by Site, Neurologic Manifestations, Neuronal Ceroid-Lipofuscinoses, Nutritional and Metabolic Diseases, Ocular Motility Disorders, Parkinson disease, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Stress Disorders, Traumatic, TDP-43 proteinopathy, Urogenital Diseases, Urogenital Neoplasms, Uterine Cervical Neoplasms, acute stress disorder, amyotrophic lateral sclerosis, autoimmune disease, autoimmune disorder of the nervous system, autonomic nervous system disorder, basal ganglia disorder, brain disorder, central nervous system disorder, cervix disorder, choreatic disease, cognitive disorder, corticobasal degeneration disorder, cranial nerve neuropathy, dementia, demyelinating disease, diabetes mellitus, endocrine system disorder, eye disorder, female reproductive system disorder, female reproductive system neoplasm, frontotemporal dementia, glucose metabolism disease, gonadal disorder, hereditary disease, immune system disorder, inborn errors of metabolism, inherited neurodegenerative disorder, juvenile neuronal ceroid lipofuscinosis, lysosomal lipid storage disorder, macular degeneration, malignant endocrine neoplasm, metabolic disease, motor neuron disorder, movement disorder, multiple sclerosis, multiple system atrophy, neoplasm, nervous system disorder, neurodegeneration with brain iron accumulation 2A, neurodegenerative disease, neuromuscular disease, ophthalmoplegia, ovarian cancer, ovarian disorder, palsy, parkinsonian disorder, post-traumatic stress disorder, primary dysautonomia, progressive supranuclear palsy, proteostasis deficiencies, psychiatric disorder, retinal degeneration, retinal disorder, spinal cord disorder, synucleinopathy, tauopathy, type 1 diabetes mellitus, type 2 diabetes mellitus, uterine cancer, uterine disorder, vaginal cancer, vaginal disorder, vulva cancer, vulvar disease | Recruiting | Not stated | 2025-03-03 | NCT06203106 | |
| Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Database | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Kufor-Rakeb syndrome, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Neuronal Ceroid-Lipofuscinoses, Nutritional and Metabolic Diseases, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, juvenile neuronal ceroid lipofuscinosis, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, neurodegenerative disease, neuronal ceroid lipofuscinosis 1, neuronal ceroid lipofuscinosis 5, parkinsonism due to ATP13A2 deficiency, progressive myoclonic epilepsy type 3 | Recruiting | Not stated | 2021-10-29 | NCT04613089 |