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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
An Efficacy and Safety Study of Clemizole HCl in Patients With Lennox-Gastaut SyndromeCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Epileptic Syndromes, Lennox-Gastaut syndrome, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, brain disorder, central nervous system disorder, epilepsy, hereditary disease, nervous system disorder, visual epilepsyRecruitingPhase 32026-10-09NCT05066217
A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's AtaxiaCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Friedreich ataxia, Nutritional and Metabolic Diseases, Spinocerebellar Degenerations, brain disorder, central nervous system disorder, cerebellar disorder, hereditary disease, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, spinal cord disorderActive, not recruitingPhase 32026-10-02NCT06953583
REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman SyndromeAbnormalities, Multiple, Angelman syndrome, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Imprinting Disorders, central nervous system disorder, chromosomal disorder, hereditary disease, movement disorder, nervous system disorderActive, not recruitingPhase 32026-09-29NCT06914609
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 (NPC)Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, 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, sphingolipidosisRecruitingPhase 32026-09-28NCT07082725
A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4Congenital, Hereditary, and Neonatal Diseases and Abnormalities, congenital myasthenic syndrome, hereditary disease, nervous system disorder, neuromuscular disease, neuromuscular junction diseaseRecruitingNot stated2026-09-25NCT06078553
ADAPT Forward - Master Protocol of a Platform Study to Evaluate the Safety and Efficacy of Multiple Regimens in Participants With Myasthenia GravisNeoplasms by Site, Paraneoplastic Syndromes, Nervous System, autoimmune disease, autoimmune disorder of the nervous system, generalized myasthenia gravis, immune system disorder, myasthenia gravis, neoplasm, nervous system disorder, neurodegenerative disease, neuromuscular disease, neuromuscular junction disease, paraneoplastic syndromeRecruitingNot stated2026-09-24NCT07294170
ADAPT Forward 1 - ISA1 - a Study to Evaluate Empasiprubart IV as add-on Therapy to Efgartigimod IV in Participants With AChR-Ab Seropositive Generalized Myasthenia Gravis With a Partial Clinical Response to EfgartigimodNeoplasms by Site, Paraneoplastic Syndromes, Nervous System, autoimmune disease, autoimmune disorder of the nervous system, generalized myasthenia gravis, immune system disorder, myasthenia gravis, neoplasm, nervous system disorder, neurodegenerative disease, neuromuscular disease, neuromuscular junction disease, paraneoplastic syndromeRecruitingPhase 22026-09-24NCT07284420
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 GangliosidosisBrain 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, sphingolipidosisRecruitingPhase 32026-09-24NCT07054515
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 GangliosidosisBrain 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, sphingolipidosisActive, not recruitingPhase 32026-09-22NCT07082543
Efficacy and Safety of Erenumab in Pediatric Participants With Episodic MigraineHeadache, Neurologic Manifestations, Pain, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, brain disorder, central nervous system disorder, headache disorder, hypnic headache, migraine disorder, nervous system disorderActive, not recruitingPhase 32026-09-21NCT03836040
Open Label Extension to Assess Long Term Safety and Efficacy of KL1333 in Patients With Primary Mitochondrial DiseaseNutritional and Metabolic Diseases, inborn mitochondrial metabolism disorder, metabolic diseaseRecruitingPhase 22026-09-16NCT07514338
Efficacy and Safety of a New Formulation of Oral Cladribine Compared With Placebo in Participants With Generalized Myasthenia Gravis (MyClad)Neoplasms by Site, Paraneoplastic Syndromes, Nervous System, autoimmune disease, autoimmune disorder of the nervous system, immune system disorder, myasthenia gravis, neoplasm, nervous system disorder, neurodegenerative disease, neuromuscular disease, neuromuscular junction disease, paraneoplastic syndromeRecruitingPhase 32026-09-16NCT06463587
Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History StudyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Dyskinesias, Friedreich ataxia, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Rare Diseases, Signs and Symptoms, Spinocerebellar Degenerations, atactic disorder, non-human animal, brain disorder, central nervous system disorder, cerebellar disorder, hereditary disease, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, spinal cord disorderRecruitingNot stated2026-09-01NCT06016946
EMBRACE Tremor BiFUScentral nervous system disorder, essential tremor, movement disorder, nervous system disorderRecruitingNot applicable2026-08-31NCT06974916
Disease Biosignatures in ALS/FTD Spectrum: New Impactful Biological Perspectives Beyond Clinical ApproachesFrontotemporal Lobar Degeneration, Nutritional and Metabolic Diseases, TDP-43 proteinopathy, amyotrophic lateral sclerosis, brain disorder, central nervous system disorder, cognitive disorder, dementia, frontotemporal dementia, intracranial hypertension, metabolic disease, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, proteostasis deficiencies, pseudotumor cerebri, psychiatric disorder, spinal cord disorderActive, not recruitingNot stated2026-08-28NCT06856850
Exploring the Olfactory Mucosa, Blood and Urine for the Identification of Early Biomarkers of Parkinson's Disease, Atypical Parkinsonisms and Neurocognitive Disorders Due to Lewy Body DiseaseLewy body dementia, Parkinson disease, Pathologic Processes, Pathological Conditions, Signs and Symptoms, autonomic nervous system disorder, basal ganglia disorder, brain disorder, cardiovascular disorder, central nervous system disorder, cognitive disorder, dementia, disease, familial cylindromatosis, hypotensive disorder, movement disorder, multiple system atrophy, nervous system disorder, neurodegenerative disease, parkinsonian disorder, primary dysautonomia, psychiatric disorder, synucleinopathy, vascular disorderActive, not recruitingNot stated2026-08-28NCT06846658
EF-41/KEYNOTE D58: Phase 3 Study of Optune Concomitant With Temozolomide Plus Pembrolizumab in Newly Diagnosed GlioblastomaNeoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, astrocytoma (excluding glioblastoma), epithelial neoplasm, glioblastoma, glioma, neoplasm, neuroepithelial neoplasm, primitive neuroectodermal tumorRecruitingPhase 32026-08-19NCT06556563
A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, congenital myasthenic syndrome, hereditary disease, nervous system disorder, neuromuscular disease, neuromuscular junction diseaseActive, not recruitingPhase 12026-07-24NCT06436742
A Trial of Amlenetug (Lu AF82422) in Participants With Multiple System Atrophy (MSA)autonomic nervous system disorder, basal ganglia disorder, brain disorder, central nervous system disorder, movement disorder, multiple system atrophy, nervous system disorder, neurodegenerative disease, primary dysautonomia, synucleinopathyActive, not recruitingPhase 32026-07-14NCT06706622
A Study to Assess Efficacy and Safety of Efgartigimod PH20 SC in Adults With Ocular Myasthenia GravisMyasthenia Gravis, Ocular, Neoplasms by Site, Paraneoplastic Syndromes, Nervous System, autoimmune disease, autoimmune disorder of the nervous system, immune system disorder, myasthenia gravis, neoplasm, nervous system disorder, neurodegenerative disease, neuromuscular disease, neuromuscular junction disease, paraneoplastic syndromeActive, not recruitingPhase 32026-07-02NCT06558279
A Study to Learn About the Safety of Diranersen (BIIB080) Injections and Whether They Can Improve Symptoms of Participants With Mild Cognitive Impairment Due to Alzheimer's Disease (AD) or Mild AD Dementia Between 50 to 80 Years of AgeAlzheimer's Disease Dementia; Mild Cognitive Impairment Due to Alzheimer's DiseaseActive, not recruitingPhase 22026-06-30NCT05399888
KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial DiseasesBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, MELAS syndrome, Nutritional and Metabolic Diseases, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, inborn errors of metabolism, inborn mitochondrial metabolism disorder, inborn mitochondrial myopathy, maternally-inherited diabetes and deafness, metabolic disease, mitochondrial encephalomyopathy, muscle tissue disorder, musculoskeletal system disorder, nervous system disorder, neuromuscular disease, vascular disorderRecruitingPhase 32026-06-29NCT06451757
Efficacy of KL1333 in Adult Patients With Primary Mitochondrial DiseaseMuscle Weakness, Neurologic Manifestations, Neuromuscular Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, diabetes mellitus, endocrine system disorder, glucose metabolism disease, metabolic disease, muscle tissue disorder, musculoskeletal system disorder, nervous system disorder, neuromuscular diseaseRecruitingPhase 22026-06-26NCT05650229
A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND)central nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyActive, not recruitingPhase 32026-05-22NCT05067790
Neurosurgical Outcome NetworkAneurysm, Arterial Occlusive Diseases, Cardiovascular Abnormalities, Chiari malformation type I, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Craniocerebral Trauma, Craniofacial Pain, Drug Resistant Epilepsy, Dyskinesias, Facial Pain, Hematoma, Hematoma, Epidural, Spinal, Hematoma, Subdural, Hemorrhage, Intracranial Hemorrhage, Traumatic, Intracranial Hemorrhages, Moyamoya disease, Muscle Hypertonia, Muscle Spasticity, Neurologic Manifestations, Neuromuscular Manifestations, Pain, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Stomatognathic Diseases, Tremor, anxiety disorder, arteriovenous hemangioma/malformation, brain disorder, cardiovascular disorder, carotid artery disorder, central nervous system disorder, cerebral arterial disease, cerebrovascular disorder, cranial nerve neuropathy, depressive disorder, dystonic disorder, epilepsy, facial nerve disorder, facial neuralgia, hydrocephalus, injury, intracranial arterial disease, mouth disorder, movement disorder, muscle tissue disorder, musculoskeletal system disorder, nervous system disorder, nervous system injury, neuralgia, neuromuscular disease, normal pressure hydrocephalus, obsessive-compulsive disorder, peripheral nervous system disorder, psychiatric disorder, subarachnoid hemorrhage, trigeminal nerve disorder, trigeminal neuralgia, vascular disorder, vascular malformationRecruitingNot stated2026-03-30NCT06724029