154 results · Page 1 of 7

Swipe the table sideways for more columns.

TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Agalsidase Beta Long-Term Treatment Outcome for Fabry Disease Patients With IVS4 Mutation in TaiwanBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fabry disease, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, X-linked disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis, vascular disorderActive, not recruitingNot stated2026-10-08NCT06052800
A Basket Clinical Study to Assess Glycerol Tributyrate in Patients With Mitochondrial Encephalopathy, Lactic Acidosis, Stroke-like Episodes (MELAS) or Leber's Hereditary Optic Neuropathy-Plus (LHON-Plus)Acid-Base Imbalance, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Cognition Disorders, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyskinesias, Ear Diseases, MELAS syndrome, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathological Conditions, Signs and Symptoms, Sensation Disorders, Signs and Symptoms, Tremor, acidosis disorder, anxiety disorder, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, cognitive disorder, dystonic disorder, eye disorder, headache disorder, hearing disorder, hearing loss disorder, hereditary disease, hypnic headache, inborn errors of metabolism, inborn mitochondrial metabolism disorder, inborn mitochondrial myopathy, lactic acidosis, metabolic disease, migraine disorder, mitochondrial encephalomyopathy, muscle tissue disorder, musculoskeletal system disorder, nervous system disorder, neuromuscular disease, otorhinolaryngologic disease, psychiatric disorder, vascular disorder, vision disorder, visual epilepsyNot yet recruitingPhase 1 / Phase 22026-10-05NCT06792500
Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry DiseaseBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fabry disease, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, X-linked disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis, vascular disorderRecruitingPhase 1 / Phase 22026-09-24NCT06270316
Anderson-Fabry Disease Fitness Improvement TrainingBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fabry disease, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, X-linked disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis, vascular disorderRecruitingNot applicable2026-09-21NCT07495410
Dementia Risk Registry Across the Multidimensional Lifespan: A 10-Year StudyCerebral Small Vessel Diseases, Mixed Dementias, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, cognitive disorder, dementia, nervous system disorder, psychiatric disorder, vascular disorderRecruitingNot stated2026-09-17NCT06164262
Optimal Target Low-density Lipoprotein Cholesterol Level for Small Vessel Occlusion StrokeCerebral Small Vessel Diseases, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, ischemic stroke, nervous system disorder, stroke disorder, vascular disorderRecruitingNot applicable2026-09-11NCT06649240
A Study of Patients With Fabry Disease (US Specific)Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fabry disease, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, X-linked disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis, vascular disorderRecruitingNot stated2026-09-09NCT06906367
COMMAND Early Feasibility Study: Implantable BCI to Control a Digital Device for People With ParalysisCerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Infarction, Muscular Disorders, Atrophic, Necrosis, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Stroke, Lacunar, TDP-43 proteinopathy, Thrombotic Stroke, amyotrophic lateral sclerosis, brain disorder, brain infarction, brain ischemia, brain stem infarction, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, injury, ischemic disease, ischemic stroke, metabolic disease, motor neuron disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, nervous system injury, neurodegenerative disease, neuromuscular disease, palsy, proteostasis deficiencies, quadriplegia, spinal cord disorder, spinal cord injury, spinal muscular atrophy, stroke disorder, vascular disorderActive, not recruitingNot applicable2026-09-08NCT05035823
Phase 2 Study Evaluating Rapcabtagene Autoleucel in Participants With Severe Active GPA or MPACerebral Small Vessel Diseases, Skin and Connective Tissue Diseases, anti-neutrophil cytoplasmic antibody-associated vasculitis, autoimmune disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, granulomatosis with polyangiitis, immune system disorder, interstitial lung disease, lung disorder, microscopic polyangiitis, necrotizing vasculitis, nervous system disorder, respiratory system disorder, skin disorder, skin vascular disease, vascular disorder, vasculitisActive, not recruitingPhase 22026-09-04NCT06868290
Florida Cerebrovascular Disease Biorepository and Genomics CenterArterial Occlusive Diseases, Cardiovascular Abnormalities, Cerebral Small Vessel Diseases, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Embolism and Thrombosis, Fistula, Hemorrhage, Hypoxia, Hypoxia, Brain, Infarction, Intracranial Embolism and Thrombosis, Intracranial Hemorrhages, Leukoaraiosis, Leukoencephalopathies, Moyamoya disease, Necrosis, Pathologic Processes, Pathological Conditions, Anatomical, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Signs and Symptoms, Respiratory, Subarachnoid Hemorrhage, Aneurysmal, Thromboembolism, Vascular Fistula, arteriosclerosis disorder, arteriovenous hemangioma/malformation, brain disorder, brain hypoxia - ischemia, brain infarction, brain ischemia, cardiovascular disorder, carotid artery disorder, central nervous system disorder, cerebral arterial disease, cerebral infarction, cerebrovascular disorder, cognitive disorder, congenital arteriovenous fistula, dementia, fibromuscular dysplasia, hereditary disease, intracerebral hemorrhage, intracranial arterial disease, intracranial arteriosclerosis, intracranial thrombosis, ischemic disease, ischemic stroke, nervous system disorder, psychiatric disorder, reversible cerebral vasoconstriction syndrome, stroke disorder, subarachnoid hemorrhage, transient ischemic attack, vascular dementia, vascular disorder, vascular malformationActive, not recruitingNot stated2026-09-03NCT05491980
A Study of Replagal in Children and Adults With Fabry Disease in IndiaBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fabry disease, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, X-linked disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis, vascular disorderRecruitingPhase 42026-09-03NCT05067868
Effect of the Mediterranean Diet in Patients Affected by CADASIL and Cerebral Amyloid Angiopathy.Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Amyloid Angiopathy, Familial, Cerebral Small Vessel Diseases, Cognition Disorders, Cognitive Dysfunction, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Infarction, Necrosis, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, amyloidosis, brain disorder, brain infarction, brain ischemia, cardiovascular disorder, central nervous system disorder, cerebral amyloid angiopathy, cerebral arterial disease, cerebral infarction, cerebrovascular disorder, cognitive disorder, dementia, hereditary amyloidosis, hereditary disease, inborn errors of metabolism, intracranial arterial disease, ischemic disease, metabolic disease, nervous system disorder, proteostasis deficiencies, psychiatric disorder, stroke disorder, vascular dementia, vascular disorderRecruitingNot stated2026-09-01NCT06933212
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA VariantsBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fabry disease, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, X-linked disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis, vascular disorderRecruitingPhase 32026-09-01NCT06904261
Genotype, Clinical Features and Imaging of Neuroradiological Abnormalities in CADASILCerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Infarction, Necrosis, Pathologic Processes, Pathological Conditions, Signs and Symptoms, brain disorder, brain infarction, brain ischemia, cardiovascular disorder, central nervous system disorder, cerebral arterial disease, cerebral infarction, cerebrovascular disorder, dementia, disease, hereditary disease, intracranial arterial disease, ischemic disease, nervous system disorder, stroke disorder, vascular dementia, vascular disorderRecruitingNot stated2026-08-31NCT06938100
A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry DiseaseBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fabry disease, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, X-linked disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis, vascular disorderActive, not recruitingPhase 32026-08-31NCT03737214
Quality of Life in Adults With Untreated Fabry Disease in Sweden (QoLUF)Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Fabry disease, 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, X-linked disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis, vascular disorderEnrolling by invitationNot stated2026-08-27NCT07560956
A Study to Learn How Well Lucerastat Works and How Safe it is in Untreated Adult Male Participants With Fabry DiseaseBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fabry disease, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, X-linked disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis, vascular disorderNot yet recruitingPhase 32026-08-21NCT07778667
Iptacopan in Patients With ANCA Associated VasculitisCerebral Small Vessel Diseases, Skin and Connective Tissue Diseases, anti-neutrophil cytoplasmic antibody-associated vasculitis, autoimmune disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, granulomatosis with polyangiitis, immune system disorder, interstitial lung disease, lung disorder, microscopic polyangiitis, necrotizing vasculitis, nervous system disorder, respiratory system disorder, skin disorder, skin vascular disease, vascular disorder, vasculitisActive, not recruitingPhase 22026-08-20NCT06388941
Long-Term Follow-up of Subjects Who Were Treated With ST-920Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fabry Disease, Cardiac Variant, Fabry disease, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, X-linked disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis, vascular disorderActive, not recruitingNot stated2026-08-20NCT05039866
Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal Disease StudyBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fabry disease, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, X-linked disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis, vascular disorderRecruitingNot stated2026-08-14NCT01581424
Cardiovascular Multimodality Imaging StudyAmyloid Neuropathies, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cardiovascular Abnormalities, Cerebral Small Vessel Diseases, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fabry disease, Hemic and Lymphatic Diseases, Hypersensitivity, Delayed, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, X-linked disease, amyloidosis, aortic valve disorder, aortic valve stenosis, arrhythmogenic right ventricular cardiomyopathy, brain disorder, cardiomyopathy, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, congenital heart disease, familial amyloid neuropathy, heart disorder, heart failure, heart valve disorder, hereditary amyloidosis, hereditary disease, hypertrophic cardiomyopathy, immune system disorder, inborn errors of metabolism, inherited neurodegenerative disorder, lymphatic system disorder, lymphoproliferative syndrome, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, neurodegenerative disease, neuromuscular disease, peripheral nervous system disorder, proteostasis deficiencies, sarcoidosis, sphingolipidosis, subvalvular aortic stenosis, vascular disorderRecruitingNot stated2026-08-10NCT06610019
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry DiseaseBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fabry disease, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, X-linked disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis, vascular disorderRecruitingPhase 2 / Phase 32026-08-10NCT06328608
Global Registry and Natural History Study for Mitochondrial DisordersAbnormalities, Multiple, Barth syndrome, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cardiovascular Abnormalities, Cerebral Small Vessel Diseases, Chronic Disease, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Epilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, Kearns-Sayre syndrome, Leber hereditary optic neuropathy, Leigh syndrome, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, MELAS syndrome, MERRF syndrome, NARP syndrome, Neurologic Manifestations, Nutritional and Metabolic Diseases, Ocular Motility Disorders, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Pearson syndrome, Pyruvate Metabolism, Inborn Errors, Signs and Symptoms, X-linked disease, ataxia neuropathy spectrum, brain disorder, cardiomyopathy, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, coenzyme Q10 deficiency, congenital heart disease, cranial nerve neuropathy, epilepsy, eye disorder, heart disorder, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inborn mitochondrial metabolism disorder, inborn mitochondrial myopathy, inherited retinal dystrophy, maternally-inherited diabetes and deafness, metabolic disease, mitochondrial encephalomyopathy, mitochondrial neurogastrointestinal encephalomyopathy, muscle tissue disorder, musculoskeletal system disorder, myelodysplastic syndrome, nervous system disorder, neuromuscular disease, ophthalmoplegia, palsy, progressive external ophthalmoplegia, progressive myoclonus epilepsy, retinal degeneration, retinal disorder, retinitis pigmentosa, spinocerebellar ataxia with epilepsy, vascular disorder, very long chain acyl-CoA dehydrogenase deficiencyRecruitingNot stated2026-08-07NCT05554835
Lacunar Stroke hyperAcute Clinical Utilization of Novel Approach Regimens: Rt-PA vs. DAPT Randomised Clinical TrialCerebral Small Vessel Diseases, Infarction, Necrosis, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Stroke, Lacunar, Thrombotic Stroke, brain disorder, brain infarction, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, ischemic disease, ischemic stroke, nervous system disorder, stroke disorder, vascular disorderRecruitingPhase 42026-08-04NCT07111559
Retina is a Marker for Cerebrovascular HeathCerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Infarction, Necrosis, Pathologic Processes, Pathological Conditions, Signs and Symptoms, brain disorder, brain infarction, brain ischemia, cardiovascular disorder, central nervous system disorder, cerebral arterial disease, cerebral arteriopathy, autosomal dominant, with subcortical infarcts and leukoencephalopathy, type 1, cerebral infarction, cerebrovascular disorder, dementia, hereditary disease, intracranial arterial disease, ischemic disease, nervous system disorder, stroke disorder, vascular dementia, vascular disorderRecruitingPhase 1 / Phase 22026-08-03NCT04753970