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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of ATI-105 in Healthy AdultsCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Friedreich ataxia, Mitochondrial Dysfunction, Nutritional and Metabolic Diseases, Spinocerebellar Degenerations, brain disorder, cardiomyopathy, cardiovascular disorder, central nervous system disorder, cerebellar disorder, heart disorder, hereditary disease, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorderNot yet recruitingPhase 12026-10-09NCT07869654
A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects 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 disorderRecruitingPhase 32026-10-09NCT07778836
An Open-Label Study of CTI-1601 in Subjects 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 disorderRecruitingPhase 22026-10-09NCT06447025
At Home Use of Stimulation Suits for Managing MS SymptomsAtaxia, Motor, CNS demyelinating autoimmune disease, Dyskinesias, Muscle Hypertonia, Muscle Spasticity, Neurologic Manifestations, Neuromuscular Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, atactic disorder, non-human animal, autoimmune disease, autoimmune disorder of the nervous system, brain disorder, central nervous system disorder, cerebellar ataxia, cerebellar disorder, demyelinating disease, immune system disorder, multiple sclerosis, muscle tissue disorder, musculoskeletal system disorder, nervous system disorderRecruitingNot applicable2026-10-08NCT07202195
Biomarkers in Friedreich's AtaxiaCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyskinesias, Friedreich ataxia, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathological Conditions, Signs and Symptoms, 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 stated2026-10-06NCT02497534
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
Phenotype/Genotype Correlations in Movement DisordersCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Spinocerebellar Degenerations, brain disorder, central nervous system disorder, cerebellar disorder, essential tremor, hereditary disease, inherited neurodegenerative disorder, movement disorder, nervous system disorder, neurodegenerative disease, spinal cord disorderRecruitingNot stated2026-10-02NCT00018889
Multi-Modal Digital Monitoring of Disease Symptoms in Spinocerebellar AtaxiaCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyskinesias, Neurologic Manifestations, Spinocerebellar Ataxias, Spinocerebellar Degenerations, atactic disorder, non-human animal, brain disorder, central nervous system disorder, cerebellar ataxia, cerebellar disorder, hereditary disease, inherited neurodegenerative disorder, nervous system disorder, neurodegenerative disease, spinal cord disorderNot yet recruitingNot stated2026-10-01NCT07851376
A Study of SGT-212 Gene Therapy in Friedreich's AtaxiaCongenital, 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, movement disorder, nervous system disorder, neurodegenerative disease, spinal cord disorderRecruitingPhase 12026-09-29NCT07180355
Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's AtaxiaCardiac Hypertrophy, Cardiomegaly, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyskinesias, Friedreich ataxia, Hypertrophy, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathological Conditions, Anatomical, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Spinocerebellar Degenerations, atactic disorder, non-human animal, brain disorder, cardiomyopathy, cardiovascular disorder, central nervous system disorder, cerebellar disorder, heart disorder, hereditary disease, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, spinal cord disorderRecruitingPhase 12026-09-28NCT05302271
Rare Disease Registry Database for Movement Disorders in Southern Anhui ProvinceCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyskinesias, Neurologic Manifestations, Spinocerebellar Ataxias, Spinocerebellar Degenerations, atactic disorder, non-human animal, brain disorder, central nervous system disorder, central nervous system malformation, cerebellar ataxia, cerebellar disorder, hereditary disease, hereditary motor and sensory neuropathy, hereditary spastic paraplegia, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, peripheral nervous system disorder, polyneuropathy, spinal cord disorderActive, not recruitingNot stated2026-09-24NCT07838883
Cardiac Output and Fatigue in Friedreich's AtaxiaCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Fatigue, Friedreich ataxia, Nutritional and Metabolic Diseases, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, 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 disorderRecruitingNot applicable2026-09-22NCT07444333
Home Exercise for Individuals With Neurodegenerative DiseaseBrain 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 diseaseRecruitingNot applicable2026-09-16NCT05443906
The Pancreas Interception Center (PIC) for Early Detection, Prevention, and Novel TherapeuticsChronic Disease, Colorectal Neoplasms, Hereditary Nonpolyposis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cronkhite-Canada syndrome, Cysts, DNA repair disease, Digestive System Diseases, Digestive System Neoplasms, Disease Attributes, Dyskinesias, Familial Atypical Multiple Mole-Melanoma, Hyperpigmentation, Lynch syndrome, Melanosis, Neoplasms by Histologic Type, Neoplasms by Site, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pancreatic Cyst, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Peutz-Jeghers syndrome, Primary Immunodeficiency Diseases, Skin and Connective Tissue Diseases, Spinocerebellar Ataxias, atactic disorder, non-human animal, ataxia telangiectasia, brain disorder, cardiovascular disorder, central nervous system disorder, cerebellar ataxia, cerebellar disorder, chronic pancreatitis, classic familial adenomatous polyposis, colonic disorder, colorectal neoplasm, digestive system disorder, digestive system neoplasm, hereditary disease, hereditary neoplastic syndrome, immune system disorder, inborn error of immunity, intestinal disorder, lentigo, melanocytic neoplasm, melanocytic nevus, melanoma, cutaneous malignant, susceptibility to, 2, metabolic disease, neoplasm, nervous system disorder, neurocutaneous syndrome, pancreas disorder, pancreatic ductal adenocarcinoma, pancreatitis, skin disorder, skin pigmentation disorder, telangiectasis, vascular disorderActive, not recruitingNot stated2026-09-15NCT05692596
Natural History of Spinocerebellar Ataxia Type 7 (SCA7)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyskinesias, Neurologic Manifestations, Spinocerebellar Ataxias, Spinocerebellar Degenerations, atactic disorder, non-human animal, brain disorder, central nervous system disorder, cerebellar ataxia, cerebellar disorder, hereditary disease, inherited neurodegenerative disorder, nervous system disorder, neurodegenerative disease, spinal cord disorderActive, not recruitingNot stated2026-09-15NCT02741440
Neoadjuvant Pembrolizumab and Porustobart in Resectable pMMR/MSS Colon CancerCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Digestive System Neoplasms, Neoplasms by Site, Spinocerebellar Degenerations, brain disorder, central nervous system disorder, cerebellar disorder, colonic disorder, colonic neoplasm, colorectal neoplasm, digestive system disorder, digestive system neoplasm, hereditary disease, inherited neurodegenerative disorder, intestinal disorder, malignant colon neoplasm, neoplasm, nervous system disorder, neurodegenerative disease, spinal cord disorder, type 1 diabetes mellitus 12Not yet recruitingPhase 22026-09-08NCT07808151
Pivotal Study of N-acetyl-L-leucine for CACNA1ACongenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyskinesias, Neurologic Manifestations, Spinocerebellar Ataxias, Spinocerebellar Degenerations, atactic disorder, non-human animal, brain disorder, central nervous system disorder, cerebellar ataxia, cerebellar disorder, episodic ataxia type 2, hereditary disease, inherited neurodegenerative disorder, nervous system disorder, neurodegenerative disease, spinal cord disorder, spinocerebellar ataxia type 6RecruitingPhase 32026-09-08NCT07221292
Confirmatory Study 3 of KPS-0373 in Patients With Spinocerebellar DegenerationCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Spinocerebellar Degenerations, brain disorder, central nervous system disorder, cerebellar disorder, hereditary disease, inherited neurodegenerative disorder, nervous system disorder, neurodegenerative disease, spinal cord disorderActive, not recruitingPhase 32026-09-04NCT07040137
HSV G207 in Children With Recurrent or Refractory Cerebellar Brain TumorsCentral Nervous System Neoplasms, Primary, Cerebellar Neoplasms, Primary, Infections, Neoplasm Metastasis, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neoplastic Processes, Neuroectodermal Tumors, Primitive, Pathologic Processes, Pathological Conditions, Signs and Symptoms, astrocytoma (excluding glioblastoma), brain cancer, brain disorder, cancer of cerebellum, central nervous system cancer, central nervous system disorder, cerebellar astrocytoma, cerebellar disorder, cerebellar neoplasm, choroid plexus carcinoma, ependymoma, epithelial neoplasm, glioblastoma, glioma, gliosarcoma, medulloblastoma, mixed neoplasm, neoplasm, nervous system cancer, nervous system disorder, neuroepithelial neoplasm, oligodendroglioma, primitive neuroectodermal tumor, rhabdoid tumor, viral infectious diseaseRecruitingPhase 12026-09-04NCT03911388
A Study to Find Out How BIIB141 (Omaveloxolone) is Processed in the Body and to Learn More About Its Safety in Participants With Friedreich's Ataxia Aged 2 to 15 Years OldCongenital, 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 12026-09-03NCT06054893
Study of LX2006 Gene Therapy in Friedreich Ataxia CardiomyopathyCardiomegaly, Cardiomyopathies, Secondary, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Friedreich ataxia, Hypertrophy, Hypertrophy, Left Ventricular, Nutritional and Metabolic Diseases, Pathological Conditions, Anatomical, Pathological Conditions, Signs and Symptoms, Spinocerebellar Degenerations, aortic valve disorder, aortic valve stenosis, brain disorder, cardiomyopathy, cardiovascular disorder, central nervous system disorder, cerebellar disorder, heart disorder, heart valve disorder, hereditary disease, hypertrophic cardiomyopathy, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, spinal cord disorder, subvalvular aortic stenosisRecruitingPhase 22026-09-02NCT07721025
Clinical Course Of Disease In Participants With FA-CMCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Friedreich ataxia, Nutritional and Metabolic Diseases, Spinocerebellar Degenerations, brain disorder, cardiomyopathy, cardiovascular disorder, central nervous system disorder, cerebellar disorder, heart disorder, hereditary disease, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, spinal cord disorderRecruitingNot stated2026-09-02NCT06865482
TreatHSP Platform: Adaptive Natural History Platform for Ataxias, HSPs, and Spastic AtaxiasCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Disease Progression, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Spinocerebellar Degenerations, brain disorder, central nervous system disorder, central nervous system malformation, cerebellar disorder, hereditary disease, hereditary motor and sensory neuropathy, hereditary spastic paraplegia, inherited neurodegenerative disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, peripheral nervous system disorder, polyneuropathy, spastic ataxia, spinal cord disorderRecruitingNot stated2026-09-01NCT07798674
Optimizing Parameters of Transcranial Temporal Interference Stimulation for Spinocerebellar Ataxia Type 3Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyskinesias, Machado-Joseph disease, Neurologic Manifestations, Spinocerebellar Ataxias, Spinocerebellar Degenerations, atactic disorder, non-human animal, brain disorder, central nervous system disorder, cerebellar ataxia, cerebellar disorder, hereditary disease, inherited neurodegenerative disorder, nervous system disorder, neurodegenerative disease, spinal cord disorderNot yet recruitingNot applicable2026-09-01NCT07743866
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