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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
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
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
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
A Safety and Pharmacokinetics Trial of VO659 in SCA1, SCA3 and HDCognition Disorders, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyskinesias, Huntington disease, Machado-Joseph disease, Neurologic Manifestations, Spinocerebellar Ataxias, Spinocerebellar Degenerations, atactic disorder, non-human animal, basal ganglia disorder, brain disorder, central nervous system disorder, cerebellar ataxia, cerebellar disorder, choreatic disease, cognitive disorder, dementia, hereditary disease, inherited neurodegenerative disorder, movement disorder, nervous system disorder, neurodegenerative disease, psychiatric disorder, spinal cord disorder, spinocerebellar ataxia type 1RecruitingPhase 1 / Phase 22026-08-19NCT05822908
Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)Congenital, 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 disorderNot yet recruitingPhase 32026-08-14NCT07681713
Natural History Study of and Genetic Modifiers in Spinocerebellar AtaxiasCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyskinesias, Machado-Joseph disease, Neurologic Manifestations, Spinocerebellar Ataxias, Spinocerebellar Degenerations, Spinocerebellar ataxia 8, 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 disorder, spinocerebellar ataxia 7, spinocerebellar ataxia type 1, spinocerebellar ataxia type 10, spinocerebellar ataxia type 2, spinocerebellar ataxia type 6, spinocerebellar ataxia type 8RecruitingNot stated2026-08-03NCT01060371
Characterization of the Cardiac Phenotype of Friedreich's Ataxia (FRDA)Congenital, 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, 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 stated2026-07-29NCT02316314