Friedreich ataxia
Friedreich ataxia
Explore linked clinical trials. Standard reference: MONDO:010033934 results · Page 1 of 2
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of ATI-105 in Healthy Adults | Congenital, 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 disorder | Not yet recruiting | Phase 1 | 2026-10-09 | NCT07869654 | |
| A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia | 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 disorder | Recruiting | Phase 3 | 2026-10-09 | NCT07778836 | |
| An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia | 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 disorder | Recruiting | Phase 2 | 2026-10-09 | NCT06447025 | |
| Biomarkers in Friedreich's Ataxia | 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, central nervous system disorder, cerebellar disorder, hereditary disease, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, spinal cord disorder | Recruiting | Not stated | 2026-10-06 | NCT02497534 | |
| A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia | 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 disorder | Active, not recruiting | Phase 3 | 2026-10-02 | NCT06953583 | |
| A Study of SGT-212 Gene Therapy in Friedreich's Ataxia | Congenital, 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 disorder | Recruiting | Phase 1 | 2026-09-29 | NCT07180355 | |
| Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia | Cardiac 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 disorder | Recruiting | Phase 1 | 2026-09-28 | NCT05302271 | |
| Cardiac Output and Fatigue in Friedreich's Ataxia | Congenital, 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 disorder | Recruiting | Not applicable | 2026-09-22 | NCT07444333 | |
| 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 Old | 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 disorder | Active, not recruiting | Phase 1 | 2026-09-03 | NCT06054893 | |
| Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy | Cardiomegaly, 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 stenosis | Recruiting | Phase 2 | 2026-09-02 | NCT07721025 | |
| Clinical Course Of Disease In Participants With FA-CM | Congenital, 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 disorder | Recruiting | Not stated | 2026-09-02 | NCT06865482 | |
| Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study | Congenital, 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 disorder | Recruiting | Not stated | 2026-09-01 | NCT06016946 | |
| 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 disorder | Not yet recruiting | Phase 3 | 2026-08-14 | NCT07681713 | |
| 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 disorder | Recruiting | Not stated | 2026-07-29 | NCT02316314 | |
| Digital Outcome Assessment Using AI Active Gaming and Motion Capture in Friedreich Ataxia | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Friedreich ataxia, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Rare 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 disorder | Not yet recruiting | Not stated | 2026-07-28 | NCT07731971 | |
| Characterisation of the Cognitive Profile of Patients Suffering From Friedreich's Ataxia | 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 disorder | Active, not recruiting | Not stated | 2026-07-21 | NCT05874388 | |
| A Study to Learn How BIIB141 (Omaveloxolone) Affects the Health of Participants With Friedrich's Ataxia Who Took it During Pregnancy and/or During Breastfeeding and About the Health of Their Babies | 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 disorder | Recruiting | Not stated | 2026-07-10 | NCT06628687 | |
| GABA and GSH in FRDA | 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 disorder | Enrolling by invitation | Not stated | 2026-06-09 | NCT07635030 | |
| Efficacy of Omaveloxolone Treatment for Dysphagia in French Patients With Friedreich's Ataxia | 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 disorder | Recruiting | Not stated | 2026-04-29 | NCT07013292 | |
| A Study to Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who Are Prescribed it by Their Own Doctors | 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 disorder | Recruiting | Not stated | 2026-04-28 | NCT06623890 | |
| Friedreich Ataxia Nerve Ultrasund | 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 disorder | Recruiting | Not stated | 2026-04-02 | NCT07508631 | |
| Patient-reported, Health Economic and Psychosocial Outcomes in Friedreich Ataxia | 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 disorder | Recruiting | Not stated | 2026-04-01 | NCT05943002 | |
| Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia | Cardiomyopathies, Secondary, Congenital, 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 disorder | Active, not recruiting | Phase 1 / Phase 2 | 2025-12-23 | NCT05445323 | |
| A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia | 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 disorder | Active, not recruiting | Phase 3 | 2025-10-30 | NCT05515536 | |
| Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Chronic Disease, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Friedreich ataxia, Guillain-Barre syndrome, Lambert-Eaton myasthenic syndrome, Lysosomal Storage Diseases, Nervous System, Muscular Disorders, Atrophic, Neoplasms by Site, Nutritional and Metabolic Diseases, Paraneoplastic Syndromes, Nervous System, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Post-Infectious Disorders, Spinocerebellar Degenerations, TDP-43 proteinopathy, amyotrophic lateral sclerosis, autoimmune disease, autoimmune disorder of the nervous system, brain disorder, central nervous system disorder, central nervous system malformation, cerebellar disorder, chronic inflammatory demyelinating polyradiculoneuropathy, demyelinating disease, disorder of glycogen metabolism, facioscapulohumeral muscular dystrophy, glycogen storage disease II, hereditary disease, hereditary motor and sensory neuropathy, immune system disorder, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inborn mitochondrial metabolism disorder, inclusion body myositis, inherited neurodegenerative disorder, lysosomal storage disease, metabolic disease, motor neuron disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, myasthenia gravis, myositis disease, myotonic dystrophy, myotonic syndrome, neoplasm, nervous system disorder, neurodegenerative disease, neuromuscular disease, neuromuscular junction disease, paraneoplastic syndrome, peripheral nervous system disorder, polyneuropathy, polyradiculoneuropathy, proteostasis deficiencies, spinal cord disorder, spinal muscular atrophy | Enrolling by invitation | Not stated | 2025-09-10 | NCT06605612 |