{"disease":{"id":"friedreich-ataxia","name":"friedreich ataxia"},"drugs":{"marketed":[{"drug_id":"omaveloxolone","indication_name":"Friedreich’s ataxia","indication_type":"approved","phase":null,"line_of_therapy":null,"is_primary_indication":false,"pivotal_trial_result":null,"pivotal_trial_p_value":null,"patient_population":null,"brand_name":"Skyclarys","generic_name":"OMAVELOXOLONE","company_name":"Biogen Us","drug_phase":"marketed","molecular_target":"Nuclear factor erythroid 2-related factor 2","drug_class":"","quality_score":55,"revenue":"300","mechanism":"Skyclarys works by activating the Nrf2 pathway to increase the production of antioxidant proteins that protect cells from damage."}],"pipeline":[],"offLabel":[],"totalMarketed":1,"totalPipeline":0},"trials":{"data":[{"nct_id":"NCT06016946","title":"Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study","phase":"","overall_status":"RECRUITING","enrollment_count":3000,"lead_sponsor_name":"Friedreich's Ataxia Research Alliance","has_results":false},{"nct_id":"NCT03090789","title":"FA Clinical Outcome Measures","phase":"","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":2000,"lead_sponsor_name":"Friedreich's Ataxia Research Alliance","has_results":false},{"nct_id":"NCT06623890","title":"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","phase":"","overall_status":"RECRUITING","enrollment_count":300,"lead_sponsor_name":"Biogen","has_results":false},{"nct_id":"NCT06953583","title":"A Study to Learn More About the Effects and Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Aged 2 to 15 Years Old (BRAVE)","phase":"PHASE3","overall_status":"RECRUITING","enrollment_count":255,"lead_sponsor_name":"Biogen","has_results":false},{"nct_id":"NCT05943002","title":"Patient-reported, Health Economic and Psychosocial Outcomes in Friedreich Ataxia","phase":"","overall_status":"RECRUITING","enrollment_count":200,"lead_sponsor_name":"German Center for Neurodegenerative Diseases (DZNE)","has_results":false},{"nct_id":"NCT02255435","title":"A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":172,"lead_sponsor_name":"Biogen","has_results":true},{"nct_id":"NCT04577352","title":"A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia","phase":"PHASE2, PHASE3","overall_status":"COMPLETED","enrollment_count":146,"lead_sponsor_name":"PTC Therapeutics","has_results":true},{"nct_id":"NCT05515536","title":"A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia","phase":"PHASE3","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":130,"lead_sponsor_name":"PTC Therapeutics","has_results":false},{"nct_id":"NCT04268147","title":"Instrumented Data Exchange for Ataxia Study","phase":"","overall_status":"COMPLETED","enrollment_count":128,"lead_sponsor_name":"University of Chicago","has_results":false},{"nct_id":"NCT04346238","title":"Characterization of the Interruptions of the GAA Expansion and Study of Their Influence on the Severity of Friedreich's Ataxia","phase":"","overall_status":"UNKNOWN","enrollment_count":120,"lead_sponsor_name":"University Hospital, Montpellier","has_results":false},{"nct_id":"NCT03418740","title":"Neurology Measures in FA Children","phase":"","overall_status":"COMPLETED","enrollment_count":108,"lead_sponsor_name":"Children's Hospital of Philadelphia","has_results":true},{"nct_id":"NCT06605612","title":"Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders","phase":"","overall_status":"ENROLLING_BY_INVITATION","enrollment_count":108,"lead_sponsor_name":"LMU Klinikum","has_results":false},{"nct_id":"NCT02593773","title":"Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia Study","phase":"PHASE3","overall_status":"COMPLETED","enrollment_count":86,"lead_sponsor_name":"Amgen","has_results":true},{"nct_id":"NCT04192136","title":"NAD+ and Exercise in FA","phase":"NA","overall_status":"COMPLETED","enrollment_count":75,"lead_sponsor_name":"Children's Hospital of Philadelphia","has_results":true},{"nct_id":"NCT03214588","title":"Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich Ataxia","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":67,"lead_sponsor_name":"Neurocrine Biosciences","has_results":true},{"nct_id":"NCT04102501","title":"A Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Friedreich's Ataxia","phase":"PHASE3","overall_status":"COMPLETED","enrollment_count":65,"lead_sponsor_name":"Biojiva LLC","has_results":true},{"nct_id":"NCT05028764","title":"Determine Range of Tissue Frataxin Concentrations and Other Potential Biomarkers","phase":"","overall_status":"COMPLETED","enrollment_count":60,"lead_sponsor_name":"Larimar Therapeutics, Inc.","has_results":false},{"nct_id":"NCT01728064","title":"Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":60,"lead_sponsor_name":"PTC Therapeutics","has_results":false},{"nct_id":"NCT04564495","title":"Home Based Tele-exercise for People With Chronic Neurological Impairments","phase":"NA","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":60,"lead_sponsor_name":"Burke Medical Research Institute","has_results":false},{"nct_id":"NCT00229632","title":"Idebenone to Treat Friedreich's Ataxia","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":51,"lead_sponsor_name":"National Institute of Neurological Disorders and Stroke (NINDS)","has_results":false},{"nct_id":"NCT01898884","title":"Safety and Pharmacology Study of VP 20629 in Adults With Friedreich's Ataxia","phase":"PHASE1","overall_status":"COMPLETED","enrollment_count":46,"lead_sponsor_name":"Shire","has_results":true},{"nct_id":"NCT07013292","title":"Efficacy of Omaveloxolone Treatment for Dysphagia in French Patients With Friedreich's Ataxia","phase":"","overall_status":"NOT_YET_RECRUITING","enrollment_count":40,"lead_sponsor_name":"Centre Hospitalier Universitaire de Nice","has_results":false},{"nct_id":"NCT03917225","title":"A Clinical Study to Evaluate the Effect of MIN-102 on the Progression of Friedreich's Ataxia in Male and Female Patients","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":39,"lead_sponsor_name":"Minoryx Therapeutics, S.L.","has_results":false},{"nct_id":"NCT05285540","title":"Study to Evaluate DT-216 in Adult Patients With Friedreich Ataxia","phase":"PHASE1","overall_status":"COMPLETED","enrollment_count":39,"lead_sponsor_name":"Design Therapeutics, Inc.","has_results":false},{"nct_id":"NCT02797080","title":"Long-Term Safety Extension Study of ACTIMMUNE® (Interferon γ-1b) in Children and Young Adults With Friedreich's Ataxia","phase":"PHASE3","overall_status":"COMPLETED","enrollment_count":38,"lead_sponsor_name":"Amgen","has_results":true},{"nct_id":"NCT06054893","title":"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","phase":"PHASE1","overall_status":"RECRUITING","enrollment_count":35,"lead_sponsor_name":"Biogen","has_results":false},{"nct_id":"NCT05573698","title":"Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia","phase":"PHASE1","overall_status":"COMPLETED","enrollment_count":32,"lead_sponsor_name":"Design Therapeutics, Inc.","has_results":false},{"nct_id":"NCT07095062","title":"Electroencephalogram in Patients With Friedreich's Ataxia for the Study of the Structural and Functional Connectome.","phase":"NA","overall_status":"RECRUITING","enrollment_count":30,"lead_sponsor_name":"IRCCS Eugenio Medea","has_results":false},{"nct_id":"NCT07072676","title":"The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.","phase":"NA","overall_status":"ENROLLING_BY_INVITATION","enrollment_count":30,"lead_sponsor_name":"LMU Klinikum","has_results":false},{"nct_id":"NCT07444333","title":"Cardiac Output and Fatigue in Friedreich's Ataxia","phase":"NA","overall_status":"NOT_YET_RECRUITING","enrollment_count":30,"lead_sponsor_name":"Scott Barbuto","has_results":false},{"nct_id":"NCT04273165","title":"Safety and Efficacy of Etravirine in Friedreich Ataxia Patients","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":30,"lead_sponsor_name":"IRCCS Eugenio Medea","has_results":false},{"nct_id":"NCT05579691","title":"A Double-Blind, Placebo-Controlled, Dose Exploration Study of CTI-1601 in Adult Subjects With Friedreich's Ataxia","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":28,"lead_sponsor_name":"Larimar Therapeutics, Inc.","has_results":false},{"nct_id":"NCT04176991","title":"Single Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia","phase":"PHASE1","overall_status":"COMPLETED","enrollment_count":28,"lead_sponsor_name":"Larimar Therapeutics, Inc.","has_results":false},{"nct_id":"NCT04519567","title":"Multiple Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia","phase":"PHASE1","overall_status":"COMPLETED","enrollment_count":27,"lead_sponsor_name":"Larimar Therapeutics, Inc.","has_results":false},{"nct_id":"NCT05302271","title":"Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia","phase":"PHASE1","overall_status":"RECRUITING","enrollment_count":25,"lead_sponsor_name":"Weill Medical College of Cornell University","has_results":false},{"nct_id":"NCT03933163","title":"Micronised Resveratrol as a Treatment for Friedreich Ataxia","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":25,"lead_sponsor_name":"Murdoch Childrens Research Institute","has_results":false},{"nct_id":"NCT07123142","title":"FRIEDREICH ATAXIA- STEROIDOGENESIS","phase":"","overall_status":"COMPLETED","enrollment_count":25,"lead_sponsor_name":"Istanbul University","has_results":false},{"nct_id":"NCT03120013","title":"Rehabilitative Trial With Cerebello-Spinal tDCS in Neurodegenerative Ataxia","phase":"NA","overall_status":"COMPLETED","enrollment_count":21,"lead_sponsor_name":"Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia","has_results":true},{"nct_id":"NCT04921930","title":"Evaluation of the Effect of Artesunate in Friedreich Ataxia (FA)","phase":"PHASE1, PHASE2","overall_status":"COMPLETED","enrollment_count":20,"lead_sponsor_name":"Institut National de la Santé Et de la Recherche Médicale, France","has_results":false},{"nct_id":"NCT06628687","title":"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","phase":"","overall_status":"RECRUITING","enrollment_count":20,"lead_sponsor_name":"Biogen","has_results":false},{"nct_id":"NCT05168774","title":"FRDA Investigator Initiated Study (IIS) With Elamipretide","phase":"PHASE1, PHASE2","overall_status":"COMPLETED","enrollment_count":20,"lead_sponsor_name":"Children's Hospital of Philadelphia","has_results":true},{"nct_id":"NCT04801303","title":"Evaluation of the Effects of Calcitriol's in the Neurological Symptoms of Friedreich's Ataxia Patients","phase":"PHASE4","overall_status":"COMPLETED","enrollment_count":20,"lead_sponsor_name":"Berta Alemany","has_results":false},{"nct_id":"NCT03122925","title":"TCA Cycle in the Dentate in Friedreich's Ataxia","phase":"","overall_status":"COMPLETED","enrollment_count":20,"lead_sponsor_name":"University of Minnesota","has_results":false},{"nct_id":"NCT04255680","title":"A Study to Assess Variation in Potential Biomarkers in Friedreich Ataxia","phase":"","overall_status":"COMPLETED","enrollment_count":20,"lead_sponsor_name":"Larimar Therapeutics, Inc.","has_results":false},{"nct_id":"NCT06681766","title":"A Study to Assess Nomlabofusp in Adolescents and Children With Friedreich's Ataxia","phase":"PHASE1","overall_status":"TERMINATED","enrollment_count":18,"lead_sponsor_name":"Larimar Therapeutics, Inc.","has_results":false},{"nct_id":"NCT07508631","title":"Friedreich Ataxia Nerve Ultrasund","phase":"","overall_status":"RECRUITING","enrollment_count":14,"lead_sponsor_name":"Centre Hospitalier Universitaire de Nice","has_results":false},{"nct_id":"NCT03888664","title":"Open Trail of γIFN for Friedreich Ataxia","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":12,"lead_sponsor_name":"IRCCS Eugenio Medea","has_results":false},{"nct_id":"NCT07180355","title":"A Study of SGT-212 Gene Therapy in Friedreich's Ataxia","phase":"PHASE1","overall_status":"RECRUITING","enrollment_count":10,"lead_sponsor_name":"Solid Biosciences Inc.","has_results":false},{"nct_id":"NCT06496451","title":"Measurement of Frataxin mRNA in Biofluids","phase":"","overall_status":"COMPLETED","enrollment_count":10,"lead_sponsor_name":"University of Florida","has_results":false},{"nct_id":"NCT04649866","title":"Coronary Artery Disease in Patients With Friedreich's Ataxia","phase":"NA","overall_status":"COMPLETED","enrollment_count":7,"lead_sponsor_name":"University of Pittsburgh","has_results":false}],"total":50},"guidelines":[],"source":"Drug Landscape verified database"}