Theresa Zesiewicz1, Jason L Salemi2, Susan Perlman3, Kelly L Sullivan4, Jessica D Shaw1, Yangxin Huang5, Charles Isaacs6, Clifton Gooch1, David R Lynch7, Matthew B Klein6. 1. Department of Neurology, University of South Florida, Tampa, FL, 33612 USA. 2. Department of Family & Community Medicine, Baylor College of Medicine, Houston, TX, 77098 USA. 3. Department of Neurology, University of California, Los Angeles, CA, 90095 USA. 4. Department of Epidemiology, Georgia Southern University, GA, 30458 USA. 5. Department of Epidemiology & Biostatistics, University of South Florida, Tampa, FL, 33612 USA. 6. Bioelectron Technology Corporation, Mountain View, CA, 94043 USA. 7. Children's Hospital of Pennsylvania, University of Pennsylvania, PA, 19104 USA.
Abstract
AIM: To evaluate the safety and clinical effects of EPI-743 in Friedreich's ataxia patients. EPI-743 is a compound that targets oxidoreductase enzymes essential for redox control of metabolism. METHODS: We conducted a multicenter trial that evaluated EPI-743 during a 6-month placebo-controlled phase, followed by an 18-month open-label phase. End points included low-contrast visual acuity and the Friedreich's Ataxia Rating Scale. RESULTS/ CONCLUSION:EPI-743 was demonstrated to be safe and well tolerated. There were no significant improvements in key end points during the placebo phase. However, at 24 months, EPI-743 treatment was associated with a statistically significant improvement in neurological function and disease progression relative to a natural history cohort (p < 0.001).
RCT Entities:
AIM: To evaluate the safety and clinical effects of EPI-743 in Friedreich's ataxiapatients. EPI-743 is a compound that targets oxidoreductase enzymes essential for redox control of metabolism. METHODS: We conducted a multicenter trial that evaluated EPI-743 during a 6-month placebo-controlled phase, followed by an 18-month open-label phase. End points included low-contrast visual acuity and the Friedreich's Ataxia Rating Scale. RESULTS/ CONCLUSION:EPI-743 was demonstrated to be safe and well tolerated. There were no significant improvements in key end points during the placebo phase. However, at 24 months, EPI-743 treatment was associated with a statistically significant improvement in neurological function and disease progression relative to a natural history cohort (p < 0.001).
Authors: Marie Beaudin; Mario Manto; Jeremy D Schmahmann; Massimo Pandolfo; Nicolas Dupre Journal: Nat Rev Neurol Date: 2022-03-24 Impact factor: 42.937