Journal
NEURODEGENERATIVE DISEASE MANAGEMENT
Volume 8, Issue 4, Pages 233-242Publisher
FUTURE MEDICINE LTD
DOI: 10.2217/nmt-2018-0013
Keywords
antioxidant; ataxia; clinical trial; EPI-743; FARS-Neuro; Friedreich's ataxia
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Funding
- FARA (Friedreich's Ataxia Research Alliance)
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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).
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