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Double-blind, randomized and controlled trial of EPI-743 in Friedreich's ataxia
Theresa Zesiewicz1, Jason L Salemi2, Susan Perlman3
1Department of Neurology, University of South Florida, Tampa, FL, 33612 USA.
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).