Double-blind, Randomized, Controlled Trial of EPI-743 in Friedreich’s Ataxia. Neurodegnerative Disease Management
Document Type
Article
Publication Date
7-27-2018
Publication Title
Neurodegnerative Disease Management
DOI
10.2217/nmt-2018-0013
ISSN
1758-2032
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).
Recommended Citation
Zesiewicz, Theresa A., Jason Salemi, Susan Perlman, Kelly L. Sullivan, Jessica D. Shaw, Yangxin Huang, Charles Isaacs, Clifton Gooch, David Lynch, Matthew B. Klein.
2018.
"Double-blind, Randomized, Controlled Trial of EPI-743 in Friedreich’s Ataxia. Neurodegnerative Disease Management."
Neurodegnerative Disease Management, 8 (4): Future Medicine.
doi: 10.2217/nmt-2018-0013
https://digitalcommons.georgiasouthern.edu/bee-facpubs/196
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