Double-blind, randomized and controlled trial of EPI-743 in Friedreich's ataxia

Theresa Zesiewicz; Jason L. Salemi; Susan Perlman; Kelly L. Sullivan; Jessica D. Shaw; Yangxin Huang; Charles Isaacs; Clifton Gooch; David R. Lynch; Matthew B. Klein

doi:10.2217/nmt-2018-0013

Double-blind, randomized and controlled trial of EPI-743 in Friedreich's ataxia

Theresa Zesiewicz, Jason L. Salemi, Susan Perlman, Kelly L. Sullivan, Jessica D. Shaw, Yangxin Huang, Charles Isaacs, Clifton Gooch, David R. Lynch, Matthew B. Klein

Biostatistics, Epidemiology, & Environmental Health Sciences

Research output: Contribution to journal › Article › peer-review

65 Scopus citations

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).

Original language	English
Pages (from-to)	233-242
Number of pages	10
Journal	Neurodegenerative Disease Management
Volume	8
Issue number	4
DOIs	https://doi.org/10.2217/nmt-2018-0013
State	Published - Aug 1 2018

Keywords

EPI-743
FARS-Neuro
Friedreich's ataxia
antioxidant
ataxia
clinical trial

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10.2217/nmt-2018-0013

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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).",

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AU - Zesiewicz, Theresa

AU - Salemi, Jason L.

AU - Perlman, Susan

AU - Sullivan, Kelly L.

AU - Shaw, Jessica D.

AU - Huang, Yangxin

AU - Isaacs, Charles

AU - Gooch, Clifton

AU - Lynch, David R.

AU - Klein, Matthew B.

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AB - 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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KW - FARS-Neuro

KW - Friedreich's ataxia

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JO - Neurodegenerative Disease Management

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ER -

Double-blind, randomized and controlled trial of EPI-743 in Friedreich's ataxia

Abstract

Keywords

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