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

79 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

Scopus Subject Areas

Clinical Neurology

Keywords

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

Access to Document

10.2217/nmt-2018-0013

Cite this

@article{ca22ef1d4a02441c9840e7b5f58d97cb,

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

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).",

keywords = "EPI-743, FARS-Neuro, Friedreich's ataxia, antioxidant, ataxia, clinical trial",

author = "Theresa Zesiewicz and Salemi, \{Jason L.\} and Susan Perlman and Sullivan, \{Kelly L.\} and Shaw, \{Jessica D.\} and Yangxin Huang and Charles Isaacs and Clifton Gooch and Lynch, \{David R.\} and Klein, \{Matthew B.\}",

year = "2018",

month = aug,

day = "1",

doi = "10.2217/nmt-2018-0013",

language = "English",

volume = "8",

pages = "233--242",

journal = "Neurodegenerative Disease Management",

issn = "1758-2024",

publisher = "Taylor and Francis Ltd.",

number = "4",

}

TY - JOUR

T1 - Double-blind, randomized and controlled trial of EPI-743 in Friedreich's ataxia

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.

PY - 2018/8/1

Y1 - 2018/8/1

N2 - 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).

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).

KW - EPI-743

KW - FARS-Neuro

KW - Friedreich's ataxia

KW - antioxidant

KW - ataxia

KW - clinical trial

UR - https://www.scopus.com/pages/publications/85058753523

U2 - 10.2217/nmt-2018-0013

DO - 10.2217/nmt-2018-0013

M3 - Article

SN - 1758-2024

VL - 8

SP - 233

EP - 242

JO - Neurodegenerative Disease Management

JF - Neurodegenerative Disease Management

IS - 4

ER -

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

Abstract

Scopus Subject Areas

Keywords

Access to Document

Other files and links

Fingerprint

Cite this