Format

Send to

Choose Destination
Neurodegener Dis Manag. 2018 Aug;8(4):233-242. doi: 10.2217/nmt-2018-0013. Epub 2018 Jul 27.

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

Author information

1
Department of Neurology, University of South Florida, Tampa, FL, 33612 USA.
2
Department of Family & Community Medicine, Baylor College of Medicine, Houston, TX, 77098 USA.
3
Department of Neurology, University of California, Los Angeles, CA, 90095 USA.
4
Department of Epidemiology, Georgia Southern University, GA, 30458 USA.
5
Department of Epidemiology & Biostatistics, University of South Florida, Tampa, FL, 33612 USA.
6
Bioelectron Technology Corporation, Mountain View, CA, 94043 USA.
7
Children's Hospital of Pennsylvania, University of Pennsylvania, PA, 19104 USA.

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

PMID:
30051753
DOI:
10.2217/nmt-2018-0013
[Indexed for MEDLINE]

Supplemental Content

Full text links

Icon for Atypon
Loading ...
Support Center