Clinical Trial: EPI-743 in Friedreich's Ataxia Point Mutations
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: A Phase 2A Clinical Trial of EPI-743 (Vincerinone™) on Visual Function in Friedreich's Ataxia Patients With Point Mutations
Brief Summary: The purpose of this study is to evaluate the effects of EPI-743 in patients with Friedreich's Ataxia point mutations
Detailed Summary: The primary objective of this study is to investigate whether treatment with EPI-743 has a discernible impact on visual function—including visual acuity, visual fields and color vision as well as on any of a number of functional and subject/clinician-rated scales relevant in the treatment of Friedreich's ataxia, and to determine the safety of treatment with EPI-743.
Sponsor: University of South Florida
Current Primary Outcome:
- Visual function [ Time Frame: 3 months ]Low contrast acuity
- Safety parameters [ Time Frame: 3 months ]Clinical and laboratory safety parameters
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Visual function [ Time Frame: 3 months ]Visual fields as assessed by Humphrey 30-2 exam; High contrast visual acuity
- Neurologic function [ Time Frame: 3 months ]Friedreich's Ataxia Rating Scale
- Physical function [ Time Frame: 3 months ]25-foot walk (for subjects capable of completing the test on enrollment);
- Health related quality of life [ Time Frame: 3 months ]Patient report via rating scale
- Activities of Daily Living [ Time Frame: 3 months ]Patient report via rating scale
- Cardiac indices [ Time Frame: 3 months ]Echocardiogram
- Upper extremity function [ Time Frame: 3 months ]9 hole peg test
- Disease biomarkers [ Time Frame: 3 months ]Glutathione cycle components
Original Secondary Outcome: Same as current
Information By: University of South Florida
Dates:
Date Received: October 10, 2013
Date Started: October 2013
Date Completion:
Last Updated: June 28, 2016
Last Verified: June 2016
