Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia (NCT01728064) | Clinical Trial Compass
CompletedPhase 2
Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia
United States60 participantsStarted 2012-12-31
Plain-language summary
The purpose of this study is to examine the effects of EPI-743 on visual function and neurologic function in patients with Friedreich's ataxia.
Who can participate
Age range
18 Years – 45 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Diagnosis of genetically confirmed Friedreich's ataxia
. Visual acuity at baseline more than 15 letters on EDTRS at four meters
. FARS score of 20 to 90
. Agreement to use contraception if within reproductive years (see specifics in section D1, page 21)
. Hormone replacement therapy, if used, must remain stable for the duration of the study
. Willingness and ability to comply with study procedures
. Willingness and ability to arrive at study site day prior to evaluations
. Abstention from use of dietary supplements and non-prescribed medications at least 30 days prior to initiation of treatment and for the duration of the study. This would specifically include idebenone, Coenzyme Q10 and vitamin E
Exclusion criteria
. Allergy to EPI-743 or sesame oil or nuts
. Clinically significant bleeding condition or abnormal PT/PTT INR (INR \> two; PTT \> two-times normal)
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.