Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia

Study Overview
=================
Brief 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.

Official Title
-----------------
Safety and Efficacy Study of EPI-743 on Visual Function in Patients With Friedreich's Ataxia

Conditions
-----------------
Friedreich's Ataxia

Intervention / Treatment
-----------------
* Drug: Placebo
* Drug: EPI-743 400 mg
* Drug: EPI-743 200 mg


Participation Criteria
=================
Eligibility Criteria
-----------------
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 Abstention from use of other investigative or non-approved drugs within 30 days of enrollment and for the duration of the study 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) Liver insufficiency with LFTs greater than three-times upper normal limit at screening Renal insufficiency with creatinine > 1.5 at screening Fat malabsorption syndromes Any other respiratory chain diseases of the mitochondria or inborn errors of metabolism Any other ophthalmologic conditions History of alcohol or drug abuse Clinically significant cardiomyopathy with ejection fraction < 40 percent at screening Clinically significant arrhythmia within past two years requiring treatment Anticoagulant therapy within 30 days of enrollment.

Ages Eligible for Study
-----------------
Minimum Age: 18 Years
Maximum Age: 45 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Placebo Comparator: Placebo<br>Placebo capsules three times daily | Drug: Placebo<br>* Placebo capsules<br>|
| Active Comparator: EPI-743 400 mg<br>EPI-743 at a dose of 400 mg three times daily | Drug: EPI-743 400 mg<br> <br> |
| Active Comparator: EPI-743 200 mg<br>EPI-743 at a dose of 200 mg three times daily | Drug: EPI-743 200 mg<br> <br> |

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Visual Function | Low contrast visual acuity |  | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Color vision | Roth 28 hue test |  | 
| Neurologic function | Friedreich's ataxia rating scale |  | 
| Neuromuscular function | 25-foot walk test |  | 
| Neuromuscular function | 9-hole peg test |  | 
| Quality of life | SF-36 |  | 
| Disease biomarkers | Blood biomarker levels |  | 
| Cardiac function | Echocardiogram |  | 
| Safety | Number of adverse events |  | 
| Disease improvement | Patient Global Improvement Scale |  | 
| Visual Function | Visual field exam | Baseline, Months 3, 6, 9 and 12 | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
FA, EPI-743, Friedreich's, Ataxia, FRDA, Edison