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

Completed · Phase 2 · Has a placebo group

Conditions studied: Friedreich's Ataxia

In brief

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.

Key facts

Study ID

NCT01728064

Run by

PTC Therapeutics

People needed

60

Starts

2012-12-31

Expected to finish

2016-02-29

Last updated by the study team

2020-08-31

Who can join

Age: 18 and older, up to 45. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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.

Where it is running

• University of California Los Angeles — Los Angeles, California, United States
• University of South Florida — Tampa, Florida, United States
• Childrens Hospital of Philadelphia — Philadelphia, Pennsylvania, United States

Full record on ClinicalTrials.gov

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