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.

Age range18 Years – 45 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. 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

✕. 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

Visual Function

NCT IDNCT01728064

SponsorPTC Therapeutics

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2015-10-31