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

Launched by PTC THERAPEUTICS · Nov 13, 2012

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Eligibility criteria

• Inclusion Criteria:
• • 1. Diagnosis of genetically confirmed Friedreich's ataxia
• • 2. Visual acuity at baseline more than 15 letters on EDTRS at four meters
• • 3. FARS score of 20 to 90
• • 4. Agreement to use contraception if within reproductive years (see specifics in section D1, page 21)
• • 5. Hormone replacement therapy, if used, must remain stable for the duration of the study
• • 6. Willingness and ability to comply with study procedures
• • 7. Willingness and ability to arrive at study site day prior to evaluations
• • 8. 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
• • 9. Abstention from use of other investigative or non-approved drugs within 30 days of enrollment and for the duration of the study
• Exclusion Criteria:
• • 1. Allergy to EPI-743 or sesame oil or nuts
• • 2. Clinically significant bleeding condition or abnormal PT/PTT INR (INR \> two; PTT \> two-times normal)
• • 3. Liver insufficiency with LFTs greater than three-times upper normal limit at screening
• • 4. Renal insufficiency with creatinine \> 1.5 at screening
• • 5. Fat malabsorption syndromes
• • 6. Any other respiratory chain diseases of the mitochondria or inborn errors of metabolism
• • 7. Any other ophthalmologic conditions
• • 8. History of alcohol or drug abuse
• • 9. Clinically significant cardiomyopathy with ejection fraction \< 40 percent at screening
• • 10. Clinically significant arrhythmia within past two years requiring treatment
• • 11. Anticoagulant therapy within 30 days of enrollment.