Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia
NCT ID: NCT01728064
Last Updated: 2020-08-31
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE2
60 participants
INTERVENTIONAL
2012-12-31
2016-02-29
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Placebo
Placebo capsules three times daily
Placebo
Placebo capsules
EPI-743 400 mg
EPI-743 at a dose of 400 mg three times daily
EPI-743 400 mg
EPI-743 200 mg
EPI-743 at a dose of 200 mg three times daily
EPI-743 200 mg
Interventions
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Placebo
Placebo capsules
EPI-743 400 mg
EPI-743 200 mg
Eligibility Criteria
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Inclusion Criteria
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
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.
18 Years
45 Years
ALL
No
Sponsors
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PTC Therapeutics
INDUSTRY
Responsible Party
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Locations
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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
Countries
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Related Links
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Edison Pharmaceuticals Web Site
Other Identifiers
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EPI2010-006
Identifier Type: -
Identifier Source: org_study_id
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