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

• ClinicalTrials.gov Identifier: NCT01728064
• Recruitment Status: Completed
• First Posted: November 16, 2012
• Last Update Posted: August 31, 2020
• Sponsor: PTC Therapeutics
• Information provided by (Responsible Party): PTC Therapeutics

Tracking Information

• First Submitted Date: November 13, 2012
• First Posted Date: November 16, 2012
• Last Update Posted Date: August 31, 2020
• Actual Study Start Date: December 31, 2012
• Actual Primary Completion Date: October 31, 2015 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: August 1, 2013)

Visual Function

Low contrast visual acuity

Original Primary Outcome Measures (submitted: November 13, 2012)

• Visual Function
  Low contrast visual acuity
• Visual Function
  Visual fields

Current Secondary Outcome Measures (submitted: August 1, 2013)

• 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 [ Time Frame: Baseline, Months 3, 6, 9 and 12 ]
  Visual field exam

Original Secondary Outcome Measures (submitted: November 13, 2012)

• 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

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia
• Official Title: Safety and Efficacy Study of EPI-743 on Visual Function in Patients With Friedreich's Ataxia
• 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.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
• Condition: Friedreich's Ataxia

Intervention

• Drug: Placebo
  Placebo capsules
• Drug: EPI-743 400 mg
• Drug: EPI-743 200 mg

Study Arms

• Placebo Comparator: Placebo
  Placebo capsules three times daily
  Intervention: Drug: Placebo
• Active Comparator: EPI-743 400 mg
  EPI-743 at a dose of 400 mg three times daily
  Intervention: Drug: EPI-743 400 mg
• Active Comparator: EPI-743 200 mg
  EPI-743 at a dose of 200 mg three times daily
  Intervention: Drug: EPI-743 200 mg

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Estimated Enrollment (submitted: November 13, 2012): 60
• Original Estimated Enrollment: Same as current
• Actual Study Completion Date: February 29, 2016
• Actual Primary Completion Date: October 31, 2015 (Final data collection date for primary outcome measure)

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 45 Years (Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT01728064
• Other Study ID Numbers: EPI2010-006
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Responsible Party: PTC Therapeutics
• Study Sponsor: PTC Therapeutics
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: PTC Therapeutics
• Verification Date: August 2020