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Trial record 7 of 18 for:    Friedreich Ataxia | ( Map: Florida, United States )

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01728064
Recruitment Status : Completed
First Posted : November 16, 2012
Last Update Posted : August 31, 2020
Sponsor:
Information provided by (Responsible Party):
PTC Therapeutics

Tracking Information
First Submitted Date  ICMJE November 13, 2012
First Posted Date  ICMJE November 16, 2012
Last Update Posted Date August 31, 2020
Actual Study Start Date  ICMJE December 31, 2012
Actual Primary Completion Date October 31, 2015   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 1, 2013)
Visual Function
Low contrast visual acuity
Original Primary Outcome Measures  ICMJE
 (submitted: November 13, 2012)
  • Visual Function
    Low contrast visual acuity
  • Visual Function
    Visual fields
Change History
Current Secondary Outcome Measures  ICMJE
 (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  ICMJE
 (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  ICMJE Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia
Official Title  ICMJE 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  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Friedreich's Ataxia
Intervention  ICMJE
  • Drug: Placebo
    Placebo capsules
  • Drug: EPI-743 400 mg
  • Drug: EPI-743 200 mg
Study Arms  ICMJE
  • 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

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Estimated Enrollment  ICMJE
 (submitted: November 13, 2012)
60
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE February 29, 2016
Actual Primary Completion Date October 31, 2015   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 45 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01728064
Other Study ID Numbers  ICMJE EPI2010-006
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party PTC Therapeutics
Study Sponsor  ICMJE PTC Therapeutics
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account PTC Therapeutics
Verification Date August 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP