Back to resultsEPI-743 in Friedreich's Ataxia Point Mutations
Primary Purpose
Friedreich's Ataxia
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
EPI-743
Sponsored by
About this trial
This is an interventional treatment trial for Friedreich's Ataxia focused on measuring Friedreich's ataxia point mutation
Eligibility Criteria
Inclusion Criteria:
- Diagnosis of genetically confirmed Friedreich's ataxia point mutation.
- Visual acuity at baseline more than 15 letters on high contrast EDTRS at four meters.
- FARS score of 20 to 90.
- Male or female between 18 and 65 years of age.
- Agreement to use contraception if within reproductive years
- 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 metropolitan area 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.
- Abstention from foods or beverages or bars fortified with Coenzyme Q10, vitamin E, super fortified functional foods or beverages at least 30 days prior to initiation of treatment and for the duration of the study.
- Abstention from use of other investigative or non-approved drugs within 30 days of enrollment and for the duration of the study.
- Subject can swallow multiple size 0 capsules.
- Subject has voluntarily signed an IRB approved informed consent form to participate in the study after all relevant aspects of the study have been explained and discussed with the subject.
Exclusion Criteria:
- 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.
- Clinically significant cardiomyopathy with ejection fraction < 40 percent at screening.
- Clinically significant arrhythmia within past two years requiring treatment.
- Surgery planned through the duration of the study, including follow-up.
- Pregnancy or breastfeeding.
- Anticoagulant therapy within 30 days of enrollment.
Sites / Locations
- University of South Florida
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
EPI-743
Arm Description
EPI-743, oral, 400mg three times daily for 3 months
Outcomes
Primary Outcome Measures
Visual function
Low contrast acuity
Safety parameters
Clinical and laboratory safety parameters
Secondary Outcome Measures
Visual function
Visual fields as assessed by Humphrey 30-2 exam; High contrast visual acuity
Neurologic function
Friedreich's Ataxia Rating Scale
Physical function
25-foot walk (for subjects capable of completing the test on enrollment);
Health related quality of life
Patient report via rating scale
Activities of Daily Living
Patient report via rating scale
Cardiac indices
Echocardiogram
Upper extremity function
9 hole peg test
Disease biomarkers
Glutathione cycle components
Full Information
NCT ID
NCT01962363
First Posted
October 10, 2013
Last Updated
June 28, 2016
Sponsor
University of South Florida
Collaborators
Edison Pharmaceuticals Inc, Friedreich's Ataxia Research Alliance
1. Study Identification
Unique Protocol Identification Number
NCT01962363
Brief Title
EPI-743 in Friedreich's Ataxia Point Mutations
Official Title
A Phase 2A Clinical Trial of EPI-743 (Vincerinone™) on Visual Function in Friedreich's Ataxia Patients With Point Mutations
Study Type
Interventional
2. Study Status
Record Verification Date
June 2016
Overall Recruitment Status
Completed
Study Start Date
October 2013 (undefined)
Primary Completion Date
June 2016 (Actual)
Study Completion Date
June 2016 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University of South Florida
Collaborators
Edison Pharmaceuticals Inc, Friedreich's Ataxia Research Alliance
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
The purpose of this study is to evaluate the effects of EPI-743 in patients with Friedreich's Ataxia point mutations
Detailed Description
The primary objective of this study is to investigate whether treatment with EPI-743 has a discernible impact on visual function-including visual acuity, visual fields and color vision as well as on any of a number of functional and subject/clinician-rated scales relevant in the treatment of Friedreich's ataxia, and to determine the safety of treatment with EPI-743.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Friedreich's Ataxia
Keywords
Friedreich's ataxia point mutation
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
4 (Actual)
8. Arms, Groups, and Interventions
Arm Title
EPI-743
Arm Type
Experimental
Arm Description
EPI-743, oral, 400mg three times daily for 3 months
Intervention Type
Drug
Intervention Name(s)
EPI-743
Other Intervention Name(s)
Vincerinone, alpha-tocotrienol quinone
Intervention Description
EPI-743 (alpha-tocotrienol quinone) is a small molecule therapeutic that was rationally designed to replete reduced glutathione through NQO1-catalyzed electron transfer from NADPH.
Primary Outcome Measure Information:
Title
Visual function
Description
Low contrast acuity
Time Frame
3 months
Title
Safety parameters
Description
Clinical and laboratory safety parameters
Time Frame
3 months
Secondary Outcome Measure Information:
Title
Visual function
Description
Visual fields as assessed by Humphrey 30-2 exam; High contrast visual acuity
Time Frame
3 months
Title
Neurologic function
Description
Friedreich's Ataxia Rating Scale
Time Frame
3 months
Title
Physical function
Description
25-foot walk (for subjects capable of completing the test on enrollment);
Time Frame
3 months
Title
Health related quality of life
Description
Patient report via rating scale
Time Frame
3 months
Title
Activities of Daily Living
Description
Patient report via rating scale
Time Frame
3 months
Title
Cardiac indices
Description
Echocardiogram
Time Frame
3 months
Title
Upper extremity function
Description
9 hole peg test
Time Frame
3 months
Title
Disease biomarkers
Description
Glutathione cycle components
Time Frame
3 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Diagnosis of genetically confirmed Friedreich's ataxia point mutation.
Visual acuity at baseline more than 15 letters on high contrast EDTRS at four meters.
FARS score of 20 to 90.
Male or female between 18 and 65 years of age.
Agreement to use contraception if within reproductive years
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 metropolitan area 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.
Abstention from foods or beverages or bars fortified with Coenzyme Q10, vitamin E, super fortified functional foods or beverages at least 30 days prior to initiation of treatment and for the duration of the study.
Abstention from use of other investigative or non-approved drugs within 30 days of enrollment and for the duration of the study.
Subject can swallow multiple size 0 capsules.
Subject has voluntarily signed an IRB approved informed consent form to participate in the study after all relevant aspects of the study have been explained and discussed with the subject.
Exclusion Criteria:
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.
Clinically significant cardiomyopathy with ejection fraction < 40 percent at screening.
Clinically significant arrhythmia within past two years requiring treatment.
Surgery planned through the duration of the study, including follow-up.
Pregnancy or breastfeeding.
Anticoagulant therapy within 30 days of enrollment.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Theresa A Zesiewicz, MD
Organizational Affiliation
University of South Florida
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of South Florida
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
12. IPD Sharing Statement
Learn more about this trial
EPI-743 in Friedreich's Ataxia Point Mutations
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