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EPI-743 in Friedreich's Ataxia Point Mutations

This study has been completed.
Edison Pharmaceuticals Inc
Friedreich's Ataxia Research Alliance
Information provided by (Responsible Party):
University of South Florida Identifier:
First received: October 10, 2013
Last updated: June 28, 2016
Last verified: June 2016
The purpose of this study is to evaluate the effects of EPI-743 in patients with Friedreich's Ataxia point mutations

Condition Intervention Phase
Friedreich's Ataxia
Drug: EPI-743
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase 2A Clinical Trial of EPI-743 (Vincerinone™) on Visual Function in Friedreich's Ataxia Patients With Point Mutations

Resource links provided by NLM:

Further study details as provided by University of South Florida:

Primary Outcome Measures:
  • Visual function [ Time Frame: 3 months ]
    Low contrast acuity

  • Safety parameters [ Time Frame: 3 months ]
    Clinical and laboratory safety parameters

Secondary Outcome Measures:
  • Visual function [ Time Frame: 3 months ]
    Visual fields as assessed by Humphrey 30-2 exam; High contrast visual acuity

  • Neurologic function [ Time Frame: 3 months ]
    Friedreich's Ataxia Rating Scale

  • Physical function [ Time Frame: 3 months ]
    25-foot walk (for subjects capable of completing the test on enrollment);

  • Health related quality of life [ Time Frame: 3 months ]
    Patient report via rating scale

  • Activities of Daily Living [ Time Frame: 3 months ]
    Patient report via rating scale

  • Cardiac indices [ Time Frame: 3 months ]

  • Upper extremity function [ Time Frame: 3 months ]
    9 hole peg test

  • Disease biomarkers [ Time Frame: 3 months ]
    Glutathione cycle components

Enrollment: 4
Study Start Date: October 2013
Study Completion Date: June 2016
Primary Completion Date: June 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: EPI-743
EPI-743, oral, 400mg three times daily for 3 months
Drug: EPI-743
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.
Other Name: Vincerinone, alpha-tocotrienol quinone

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.

Ages Eligible for Study:   18 Years to 65 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Diagnosis of genetically confirmed Friedreich's ataxia point mutation.
  2. Visual acuity at baseline more than 15 letters on high contrast EDTRS at four meters.
  3. FARS score of 20 to 90.
  4. Male or female between 18 and 65 years of age.
  5. Agreement to use contraception if within reproductive years
  6. Hormone replacement therapy, if used, must remain stable for the duration of the study.
  7. Willingness and ability to comply with study procedures.
  8. Willingness and ability to arrive at study site metropolitan area day prior to evaluations.
  9. 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.
  10. 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.
  11. Abstention from use of other investigative or non-approved drugs within 30 days of enrollment and for the duration of the study.
  12. Subject can swallow multiple size 0 capsules.
  13. 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:

  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. Clinically significant cardiomyopathy with ejection fraction < 40 percent at screening.
  9. Clinically significant arrhythmia within past two years requiring treatment.
  10. Surgery planned through the duration of the study, including follow-up.
  11. Pregnancy or breastfeeding.
  12. Anticoagulant therapy within 30 days of enrollment.
  Contacts and Locations
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Please refer to this study by its identifier: NCT01962363

United States, Florida
University of South Florida
Tampa, Florida, United States, 33612
Sponsors and Collaborators
University of South Florida
Edison Pharmaceuticals Inc
Friedreich's Ataxia Research Alliance
Principal Investigator: Theresa A Zesiewicz, MD University of South Florida
  More Information

Responsible Party: University of South Florida Identifier: NCT01962363     History of Changes
Other Study ID Numbers: EPI-743 PM
Study First Received: October 10, 2013
Last Updated: June 28, 2016

Keywords provided by University of South Florida:
Friedreich's ataxia point mutation

Additional relevant MeSH terms:
Cerebellar Ataxia
Friedreich Ataxia
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinocerebellar Degenerations
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases
Vitamin E
Tocotrienol, alpha
Growth Substances
Physiological Effects of Drugs
Molecular Mechanisms of Pharmacological Action
Protective Agents
Vitamins processed this record on April 26, 2017