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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Edison Pharmaceuticals Inc
ClinicalTrials.gov Identifier:
NCT01728064
First received: November 13, 2012
Last updated: December 4, 2013
Last verified: December 2013
  Purpose

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.


Condition Intervention Phase
Friedreich's Ataxia
Drug: Placebo
Drug: EPI-743 400 mg
Drug: EPI-743 200 mg
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Safety and Efficacy Study of EPI-743 on Visual Function in Patients With Friedreich's Ataxia

Resource links provided by NLM:


Further study details as provided by Edison Pharmaceuticals Inc:

Primary Outcome Measures:
  • Visual Function [ Designated as safety issue: No ]
    Low contrast visual acuity


Secondary Outcome Measures:
  • Color vision [ Designated as safety issue: No ]
    Roth 28 hue test

  • Neurologic function
    Friedreich's ataxia rating scale

  • Neuromuscular function [ Designated as safety issue: No ]
    25-foot walk test

  • Neuromuscular function [ Designated as safety issue: No ]
    9-hole peg test

  • Quality of life [ Designated as safety issue: No ]
    SF-36

  • Disease biomarkers
    Blood biomarker levels

  • Cardiac function [ Designated as safety issue: No ]
    Echocardiogram

  • Safety [ Designated as safety issue: Yes ]
    Number of adverse events

  • Disease improvement
    Patient Global Improvement Scale

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


Estimated Enrollment: 60
Study Start Date: December 2012
Estimated Study Completion Date: April 2014
Estimated Primary Completion Date: March 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Placebo Comparator: Placebo
Placebo capsules three times daily
Drug: Placebo
Placebo capsules
Active Comparator: EPI-743 400 mg
EPI-743 at a dose of 400 mg three times daily
Drug: EPI-743 400 mg
Active Comparator: EPI-743 200 mg
EPI-743 at a dose of 200 mg three times daily
Drug: EPI-743 200 mg

  Eligibility

Ages Eligible for Study:   18 Years to 45 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01728064

Locations
United States, California
University of California Los Angeles
Los Angeles, California, United States, 90095
United States, Florida
University of South Florida
Tampa, Florida, United States, 33612
United States, Pennsylvania
Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
Edison Pharmaceuticals Inc
  More Information

Additional Information:
No publications provided

Responsible Party: Edison Pharmaceuticals Inc
ClinicalTrials.gov Identifier: NCT01728064     History of Changes
Other Study ID Numbers: EPI2010-006
Study First Received: November 13, 2012
Last Updated: December 4, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Edison Pharmaceuticals Inc:
Friedreich's
Ataxia
FRDA
FA
EPI-743
Edison

Additional relevant MeSH terms:
Ataxia
Friedreich Ataxia
Brain Diseases
Central Nervous System Diseases
Cerebellar Diseases
Dyskinesias
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Metabolic Diseases
Mitochondrial Diseases
Nervous System Diseases
Neurodegenerative Diseases
Neurologic Manifestations
Signs and Symptoms
Spinal Cord Diseases
Spinocerebellar Degenerations

ClinicalTrials.gov processed this record on November 25, 2014