Trial record 4 of 8 for:    Open Studies | "Friedreich Ataxia"

EPI-743 in Friedreich's Ataxia Point Mutations

This study is currently recruiting participants. (see Contacts and Locations)
Verified June 2014 by University of South Florida
Sponsor:
Collaborators:
Edison Pharmaceuticals Inc
Friedreich's Ataxia Research Alliance
Information provided by (Responsible Party):
University of South Florida
ClinicalTrials.gov Identifier:
NCT01962363
First received: October 10, 2013
Last updated: July 7, 2014
Last verified: June 2014
  Purpose

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 Point Mutation
Drug: EPI-743
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
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 ] [ Designated as safety issue: No ]
    Low contrast acuity

  • Safety parameters [ Time Frame: 3 months ] [ Designated as safety issue: Yes ]
    Clinical and laboratory safety parameters


Secondary Outcome Measures:
  • Visual function [ Time Frame: 3 months ] [ Designated as safety issue: No ]
    Visual fields as assessed by Humphrey 30-2 exam; High contrast visual acuity

  • Neurologic function [ Time Frame: 3 months ] [ Designated as safety issue: No ]
    Friedreich's Ataxia Rating Scale

  • Physical function [ Time Frame: 3 months ] [ Designated as safety issue: No ]
    25-foot walk (for subjects capable of completing the test on enrollment);

  • Health related quality of life [ Time Frame: 3 months ] [ Designated as safety issue: No ]
    Patient report via rating scale

  • Activities of Daily Living [ Time Frame: 3 months ] [ Designated as safety issue: No ]
    Patient report via rating scale

  • Cardiac indices [ Time Frame: 3 months ] [ Designated as safety issue: Yes ]
    Echocardiogram

  • Upper extremity function [ Time Frame: 3 months ] [ Designated as safety issue: No ]
    9 hole peg test

  • Disease biomarkers [ Time Frame: 3 months ] [ Designated as safety issue: No ]
    Glutathione cycle components


Estimated Enrollment: 4
Study Start Date: October 2013
Estimated Primary Completion Date: July 2015 (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
Other Name: Vincerinone

  Eligibility

Ages Eligible for Study:   18 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
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: NCT01962363

Contacts
Contact: Jessica Shaw, MPH 813-974-5909 jshaw@health.usf.edu
Contact: Kevin Allison, BS 813-974-5909 kalliso1@health.usf.edu

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

No publications provided

Responsible Party: University of South Florida
ClinicalTrials.gov Identifier: NCT01962363     History of Changes
Other Study ID Numbers: EPI-743 PM
Study First Received: October 10, 2013
Last Updated: July 7, 2014
Health Authority: United States: Food and Drug Administration

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

ClinicalTrials.gov processed this record on September 18, 2014