<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT01728064</url>
  </required_header>
  <id_info>
    <org_study_id>EPI2010-006</org_study_id>
    <nct_id>NCT01728064</nct_id>
  </id_info>
  <brief_title>Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia</brief_title>
  <official_title>Safety and Efficacy Study of EPI-743 on Visual Function in Patients With Friedreich's Ataxia</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>PTC Therapeutics</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>PTC Therapeutics</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this study is to examine the effects of EPI-743 on visual function and&#xD;
      neurologic function in patients with Friedreich's ataxia.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">December 31, 2012</start_date>
  <completion_date type="Actual">February 29, 2016</completion_date>
  <primary_completion_date type="Actual">October 31, 2015</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Visual Function</measure>
    <description>Low contrast visual acuity</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Color vision</measure>
    <description>Roth 28 hue test</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Neurologic function</measure>
    <description>Friedreich's ataxia rating scale</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Neuromuscular function</measure>
    <description>25-foot walk test</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Neuromuscular function</measure>
    <description>9-hole peg test</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Quality of life</measure>
    <description>SF-36</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Disease biomarkers</measure>
    <description>Blood biomarker levels</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Cardiac function</measure>
    <description>Echocardiogram</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Safety</measure>
    <description>Number of adverse events</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Disease improvement</measure>
    <description>Patient Global Improvement Scale</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Visual Function</measure>
    <time_frame>Baseline, Months 3, 6, 9 and 12</time_frame>
    <description>Visual field exam</description>
  </secondary_outcome>
  <number_of_arms>3</number_of_arms>
  <enrollment type="Anticipated">60</enrollment>
  <condition>Friedreich's Ataxia</condition>
  <arm_group>
    <arm_group_label>Placebo</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Placebo capsules three times daily</description>
  </arm_group>
  <arm_group>
    <arm_group_label>EPI-743 400 mg</arm_group_label>
    <arm_group_type>Active Comparator</arm_group_type>
    <description>EPI-743 at a dose of 400 mg three times daily</description>
  </arm_group>
  <arm_group>
    <arm_group_label>EPI-743 200 mg</arm_group_label>
    <arm_group_type>Active Comparator</arm_group_type>
    <description>EPI-743 at a dose of 200 mg three times daily</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebo</intervention_name>
    <description>Placebo capsules</description>
    <arm_group_label>Placebo</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>EPI-743 400 mg</intervention_name>
    <arm_group_label>EPI-743 400 mg</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>EPI-743 200 mg</intervention_name>
    <arm_group_label>EPI-743 200 mg</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Diagnosis of genetically confirmed Friedreich's ataxia&#xD;
&#xD;
          2. Visual acuity at baseline more than 15 letters on EDTRS at four meters&#xD;
&#xD;
          3. FARS score of 20 to 90&#xD;
&#xD;
          4. Agreement to use contraception if within reproductive years (see specifics in section&#xD;
             D1, page 21)&#xD;
&#xD;
          5. Hormone replacement therapy, if used, must remain stable for the duration of the study&#xD;
&#xD;
          6. Willingness and ability to comply with study procedures&#xD;
&#xD;
          7. Willingness and ability to arrive at study site day prior to evaluations&#xD;
&#xD;
          8. Abstention from use of dietary supplements and non-prescribed medications at least 30&#xD;
             days prior to initiation of treatment and for the duration of the study. This would&#xD;
             specifically include idebenone, Coenzyme Q10 and vitamin E&#xD;
&#xD;
          9. Abstention from use of other investigative or non-approved drugs within 30 days of&#xD;
             enrollment and for the duration of the study&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Allergy to EPI-743 or sesame oil or nuts&#xD;
&#xD;
          2. Clinically significant bleeding condition or abnormal PT/PTT INR (INR &gt; two; PTT &gt;&#xD;
             two-times normal)&#xD;
&#xD;
          3. Liver insufficiency with LFTs greater than three-times upper normal limit at screening&#xD;
&#xD;
          4. Renal insufficiency with creatinine &gt; 1.5 at screening&#xD;
&#xD;
          5. Fat malabsorption syndromes&#xD;
&#xD;
          6. Any other respiratory chain diseases of the mitochondria or inborn errors of&#xD;
             metabolism&#xD;
&#xD;
          7. Any other ophthalmologic conditions&#xD;
&#xD;
          8. History of alcohol or drug abuse&#xD;
&#xD;
          9. Clinically significant cardiomyopathy with ejection fraction &lt; 40 percent at screening&#xD;
&#xD;
         10. Clinically significant arrhythmia within past two years requiring treatment&#xD;
&#xD;
         11. Anticoagulant therapy within 30 days of enrollment.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>45 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>University of California Los Angeles</name>
      <address>
        <city>Los Angeles</city>
        <state>California</state>
        <zip>90095</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of South Florida</name>
      <address>
        <city>Tampa</city>
        <state>Florida</state>
        <zip>33612</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Childrens Hospital of Philadelphia</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <zip>19104</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <link>
    <url>http://edisonpharma.com</url>
    <description>Edison Pharmaceuticals Web Site</description>
  </link>
  <verification_date>August 2020</verification_date>
  <study_first_submitted>November 13, 2012</study_first_submitted>
  <study_first_submitted_qc>November 13, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">November 16, 2012</study_first_posted>
  <disposition_first_submitted>August 17, 2020</disposition_first_submitted>
  <disposition_first_submitted_qc>August 24, 2020</disposition_first_submitted_qc>
  <disposition_first_posted type="Actual">August 31, 2020</disposition_first_posted>
  <last_update_submitted>August 24, 2020</last_update_submitted>
  <last_update_submitted_qc>August 24, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">August 31, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Friedreich's</keyword>
  <keyword>Ataxia</keyword>
  <keyword>FRDA</keyword>
  <keyword>FA</keyword>
  <keyword>EPI-743</keyword>
  <keyword>Edison</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Ataxia</mesh_term>
    <mesh_term>Cerebellar Ataxia</mesh_term>
    <mesh_term>Friedreich Ataxia</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Ubiquinone</mesh_term>
  </intervention_browse>
  <!-- Results have not yet been posted, but a results disposition has been submitted           -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT01728064
org study id: EPI2010-006
nct id: NCT01728064
lead sponsor: 
has dmc: Yes
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.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
measure: Visual Function
description: Low contrast visual acuity
measure: Color vision
description: Roth 28 hue test
measure: Neurologic function
description: Friedreich's ataxia rating scale
measure: Neuromuscular function
description: 25-foot walk test
measure: Neuromuscular function
description: 9-hole peg test
measure: Quality of life
description: SF-36
measure: Disease biomarkers
description: Blood biomarker levels
measure: Cardiac function
description: Echocardiogram
measure: Safety
description: Number of adverse events
measure: Disease improvement
description: Patient Global Improvement Scale
measure: Visual Function
time frame: Baseline, Months 3, 6, 9 and 12
description: Visual field exam
arm group label: Placebo
arm group type: Placebo Comparator
description: Placebo capsules three times daily
arm group label: EPI-743 400 mg
arm group type: Active Comparator
description: EPI-743 at a dose of 400 mg three times daily
arm group label: EPI-743 200 mg
arm group type: Active Comparator
description: EPI-743 at a dose of 200 mg three times daily
intervention type: Drug
intervention name: Placebo
description: Placebo capsules
arm group label: Placebo
intervention type: Drug
intervention name: EPI-743 400 mg
arm group label: EPI-743 400 mg
intervention type: Drug
intervention name: EPI-743 200 mg
arm group label: EPI-743 200 mg
criteria: 
gender: All
minimum age: 18 Years
maximum age: 45 Years
healthy volunteers: No
facility: 
facility: 
facility: 
country: United States
url: http://edisonpharma.com
description: Edison Pharmaceuticals Web Site
responsible party type: Sponsor
mesh term: Ataxia
mesh term: Cerebellar Ataxia
mesh term: Friedreich Ataxia
mesh term: Ubiquinone