<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/NCT02251457</url>
  </required_header>
  <id_info>
    <org_study_id>IN-US-259-1605</org_study_id>
    <secondary_id>IN-US-259-1605</secondary_id>
    <nct_id>NCT02251457</nct_id>
  </id_info>
  <brief_title>Study of Ranolazine in Myotonia Congenita, Paramyotonia Congenita and Myotonic Dystrophy Type 1</brief_title>
  <official_title>Open Label Trial of Ranolazine in Myotonia Congenita, Paramyotonia Congenita, &amp; Myotonic Dystrophy Type 1</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Ohio State University</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Gilead Sciences</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
  </sponsors>
  <source>Ohio State University</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this study is to gather preliminary data to determine if ranolazine is a safe&#xD;
      and effective treatment for the symptoms of myotonia congenital, paramyotonia congenita, and&#xD;
      myotonic dystrophy type 1. The duration of the study is 5 weeks.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Recent advances in the understanding of myotonia congenita have identified potential areas&#xD;
      that could possibly respond to treatment in a drug study. The drug ranolazine (trade name&#xD;
      Ranexa) is a FDA-approved medication to treat chest pain in patients with heart disease.&#xD;
      Ranolazine has been studied in mice with myotonia congenita. The data from this animal model&#xD;
      suggest that ranolazine may improve the symptoms and signs of myotonia. All individuals that&#xD;
      participate will be placed on active drug. The investigators want to see if this drug is safe&#xD;
      to take without causing too many side effects for people with myotonia congenita,&#xD;
      paramyotonia congenital and myotonic dystrophy type 1. Participants will go to The Ohio State&#xD;
      University for study visits. Participants will take ranolazine for four weeks. Participants&#xD;
      can expect a total of 4 study visits and 2 phone calls over the 5 week period.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date>August 2014</start_date>
  <completion_date type="Actual">December 18, 2017</completion_date>
  <primary_completion_date type="Actual">December 18, 2017</primary_completion_date>
  <phase>Phase 1</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Questionnaires: Short Form Health Survey (SF-36) and Individualized Neuromuscular Quality of Life Questionnaire (INQoL)</measure>
    <time_frame>1 month</time_frame>
    <description>quality of life measurements for overall health and neuromuscular disease</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Muscle tasks</measure>
    <time_frame>1 month</time_frame>
    <description>The subject is observed and timed while rising from an arm chair, walking 3 meters, turning, walking back, and sitting down again</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Electromyography (EMG) Myotonia</measure>
    <time_frame>1 month</time_frame>
    <description>To see if the electrical potentials produced by the muscle fibers change.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Electrocardiogram (ECG)</measure>
    <time_frame>1 month</time_frame>
    <description>to measure heart function and observe QT interval (a measure of the time between the start of the Q wave and the end of the T wave in the heart's electrical cycle)</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">35</enrollment>
  <condition>Myotonia Congenita</condition>
  <condition>Paramyotonia Congenita</condition>
  <condition>Myotonic Dystrophy 1</condition>
  <arm_group>
    <arm_group_label>ranolazine</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>ranolazine 500mg, twice daily for two weeks; 1000mg twice daily for 2 weeks</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Ranolazine</intervention_name>
    <description>Ranexa is FDA approved for chronic angina</description>
    <arm_group_label>ranolazine</arm_group_label>
    <other_name>Ranexa®</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Diagnosis of myotonia congenital, paramyotonia congenital or Myotonic Dystrophy Type 1&#xD;
             established by genetic testing in the subject or in a first-degree relative.&#xD;
&#xD;
          -  Clinically evident myotonia&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Contraindications to ranolazine use:&#xD;
&#xD;
               -  for fungus infection: ketoconazole (Nizoral), itraconazole (Sporanox, Onmel)&#xD;
&#xD;
               -  for infection: clarithromycin (Biaxin)&#xD;
&#xD;
               -  for depression: nefazodone&#xD;
&#xD;
               -  for HIV: nelfinavir (Viracept), ritonavir (Norvir), lopinavir and ritonavir&#xD;
                  (Kaletra), indinavir (Crixivan), saquinavir (Invirase).&#xD;
&#xD;
               -  for tuberculosis (TB): rifampin (Rifadin), rifabutin (Mycobutin), rifapentine&#xD;
                  (Priftin)&#xD;
&#xD;
               -  for seizures: phenobarbital, phenytoin (Phenytek, Dilantin, Dilantin-125),&#xD;
                  carbamazepine (Tegretol)&#xD;
&#xD;
               -  the herbal supplement St. John's wort&#xD;
&#xD;
                    -  you have scarring (cirrhosis) of your liver&#xD;
&#xD;
          -  Concurrent use of mexiletine, lacosamide, acetazolamide, phenytoin, quinine,&#xD;
             procainamide, Saint John wort or tocainide. Patients who were previously treated with&#xD;
             these medications may participate. They need to be off of the medication for at least&#xD;
             a week prior to enrollment.&#xD;
&#xD;
          -  QTc &gt;470 ms for men and &gt;480 ms for women.&#xD;
&#xD;
          -  Women who are pregnant or breastfeeding&#xD;
&#xD;
          -  Direct family history of sudden cardiac death&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>100 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>William D Arnold, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Ohio State University</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>The Ohio State University Wexner Medical Center</name>
      <address>
        <city>Columbus</city>
        <state>Ohio</state>
        <zip>43221</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <reference>
    <citation>Novak KR, Norman J, Mitchell JR, Pinter MJ, Rich MM. Sodium channel slow inactivation as a therapeutic target for myotonia congenita. Ann Neurol. 2015 Feb;77(2):320-32. doi: 10.1002/ana.24331. Epub 2015 Jan 9.</citation>
    <PMID>25515836</PMID>
  </reference>
  <verification_date>March 2019</verification_date>
  <study_first_submitted>September 25, 2014</study_first_submitted>
  <study_first_submitted_qc>September 26, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">September 29, 2014</study_first_posted>
  <last_update_submitted>March 1, 2019</last_update_submitted>
  <last_update_submitted_qc>March 1, 2019</last_update_submitted_qc>
  <last_update_posted type="Actual">March 5, 2019</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Ohio State University</investigator_affiliation>
    <investigator_full_name>William Arnold</investigator_full_name>
    <investigator_title>Associate Professor</investigator_title>
  </responsible_party>
  <keyword>Myotonia Congenita</keyword>
  <keyword>ranolazine</keyword>
  <keyword>Ohio State</keyword>
  <keyword>Open Label</keyword>
  <keyword>Paramyotonia Congenita</keyword>
  <keyword>Myotonic Dystrophy 1</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Myotonic Dystrophy</mesh_term>
    <mesh_term>Myotonia Congenita</mesh_term>
    <mesh_term>Myotonic Disorders</mesh_term>
    <mesh_term>Myotonia</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Ranolazine</mesh_term>
  </intervention_browse>
  <patient_data>
    <sharing_ipd>Undecided</sharing_ipd>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</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/NCT02251457
org study id: IN-US-259-1605
secondary id: IN-US-259-1605
nct id: NCT02251457
lead sponsor: 
collaborator: 
has dmc: No
The purpose of this study is to gather preliminary data to determine if ranolazine is a safe
      and effective treatment for the symptoms of myotonia congenital, paramyotonia congenita, and
      myotonic dystrophy type 1. The duration of the study is 5 weeks.
Recent advances in the understanding of myotonia congenita have identified potential areas
      that could possibly respond to treatment in a drug study. The drug ranolazine (trade name
      Ranexa) is a FDA-approved medication to treat chest pain in patients with heart disease.
      Ranolazine has been studied in mice with myotonia congenita. The data from this animal model
      suggest that ranolazine may improve the symptoms and signs of myotonia. All individuals that
      participate will be placed on active drug. The investigators want to see if this drug is safe
      to take without causing too many side effects for people with myotonia congenita,
      paramyotonia congenital and myotonic dystrophy type 1. Participants will go to The Ohio State
      University for study visits. Participants will take ranolazine for four weeks. Participants
      can expect a total of 4 study visits and 2 phone calls over the 5 week period.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Questionnaires: Short Form Health Survey (SF-36) and Individualized Neuromuscular Quality of Life Questionnaire (INQoL)
time frame: 1 month
description: quality of life measurements for overall health and neuromuscular disease
measure: Muscle tasks
time frame: 1 month
description: The subject is observed and timed while rising from an arm chair, walking 3 meters, turning, walking back, and sitting down again
measure: Electromyography (EMG) Myotonia
time frame: 1 month
description: To see if the electrical potentials produced by the muscle fibers change.
measure: Electrocardiogram (ECG)
time frame: 1 month
description: to measure heart function and observe QT interval (a measure of the time between the start of the Q wave and the end of the T wave in the heart's electrical cycle)
arm group label: ranolazine
arm group type: Experimental
description: ranolazine 500mg, twice daily for two weeks; 1000mg twice daily for 2 weeks
intervention type: Drug
intervention name: Ranolazine
description: Ranexa is FDA approved for chronic angina
arm group label: ranolazine
other name: Ranexa®
criteria: 
gender: All
minimum age: 18 Years
maximum age: 100 Years
healthy volunteers: No
last name: William D Arnold, MD
role: Principal Investigator
affiliation: Ohio State University
facility: 
country: United States
citation: Novak KR, Norman J, Mitchell JR, Pinter MJ, Rich MM. Sodium channel slow inactivation as a therapeutic target for myotonia congenita. Ann Neurol. 2015 Feb;77(2):320-32. doi: 10.1002/ana.24331. Epub 2015 Jan 9.
PMID: 25515836
responsible party type: Principal Investigator
investigator affiliation: Ohio State University
investigator full name: William Arnold
investigator title: Associate Professor
mesh term: Myotonic Dystrophy
mesh term: Myotonia Congenita
mesh term: Myotonic Disorders
mesh term: Myotonia
mesh term: Ranolazine
sharing ipd: Undecided