<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/NCT03254199</url>
  </required_header>
  <id_info>
    <org_study_id>FLX-787-204</org_study_id>
    <nct_id>NCT03254199</nct_id>
  </id_info>
  <brief_title>A Study to Assess the Safety and Effectiveness of FLX-787 in Subjects With Charcot-Marie-Tooth Disease Experiencing Muscle Cramps.</brief_title>
  <acronym>COMMIT</acronym>
  <official_title>A Randomized, Double-Blind, Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of FLX-787-ODT for Treatment of Muscle Cramps in Adult Subjects With Charcot-Marie-Tooth Disease</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Flex Pharma, Inc.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Flex Pharma, Inc.</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      The COMMIT Study will assess the safety and effectiveness of FLX-787 in men and women with&#xD;
      Charcot-Marie-Tooth disease (CMT) experiencing muscle cramps. Participants will be asked to&#xD;
      take two study products during the course of the study. One of these study products will be a&#xD;
      placebo.&#xD;
&#xD;
      Approximately 120 participants in 20 study centers across the United States are expected to&#xD;
      take part. Participants will be in the study for approximately 3 months and visit the study&#xD;
      clinic 3 times.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Terminated</overall_status>
  <why_stopped>
    Sponsor&#xD;
  </why_stopped>
  <start_date type="Actual">October 16, 2017</start_date>
  <completion_date type="Actual">July 27, 2018</completion_date>
  <primary_completion_date type="Actual">July 27, 2018</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <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>
    <masking_description>Double-blind</masking_description>
  </study_design_info>
  <primary_outcome>
    <measure>Cramp frequency</measure>
    <time_frame>28 days</time_frame>
    <description>Cramp frequency measured over the 28-day treatment period</description>
  </primary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">27</enrollment>
  <condition>Charcot-Marie-Tooth Disease</condition>
  <arm_group>
    <arm_group_label>Experimental</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <arm_group>
    <arm_group_label>Placebo Comparator</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>FLX-787-ODT (orally disintegrating tablet)</intervention_name>
    <description>FLX-787-ODT taken three times daily for 28 days</description>
    <arm_group_label>Experimental</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebo ODT</intervention_name>
    <description>Placebo ODT taken three times daily for 28 days</description>
    <arm_group_label>Placebo Comparator</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Presence of symptoms of CMT since at least 6 months prior to Screening, and confirmed&#xD;
             diagnosis of CMT as defined by:&#xD;
&#xD;
               1. Genetic confirmation of a mutation known to cause CMT, or&#xD;
&#xD;
               2. Clinical and electrophysiological evidence of CMT and a genetic confirmation in a&#xD;
                  family member. Clinical features include length dependent sensory and motor loss,&#xD;
                  with sensorimotor axonal or demyelinating changes on a nerve conduction study.&#xD;
&#xD;
          -  Weekly muscle cramping (defined as: a sustained muscle contraction that's most often&#xD;
             painful and lasts seconds to minutes)&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Presence of major gastrointestinal disorders, such as inflammatory bowel disease,&#xD;
             diverticulitis, active peptic ulcer disease, or significant gastroesophageal reflux&#xD;
             disease (i.e., not well-controlled on antacids or proton pump inhibitors), or oral or&#xD;
             esophageal lesions/ulcers&#xD;
&#xD;
          -  Presence of significant swallowing problems&#xD;
&#xD;
          -  Unable or unwilling to discontinue medications for cramps and/or opiates&#xD;
&#xD;
          -  Inability to tolerate a spicy sensation in the mouth or stomach&#xD;
&#xD;
          -  Actively using illicit drugs or history of chronic substance abuse within the past&#xD;
             year prior to screening, including abuse of alcohol&#xD;
&#xD;
          -  Intention to change the current level of tobacco use or use of nicotine-containing&#xD;
             products (i.e., new smokers or those actively trying to quit may not enrolled)&#xD;
&#xD;
          -  Participated in a clinical study (except natural history studies without&#xD;
             administration of an investigational product) within 30 days prior to screening&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Nicolas Johnson, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>University of Utah</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Mayo Clinic</name>
      <address>
        <city>Scottsdale</city>
        <state>Arizona</state>
        <zip>85259</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Colorado</name>
      <address>
        <city>Aurora</city>
        <state>Colorado</state>
        <zip>80045</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital for Special Care</name>
      <address>
        <city>New Britain</city>
        <state>Connecticut</state>
        <zip>06053</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Florida</name>
      <address>
        <city>Gainesville</city>
        <state>Florida</state>
        <zip>32607</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Mayo Clinic</name>
      <address>
        <city>Jacksonville</city>
        <state>Florida</state>
        <zip>32224</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>University of Iowa</name>
      <address>
        <city>Iowa City</city>
        <state>Iowa</state>
        <zip>52242</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Kansas Medical Center</name>
      <address>
        <city>Kansas City</city>
        <state>Kansas</state>
        <zip>66160</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Massachusetts General Hospital</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02114</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Brighman and Women's Hospital</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02115</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Minnesota</name>
      <address>
        <city>Minneapolis</city>
        <state>Minnesota</state>
        <zip>55455</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Saint Louis University</name>
      <address>
        <city>Saint Louis</city>
        <state>Missouri</state>
        <zip>63104</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Columbia University Medical Center</name>
      <address>
        <city>New York</city>
        <state>New York</state>
        <zip>10032</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Rochester Medical Center</name>
      <address>
        <city>Rochester</city>
        <state>New York</state>
        <zip>14642</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Oregon Health Science University</name>
      <address>
        <city>Portland</city>
        <state>Oregon</state>
        <zip>97239</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Penn State Milton S. Hershey Medical Center</name>
      <address>
        <city>Hershey</city>
        <state>Pennsylvania</state>
        <zip>17033</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Temple University</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <zip>19140</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The University of Utah</name>
      <address>
        <city>Salt Lake City</city>
        <state>Utah</state>
        <zip>84112</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Vermont Medical Center</name>
      <address>
        <city>Burlington</city>
        <state>Vermont</state>
        <zip>05405</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Saint Luke's Rehabilitation Institute</name>
      <address>
        <city>Spokane</city>
        <state>Washington</state>
        <zip>99202</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>August 2018</verification_date>
  <study_first_submitted>August 16, 2017</study_first_submitted>
  <study_first_submitted_qc>August 16, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">August 18, 2017</study_first_posted>
  <last_update_submitted>August 28, 2018</last_update_submitted>
  <last_update_submitted_qc>August 28, 2018</last_update_submitted_qc>
  <last_update_posted type="Actual">August 29, 2018</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Charcot-Marie-Tooth</keyword>
  <keyword>CMT</keyword>
  <keyword>Muscle Cramps</keyword>
  <keyword>Muscle Cramping</keyword>
  <keyword>FLX-787</keyword>
  <keyword>Hereditary Sensory and Motor Neuropathy</keyword>
  <keyword>Neurogenetic disorder</keyword>
  <keyword>Nervous System Malformations</keyword>
  <keyword>Nervous System Diseases</keyword>
  <keyword>Neurodegenerative Diseases</keyword>
  <keyword>Neuromuscular Diseases</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Muscle Cramp</mesh_term>
    <mesh_term>Tooth Diseases</mesh_term>
    <mesh_term>Charcot-Marie-Tooth Disease</mesh_term>
    <mesh_term>Nerve Compression Syndromes</mesh_term>
    <mesh_term>Hereditary Sensory and Motor Neuropathy</mesh_term>
    <mesh_term>Spasm</mesh_term>
  </condition_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/NCT03254199
org study id: FLX-787-204
nct id: NCT03254199
lead sponsor: 
has dmc: No
is fda regulated drug: Yes
is fda regulated device: No
The COMMIT Study will assess the safety and effectiveness of FLX-787 in men and women with
      Charcot-Marie-Tooth disease (CMT) experiencing muscle cramps. Participants will be asked to
      take two study products during the course of the study. One of these study products will be a
      placebo.

      Approximately 120 participants in 20 study centers across the United States are expected to
      take part. Participants will be in the study for approximately 3 months and visit the study
      clinic 3 times.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
masking description: Double-blind
measure: Cramp frequency
time frame: 28 days
description: Cramp frequency measured over the 28-day treatment period
arm group label: Experimental
arm group type: Experimental
arm group label: Placebo Comparator
arm group type: Placebo Comparator
intervention type: Drug
intervention name: FLX-787-ODT (orally disintegrating tablet)
description: FLX-787-ODT taken three times daily for 28 days
arm group label: Experimental
intervention type: Drug
intervention name: Placebo ODT
description: Placebo ODT taken three times daily for 28 days
arm group label: Placebo Comparator
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Nicolas Johnson, MD
role: Principal Investigator
affiliation: University of Utah
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country: United States
responsible party type: Sponsor
mesh term: Muscle Cramp
mesh term: Tooth Diseases
mesh term: Charcot-Marie-Tooth Disease
mesh term: Nerve Compression Syndromes
mesh term: Hereditary Sensory and Motor Neuropathy
mesh term: Spasm
sharing ipd: Undecided