<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/NCT03255213</url>
  </required_header>
  <id_info>
    <org_study_id>Pro00077344</org_study_id>
    <nct_id>NCT03255213</nct_id>
  </id_info>
  <brief_title>Lingual Muscle Training in Late-Onset Pompe Disease (LOPD)</brief_title>
  <official_title>Lingual Muscle Training in Late-Onset Pompe Disease (LOPD)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Duke University</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Duke University</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      This study is being done to test the effects of Lingual Muscle Therapy (LMT) in patients with&#xD;
      Late-Onset Pompe Disease (LOPD) who have tongue weakness. The results of this study will help&#xD;
      design future research studies about LMT in LOPD.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">March 21, 2018</start_date>
  <completion_date type="Actual">April 23, 2020</completion_date>
  <primary_completion_date type="Actual">April 23, 2020</primary_completion_date>
  <phase>N/A</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>Safety of lingual muscle training</measure>
    <time_frame>13 weeks</time_frame>
    <description>Safety measured by pain rating scale</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Feasibility of lingual muscle training as measured by questionnaires</measure>
    <time_frame>13 weeks</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>Change in lingual strength</measure>
    <time_frame>baseline, 13 weeks</time_frame>
    <description>Change from pre-test to post-test</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">5</enrollment>
  <condition>Glycogen Storage Disease</condition>
  <arm_group>
    <arm_group_label>Late Onset Pompe Disease who have tongue weakness</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Lingual Muscle Training</intervention_name>
    <description>Lingual Muscle Training using the Iowa Oral Performance Instrument (OIPI) device</description>
    <arm_group_label>Late Onset Pompe Disease who have tongue weakness</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion criteria:&#xD;
&#xD;
          -  Age ≥ 12 years&#xD;
&#xD;
          -  Lingual weakness as measured by quantitative muscle testing (≤ 5% lower limit of&#xD;
             normal for stratified age)&#xD;
&#xD;
          -  Confirmed diagnosis of LOPD&#xD;
&#xD;
          -  On enzyme replacement therapy for ≥ 26 weeks at pretest&#xD;
&#xD;
          -  Able to follow directions for study participation&#xD;
&#xD;
          -  Able to complete a home-based LMT regimen&#xD;
&#xD;
          -  Access to reliable internet connection&#xD;
&#xD;
          -  Access to use of electronic device that allows for FaceTime, Jabber, or WebEx use&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Neurodegenerative conditions (e.g. stroke, dementia) or other serious neurologic&#xD;
             condition that would prevent meaningful study participation as determined at the&#xD;
             discretion of the principle investigator&#xD;
&#xD;
          -  Any current or past history of seizures.&#xD;
&#xD;
          -  Head and neck cancer or radiation treatment to head/neck&#xD;
&#xD;
          -  Inability to tolerate IOPI device tongue bulb&#xD;
&#xD;
          -  Inability to give legally effective consent&#xD;
&#xD;
          -  Inability to read and understand English&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>12 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Jones Harrison, PhD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Duke University</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Duke University Medical Center and affiliated practices</name>
      <address>
        <city>Durham</city>
        <state>North Carolina</state>
        <zip>27710</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>July 2021</verification_date>
  <study_first_submitted>August 3, 2017</study_first_submitted>
  <study_first_submitted_qc>August 17, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">August 21, 2017</study_first_posted>
  <last_update_submitted>July 27, 2021</last_update_submitted>
  <last_update_submitted_qc>July 27, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">July 28, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Glycogen Storage Disease Type II</mesh_term>
    <mesh_term>Glycogen Storage Disease</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</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/NCT03255213
org study id: Pro00077344
nct id: NCT03255213
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
This study is being done to test the effects of Lingual Muscle Therapy (LMT) in patients with
      Late-Onset Pompe Disease (LOPD) who have tongue weakness. The results of this study will help
      design future research studies about LMT in LOPD.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Safety of lingual muscle training
time frame: 13 weeks
description: Safety measured by pain rating scale
measure: Feasibility of lingual muscle training as measured by questionnaires
time frame: 13 weeks
measure: Change in lingual strength
time frame: baseline, 13 weeks
description: Change from pre-test to post-test
arm group label: Late Onset Pompe Disease who have tongue weakness
arm group type: Experimental
intervention type: Other
intervention name: Lingual Muscle Training
description: Lingual Muscle Training using the Iowa Oral Performance Instrument (OIPI) device
arm group label: Late Onset Pompe Disease who have tongue weakness
criteria: 
gender: All
minimum age: 12 Years
maximum age: N/A
healthy volunteers: No
last name: Jones Harrison, PhD
role: Principal Investigator
affiliation: Duke University
facility: 
country: United States
responsible party type: Sponsor
mesh term: Glycogen Storage Disease Type II
mesh term: Glycogen Storage Disease
sharing ipd: No