<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/NCT01701154</url>
  </required_header>
  <id_info>
    <org_study_id>Pro00024468</org_study_id>
    <nct_id>NCT01701154</nct_id>
  </id_info>
  <brief_title>The Effects of Respiratory Muscle Strength Training (RMST) on Inspiratory and Expiratory Muscle Strength in Adults and Children With Pompe Disease</brief_title>
  <official_title>The Effects of Respiratory Muscle Strength Training (RMST) on Inspiratory and Expiratory Muscle Strength in Adults and Children With Pompe Disease</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>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of the study is to determine if respiratory muscle strength training will be&#xD;
      beneficial for inspiratory and expiratory muscle strength in adults and children with Pompe&#xD;
      disease.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Respiratory muscle weakness results in substantial morbidity and mortality in individuals&#xD;
      with almost all forms of neuromuscular disease (NMD), including both the infantile and adult&#xD;
      phenotypes of Pompe disease. Although individual patterns of involvement vary, respiratory&#xD;
      weakness in Pompe disease typically affects both the inspiratory and expiratory muscle&#xD;
      systems. Our pilot data in two individuals with late-onset Pompe disease suggest that RMST&#xD;
      may be a treatment for the progressive respiratory weakness encountered in this condition.&#xD;
&#xD;
      Purpose of the Study&#xD;
&#xD;
        -  Determine the effect of respiratory muscle strength training on maximum inspiratory&#xD;
           pressure, maximum expiratory pressure, upright % predicted forced vital capacity, and&#xD;
           aspects of cough function.&#xD;
&#xD;
        -  Determine the effect of RMST on functional outcome measures of gross motor function&#xD;
           appropriate for individual participants in terms of age and motor skills.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date>December 2010</start_date>
  <completion_date type="Actual">December 15, 2012</completion_date>
  <primary_completion_date type="Actual">December 15, 2012</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Cohort</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Change in maximum inspiratory pressure</measure>
    <time_frame>baseline, week 12</time_frame>
    <description>change from pre-test to post-test</description>
  </primary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Actual">11</enrollment>
  <condition>Pompe Disease</condition>
  <arm_group>
    <arm_group_label>Pompe</arm_group_label>
    <description>Adults and children with Pompe disease.</description>
  </arm_group>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>respiratory muscle strength training</intervention_name>
    <arm_group_label>Pompe</arm_group_label>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        Adults and children with Pompe disease&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  include skin fibroblast acid α-glucosidase (GAA) activity ≤1% of the normal mean, a&#xD;
             minimum age of 3 years, the ability to participate in an intensive RMST research&#xD;
             program, and the ability to maintain a consistent amount of nonresearch related&#xD;
             physical activity over the course of the study.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  include medical problems which preclude meaningful participation in the study, the&#xD;
             inability to perform high effort respiratory tasks using maximum intensity,&#xD;
             respiratory weakness so profound that RMST cannot be completed at the minimum pressure&#xD;
             thresholds of available respiratory trainers, and the inability to safely perform the&#xD;
             protocol. For example, high effort respiratory tasks are occasionally associated with&#xD;
             mild, transient dizziness which quickly dissipates following a brief rest. If severe&#xD;
             and/or prolonged dizziness were to occur in a particular case, then exclusion from the&#xD;
             study would be necessary.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>3 Years</minimum_age>
    <maximum_age>99 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Harrison Jones, PhD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Duke University</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Duke University Health System</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>May 2023</verification_date>
  <study_first_submitted>October 3, 2012</study_first_submitted>
  <study_first_submitted_qc>October 3, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">October 4, 2012</study_first_posted>
  <last_update_submitted>May 25, 2023</last_update_submitted>
  <last_update_submitted_qc>May 25, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">May 30, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Pompe respiratory</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Glycogen Storage Disease Type II</mesh_term>
  </condition_browse>
  <!-- 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/NCT01701154
org study id: Pro00024468
nct id: NCT01701154
lead sponsor: 
has dmc: Yes
The purpose of the study is to determine if respiratory muscle strength training will be
      beneficial for inspiratory and expiratory muscle strength in adults and children with Pompe
      disease.
Respiratory muscle weakness results in substantial morbidity and mortality in individuals
      with almost all forms of neuromuscular disease (NMD), including both the infantile and adult
      phenotypes of Pompe disease. Although individual patterns of involvement vary, respiratory
      weakness in Pompe disease typically affects both the inspiratory and expiratory muscle
      systems. Our pilot data in two individuals with late-onset Pompe disease suggest that RMST
      may be a treatment for the progressive respiratory weakness encountered in this condition.

      Purpose of the Study

        -  Determine the effect of respiratory muscle strength training on maximum inspiratory
           pressure, maximum expiratory pressure, upright % predicted forced vital capacity, and
           aspects of cough function.

        -  Determine the effect of RMST on functional outcome measures of gross motor function
           appropriate for individual participants in terms of age and motor skills.
observational model: Cohort
time perspective: Prospective
measure: Change in maximum inspiratory pressure
time frame: baseline, week 12
description: change from pre-test to post-test
arm group label: Pompe
description: Adults and children with Pompe disease.
intervention type: Other
intervention name: respiratory muscle strength training
arm group label: Pompe
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 3 Years
maximum age: 99 Years
healthy volunteers: No
last name: Harrison Jones, PhD
role: Principal Investigator
affiliation: Duke University
facility: 
country: United States
responsible party type: Sponsor
mesh term: Glycogen Storage Disease Type II