<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/NCT02199340</url>
  </required_header>
  <id_info>
    <org_study_id>11AR14</org_study_id>
    <nct_id>NCT02199340</nct_id>
  </id_info>
  <brief_title>The iStep Study: Development and Validation of an Incremental Exercise Step Test for Children With Cystic Fibrosis</brief_title>
  <official_title>The iStep Study: Development and Validation of an Incremental Exercise Step Test for Children With Cystic Fibrosis</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Great Ormond Street Hospital for Children NHS Foundation Trust</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Great Ormond Street Hospital for Children NHS Foundation Trust</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The progression of lung disease in cystic fibrosis (CF) results inevitably in a reduction in&#xD;
      exercise capacity. The assessment of fitness and exercise capacity in CF is an important&#xD;
      measure of the impact of the disease process, particularly if it is repeated over time. With&#xD;
      recent advances in clinical management, CF lung disease in children can be relatively mild&#xD;
      and exercise tolerance good. The currently available field tests e.g. 3 minute step test, are&#xD;
      often completed too easily. These tests provide limited information relating to maximal&#xD;
      exercise performance. By contrast, the maximal CardioPulmonary Exercise Test (CPET), a&#xD;
      progressive, incremental, gold standard exercise test with breath by breath analysis of&#xD;
      expired gas, has proved to be a valuable means of assessing exercise response in patients&#xD;
      with CF. Its only limitation is the requirement for specialist laboratory facilities,&#xD;
      equipment and staff.&#xD;
&#xD;
      A new field test for evaluating exercise capacity in children is needed. This should be&#xD;
      portable, easy to administer and simple to perform by young children, while providing a&#xD;
      higher intensity of exercise which correlates with day to day activity patterns of children,&#xD;
      and clinically relevant information in the short term and longitudinally. This test needs to&#xD;
      be a good surrogate measure of exercise capacity when formal CPET is unable to be undertaken.&#xD;
      By providing accurate and useful information the results can be used to prescribe and train&#xD;
      individuals with CF safely and effectively and can also be used in the short and long term&#xD;
      for guidance of the medical management of these complex patients.&#xD;
&#xD;
      The aim of this study is to develop and validate the use of a new incremental step test to&#xD;
      assess exercise tolerance/capacity in children with CF, compare this with the gold standard&#xD;
      CPET and to provide normative healthy control comparison data The main objectives of the&#xD;
      study are&#xD;
&#xD;
        1. To develop an incremental step test to assess exercise tolerance / capacity in children&#xD;
           with CF.&#xD;
&#xD;
        2. To compare the incremental step test with the gold standard CPET&#xD;
&#xD;
        3. To assess the level of exercise response produced by the incremental step test&#xD;
&#xD;
        4. To assess the correlation between independent variables of lung function measurements,&#xD;
           age, weight and height with VO2peak and other exercise test outcomes&#xD;
&#xD;
        5. To assess the repeatability and evaluate the normal variability of the new incremental&#xD;
           step test&#xD;
&#xD;
        6. To provide healthy control normative data for comparison&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date>July 2011</start_date>
  <completion_date type="Actual">October 2014</completion_date>
  <primary_completion_date type="Actual">October 2014</primary_completion_date>
  <phase>N/A</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Crossover Assignment</intervention_model>
    <primary_purpose>Basic Science</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Peak oxygen uptake (VO2peak)</measure>
    <time_frame>Measured during the exercise test, day 1, maximum of 20 minutes</time_frame>
    <description>Gold standard exercise test to determine peak oxygen uptake during exercise</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Secondary breath by breath analysis outcome measures</measure>
    <time_frame>Measured during the exercise test, day 1, maximum of 20 minutes</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Newly developed iStep exercise test outcome variables</measure>
    <time_frame>Measured during the exercise test, day 1, maximum of 20 minutes</time_frame>
    <description>Standard field exercise test outcomes of heart rate, oxygen saturations, time, level achieved, rate of perceived exertion, perceived breathlessness</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Height, weight and body mass index measures</measure>
    <time_frame>Measured at baseline on the day of testing, day 1, maximum of 5 minutes</time_frame>
    <description>Height, weight and body mass index will be measured for descriptive purposes only</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Reasons for termination of the incremental step test</measure>
    <time_frame>Measured at the end of the exercise test, day 1, maximum of 5 minutes</time_frame>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">24</enrollment>
  <condition>Cystic Fibrosis</condition>
  <arm_group>
    <arm_group_label>Cystic fibrosis</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>iStep exercise test&#xD;
CPET exercise test</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Healthy control</arm_group_label>
    <arm_group_type>Active Comparator</arm_group_type>
    <description>- iStep exercise test</description>
  </arm_group>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>iStep exercise test</intervention_name>
    <description>newly developed submaximal exercise test</description>
    <arm_group_label>Cystic fibrosis</arm_group_label>
    <arm_group_label>Healthy control</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>CPET exercise test</intervention_name>
    <description>maximal gold standard exercise test</description>
    <arm_group_label>Cystic fibrosis</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
        Clinical population inclusion&#xD;
&#xD;
          -  Parental/guardian consent&#xD;
&#xD;
          -  Participant assent&#xD;
&#xD;
          -  Male or female&#xD;
&#xD;
          -  Confirmed Cystic fibrosis diagnosis (by genetic genotype or positive sweat test)&#xD;
&#xD;
          -  Receiving physiotherapy as part of their usual care&#xD;
&#xD;
          -  Height &gt;125cm to allow cycle ergometer use&#xD;
&#xD;
          -  6-16 years&#xD;
&#xD;
        Healthy control population inclusion&#xD;
&#xD;
          -  Parental/guardian consent&#xD;
&#xD;
          -  Participant assent&#xD;
&#xD;
          -  Male or female&#xD;
&#xD;
          -  6-16 years&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
        Clinical population exclusion&#xD;
&#xD;
          -  &lt;6 years and &gt;16 years&#xD;
&#xD;
          -  No consent / assent&#xD;
&#xD;
          -  Frank haemoptysis in the last 48 hours&#xD;
&#xD;
          -  Acute infective exacerbation (defined as increased temperature, additional&#xD;
             antibiotics)&#xD;
&#xD;
          -  Poor dietary intake/hydration&#xD;
&#xD;
          -  Oxygen dependent at rest&#xD;
&#xD;
          -  Arterial oxygen saturation (SaO2) &lt;90% at rest&#xD;
&#xD;
          -  Burkholderia cepacia&#xD;
&#xD;
          -  Coexisting insulin dependent diabetes mellitus&#xD;
&#xD;
          -  Methicillin-resistant Staphylococcus aureus (MRSA) positive&#xD;
&#xD;
        Healthy control population exclusion&#xD;
&#xD;
          -  &lt;6 years and &gt;16 years&#xD;
&#xD;
          -  No consent / assent&#xD;
&#xD;
          -  Acute infection (defined as increased temperature and antibiotics)&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>6 Years</minimum_age>
    <maximum_age>16 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Sarah Rand, BA, BSc, MSc</last_name>
    <role>Principal Investigator</role>
    <affiliation>UCL Institute of Child Health</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Great Ormond Street Hospital for Children NHS Foundation Trust</name>
      <address>
        <city>London</city>
        <zip>WC1N 1EH</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United Kingdom</country>
  </location_countries>
  <verification_date>April 2016</verification_date>
  <study_first_submitted>June 12, 2014</study_first_submitted>
  <study_first_submitted_qc>July 22, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">July 24, 2014</study_first_posted>
  <last_update_submitted>April 18, 2016</last_update_submitted>
  <last_update_submitted_qc>April 18, 2016</last_update_submitted_qc>
  <last_update_posted type="Estimate">April 19, 2016</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Exercise testing</keyword>
  <keyword>Children</keyword>
  <keyword>Exercise capacity</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Cystic Fibrosis</mesh_term>
    <mesh_term>Fibrosis</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/NCT02199340
org study id: 11AR14
nct id: NCT02199340
lead sponsor: 
has dmc: Yes
The progression of lung disease in cystic fibrosis (CF) results inevitably in a reduction in
      exercise capacity. The assessment of fitness and exercise capacity in CF is an important
      measure of the impact of the disease process, particularly if it is repeated over time. With
      recent advances in clinical management, CF lung disease in children can be relatively mild
      and exercise tolerance good. The currently available field tests e.g. 3 minute step test, are
      often completed too easily. These tests provide limited information relating to maximal
      exercise performance. By contrast, the maximal CardioPulmonary Exercise Test (CPET), a
      progressive, incremental, gold standard exercise test with breath by breath analysis of
      expired gas, has proved to be a valuable means of assessing exercise response in patients
      with CF. Its only limitation is the requirement for specialist laboratory facilities,
      equipment and staff.

      A new field test for evaluating exercise capacity in children is needed. This should be
      portable, easy to administer and simple to perform by young children, while providing a
      higher intensity of exercise which correlates with day to day activity patterns of children,
      and clinically relevant information in the short term and longitudinally. This test needs to
      be a good surrogate measure of exercise capacity when formal CPET is unable to be undertaken.
      By providing accurate and useful information the results can be used to prescribe and train
      individuals with CF safely and effectively and can also be used in the short and long term
      for guidance of the medical management of these complex patients.

      The aim of this study is to develop and validate the use of a new incremental step test to
      assess exercise tolerance/capacity in children with CF, compare this with the gold standard
      CPET and to provide normative healthy control comparison data The main objectives of the
      study are

        1. To develop an incremental step test to assess exercise tolerance / capacity in children
           with CF.

        2. To compare the incremental step test with the gold standard CPET

        3. To assess the level of exercise response produced by the incremental step test

        4. To assess the correlation between independent variables of lung function measurements,
           age, weight and height with VO2peak and other exercise test outcomes

        5. To assess the repeatability and evaluate the normal variability of the new incremental
           step test

        6. To provide healthy control normative data for comparison
allocation: Randomized
intervention model: Crossover Assignment
primary purpose: Basic Science
masking: None (Open Label)
measure: Peak oxygen uptake (VO2peak)
time frame: Measured during the exercise test, day 1, maximum of 20 minutes
description: Gold standard exercise test to determine peak oxygen uptake during exercise
measure: Secondary breath by breath analysis outcome measures
time frame: Measured during the exercise test, day 1, maximum of 20 minutes
measure: Newly developed iStep exercise test outcome variables
time frame: Measured during the exercise test, day 1, maximum of 20 minutes
description: Standard field exercise test outcomes of heart rate, oxygen saturations, time, level achieved, rate of perceived exertion, perceived breathlessness
measure: Height, weight and body mass index measures
time frame: Measured at baseline on the day of testing, day 1, maximum of 5 minutes
description: Height, weight and body mass index will be measured for descriptive purposes only
measure: Reasons for termination of the incremental step test
time frame: Measured at the end of the exercise test, day 1, maximum of 5 minutes
arm group label: Cystic fibrosis
arm group type: Experimental
description: iStep exercise test
CPET exercise test
arm group label: Healthy control
arm group type: Active Comparator
description: - iStep exercise test
intervention type: Other
intervention name: iStep exercise test
description: newly developed submaximal exercise test
arm group label: Cystic fibrosis
arm group label: Healthy control
intervention type: Other
intervention name: CPET exercise test
description: maximal gold standard exercise test
arm group label: Cystic fibrosis
criteria: 
gender: All
minimum age: 6 Years
maximum age: 16 Years
healthy volunteers: No
last name: Sarah Rand, BA, BSc, MSc
role: Principal Investigator
affiliation: UCL Institute of Child Health
facility: 
country: United Kingdom
responsible party type: Sponsor
mesh term: Cystic Fibrosis
mesh term: Fibrosis
sharing ipd: Undecided