<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/NCT03292718</url>
  </required_header>
  <id_info>
    <org_study_id>s60787</org_study_id>
    <nct_id>NCT03292718</nct_id>
  </id_info>
  <brief_title>Clinical Trial to Assess Influence of MyCyFAPP Use on GI Related QOL in Children With Cystic Fibrosis</brief_title>
  <acronym>MyCyFAPP</acronym>
  <official_title>Innovative Approach for Self-management and Social Welfare of Cystic Fibrosis Patients in Europe: Development, Validation and Implementation of a Telematics Tool. WP6.2: Impact Assessment Through a European Multicentre Clinical Trial: Validation of MyCyFAPP as a Portable System for Self-management in Children With CF</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Universitaire Ziekenhuizen KU Leuven</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Hospital Universitario La Fe</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>European Commission</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Universitaire Ziekenhuizen KU Leuven</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>
      Interventional trial to study the influence of the use of MyCyFAPP (mobile application) on&#xD;
      the gastro-intestinal related quality of life.&#xD;
&#xD;
      This mobile APP has been developed during previous workpackages of the Horizon2020 Project&#xD;
      and contains several modules:&#xD;
&#xD;
        -  mathematical prediction model to calculate the needed dose for pancreatic enzyme&#xD;
           replacement therapy&#xD;
&#xD;
        -  educational games and other educational material&#xD;
&#xD;
        -  communication with doctor/dietician through professional webtool&#xD;
&#xD;
        -  diary to register symptoms and data on nutrition.&#xD;
&#xD;
      The app will be introduced and used during 6 months. Primary outcome parameter will be change&#xD;
      in modified PedsQL GI after 3 months. PedsQL GI is an existing questionnaire that evaluates&#xD;
      gastro-intestinal related quality of life in children. We validated it for use in cystic&#xD;
      fibrosis in a previous observational study.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Not yet recruiting</last_known_status>
  <start_date type="Anticipated">October 2017</start_date>
  <completion_date type="Anticipated">December 2018</completion_date>
  <primary_completion_date type="Anticipated">December 2018</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>
    <intervention_model_description>Single group, open label, with intervention</intervention_model_description>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>change in Modified PedsQL GI</measure>
    <time_frame>3 months</time_frame>
    <description>Modified PedsQL GI will be assessed at month 0 and month 3 by applying questionnaires to children and their parents</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>change in CFQ-R</measure>
    <time_frame>3 months and 6 months</time_frame>
    <description>CFQ-R will be assessed by questionnaires</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>change in VAS</measure>
    <time_frame>3 months and 6 months</time_frame>
    <description>VAS will be assessed by questionnaires</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>change in Modified PedsQL GI</measure>
    <time_frame>6 months</time_frame>
    <description>Modified PedsQL GI will be assessed at month 0 and month 6 by applying questionnaires to children and their parents</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>change in lung function</measure>
    <time_frame>3 and 6 months</time_frame>
    <description>spirometry will be performed</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Anticipated">200</enrollment>
  <condition>Cystic Fibrosis in Children</condition>
  <arm_group>
    <arm_group_label>use of MyCyFAPP</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>use of MyCyFAPP during 6 months</description>
  </arm_group>
  <intervention>
    <intervention_type>Device</intervention_type>
    <intervention_name>MyCyFAPP</intervention_name>
    <description>use of the MycyFAPP with all its features during 6 months</description>
    <arm_group_label>use of MyCyFAPP</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Diagnosis of CF as evidenced by one or more clinical feature consistent with the CF&#xD;
             phenotype or positive CF newborn screen AND one or more of the following criteria:&#xD;
&#xD;
               1. A documented sweat chloride ≥ 60 mEq/L by quantitative pilocarpine iontophoresis&#xD;
                  (QPIT)&#xD;
&#xD;
               2. A documented genotype with two disease-causing mutations in the CFTR gene&#xD;
&#xD;
          2. Having pancreatic insufficiency (stool elastase &lt; 200 mcg/g stool) and using PERT&#xD;
&#xD;
          3. Age ≥ 24 months and &lt; 18 years at screening visit&#xD;
&#xD;
          4. Informed consent by parent or legal guardian; assent for children from age 12 years on&#xD;
&#xD;
        6. Inclusion visit coincides with scheduled routine clinic visit 7. Ability and willingness&#xD;
        to comply with APP use and evaluations at time of routine clinic visits as judged by the&#xD;
        site investigator 8. Availability of wifi connection at home so that connection to the&#xD;
        internet is feasible at home at least weekly.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Acute infection associated with decreased appetite or fever at time of run-in visit&#xD;
&#xD;
          2. Acute abdominal pain necessitating an intervention at time of run-in visit&#xD;
&#xD;
          3. Physical findings that would compromise the safety of the participant or the quality&#xD;
             of the study data as determined by site investigator&#xD;
&#xD;
          4. Investigational drug use within 30 days prior to run-in visit&#xD;
&#xD;
          5. Started with CFTR modulator treatment less than 3 months before start of run-in visit&#xD;
&#xD;
          6. Inability to use the APP due to patient specific factors such as language or learning&#xD;
             difficulties&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>2 Years</minimum_age>
    <maximum_age>18 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_contact>
    <last_name>Mieke Boon, MD PhD</last_name>
    <phone>+3216343820</phone>
    <email>mieke.boon@uzleuven.be</email>
  </overall_contact>
  <link>
    <url>http://www.mycyfapp.eu/en/</url>
    <description>Related Info</description>
  </link>
  <verification_date>September 2017</verification_date>
  <study_first_submitted>September 20, 2017</study_first_submitted>
  <study_first_submitted_qc>September 20, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">September 25, 2017</study_first_posted>
  <last_update_submitted>September 20, 2017</last_update_submitted>
  <last_update_submitted_qc>September 20, 2017</last_update_submitted_qc>
  <last_update_posted type="Actual">September 25, 2017</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Universitaire Ziekenhuizen KU Leuven</investigator_affiliation>
    <investigator_full_name>Mieke Boon, MD PhD</investigator_full_name>
    <investigator_title>Mieke Boon</investigator_title>
  </responsible_party>
  <keyword>PERT</keyword>
  <keyword>mobile application</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>
  <!-- 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/NCT03292718
org study id: s60787
nct id: NCT03292718
lead sponsor: 
collaborator: 
collaborator: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
Interventional trial to study the influence of the use of MyCyFAPP (mobile application) on
      the gastro-intestinal related quality of life.

      This mobile APP has been developed during previous workpackages of the Horizon2020 Project
      and contains several modules:

        -  mathematical prediction model to calculate the needed dose for pancreatic enzyme
           replacement therapy

        -  educational games and other educational material

        -  communication with doctor/dietician through professional webtool

        -  diary to register symptoms and data on nutrition.

      The app will be introduced and used during 6 months. Primary outcome parameter will be change
      in modified PedsQL GI after 3 months. PedsQL GI is an existing questionnaire that evaluates
      gastro-intestinal related quality of life in children. We validated it for use in cystic
      fibrosis in a previous observational study.
allocation: N/A
intervention model: Single Group Assignment
intervention model description: Single group, open label, with intervention
primary purpose: Treatment
masking: None (Open Label)
measure: change in Modified PedsQL GI
time frame: 3 months
description: Modified PedsQL GI will be assessed at month 0 and month 3 by applying questionnaires to children and their parents
measure: change in CFQ-R
time frame: 3 months and 6 months
description: CFQ-R will be assessed by questionnaires
measure: change in VAS
time frame: 3 months and 6 months
description: VAS will be assessed by questionnaires
measure: change in Modified PedsQL GI
time frame: 6 months
description: Modified PedsQL GI will be assessed at month 0 and month 6 by applying questionnaires to children and their parents
measure: change in lung function
time frame: 3 and 6 months
description: spirometry will be performed
arm group label: use of MyCyFAPP
arm group type: Experimental
description: use of MyCyFAPP during 6 months
intervention type: Device
intervention name: MyCyFAPP
description: use of the MycyFAPP with all its features during 6 months
arm group label: use of MyCyFAPP
criteria: 
gender: All
minimum age: 2 Years
maximum age: 18 Years
healthy volunteers: No
last name: Mieke Boon, MD PhD
phone: +3216343820
email: mieke.boon@uzleuven.be
url: http://www.mycyfapp.eu/en/
description: Related Info
responsible party type: Principal Investigator
investigator affiliation: Universitaire Ziekenhuizen KU Leuven
investigator full name: Mieke Boon, MD PhD
investigator title: Mieke Boon
mesh term: Cystic Fibrosis
mesh term: Fibrosis