<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/NCT04447196</url>
  </required_header>
  <id_info>
    <org_study_id>2020-A00215-34</org_study_id>
    <nct_id>NCT04447196</nct_id>
  </id_info>
  <brief_title>Prevalence of Rest Dyspnea and Impact of Non Invasive Ventilation on Breathing Sensations in CCHS Patients</brief_title>
  <acronym>DyspnOndine</acronym>
  <official_title>Prevalence of Rest Dyspnea and Impact of Non Invasive Ventilation on Breathing Sensations in Patients With Congenital Central Hypoventilation Syndrome</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Association pour le Développement et l'Organisation de la Recherche en Pneumologie et sur le Sommeil</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Association pour le Développement et l'Organisation de la Recherche en Pneumologie et sur le Sommeil</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>
      Congenital central hypoventilation syndrome (CCHS) is a neuro-respiratory disease&#xD;
      characterized by lifethreatening sleep-related hypoventilation involving an alteration of&#xD;
      CO2/H+ chemosensitivity. This suggests cortical activity during awakening to maintain&#xD;
      breathing. Cortical activity to keep breathing is usually associated with breathing&#xD;
      discomfort ; this is the case in healthy subjects under non invasive ventilation (NIV) or&#xD;
      with expiratory charge as well as in patients with amyotrophic lateral sclerosis. This can&#xD;
      suggest that CCHS may be breathless at rest and this discomfort could be reduced by NIV.&#xD;
&#xD;
      The objective is to evaluate dyspnea with a multi dimensional score, MDP, in CCHS patient at&#xD;
      rest in every day life and during 1H session of NIV.&#xD;
&#xD;
      The investigators will perform a prospective, including 20 CCHS patients. MDP scores will be&#xD;
      measure before and after 1H-non invasive ventilation as well as a visual scale of 100mm in&#xD;
      order to evaluate variation of breathing discomfort before/after NIV.&#xD;
&#xD;
      The investigators expect that CCHS patients don't have rest dyspnea but NIV would improve&#xD;
      breathing discomfort that would mean they have latent rest dyspnea.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Not yet recruiting</last_known_status>
  <start_date type="Anticipated">December 15, 2021</start_date>
  <completion_date type="Anticipated">October 1, 2022</completion_date>
  <primary_completion_date type="Anticipated">October 1, 2022</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>&quot;Immediate unpleasantness&quot; corresponding to A.1 item of the multidimensional dyspnea profile</measure>
    <time_frame>Change in MDP score between baseline and after 1 hour of NIV</time_frame>
    <description>Measure MDP scores before and after NIV</description>
  </primary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Anticipated">20</enrollment>
  <condition>CCHS</condition>
  <condition>Congenital Central Hypoventilation Syndrome</condition>
  <condition>Dyspnea</condition>
  <arm_group>
    <arm_group_label>CCHS</arm_group_label>
    <description>20 patients presenting with central hypoventilation syndrome during spontaneous breathing and with Non Invasive Ventilation</description>
  </arm_group>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>MDP</intervention_name>
    <description>Multi dimensional dyspnea profile scores at rest and during non invasive ventilation</description>
    <arm_group_label>CCHS</arm_group_label>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        Adults with CCHS who already require long terme NIV but not phrenic nerve stimulation&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  patients with CCHS confirmed by genetic test&#xD;
&#xD;
          -  patients with long term non invasive ventilation&#xD;
&#xD;
          -  no recent condition that could impact breathing sensations such as infection,&#xD;
             pulmonary embolism, heart failure&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  pregnancy&#xD;
&#xD;
          -  phrenic nerve stimulation&#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_contact>
    <last_name>MARLENE BRET</last_name>
    <phone>0698674449</phone>
    <email>marlene.bret@aphp.fr</email>
  </overall_contact>
  <location>
    <facility>
      <name>INSERM-UPC UMR_S 1158, Service de Pneumologie et Réanimation, GH Pitié-Salpêtrière</name>
      <address>
        <city>Paris</city>
        <zip>75013</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>France</country>
  </location_countries>
  <verification_date>July 2021</verification_date>
  <study_first_submitted>May 30, 2020</study_first_submitted>
  <study_first_submitted_qc>June 24, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 25, 2020</study_first_posted>
  <last_update_submitted>July 9, 2021</last_update_submitted>
  <last_update_submitted_qc>July 9, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">July 12, 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>Dyspnea</mesh_term>
    <mesh_term>Hypoventilation</mesh_term>
    <mesh_term>Sleep Apnea, Central</mesh_term>
    <mesh_term>Syndrome</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/NCT04447196
org study id: 2020-A00215-34
nct id: NCT04447196
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
Congenital central hypoventilation syndrome (CCHS) is a neuro-respiratory disease
      characterized by lifethreatening sleep-related hypoventilation involving an alteration of
      CO2/H+ chemosensitivity. This suggests cortical activity during awakening to maintain
      breathing. Cortical activity to keep breathing is usually associated with breathing
      discomfort ; this is the case in healthy subjects under non invasive ventilation (NIV) or
      with expiratory charge as well as in patients with amyotrophic lateral sclerosis. This can
      suggest that CCHS may be breathless at rest and this discomfort could be reduced by NIV.

      The objective is to evaluate dyspnea with a multi dimensional score, MDP, in CCHS patient at
      rest in every day life and during 1H session of NIV.

      The investigators will perform a prospective, including 20 CCHS patients. MDP scores will be
      measure before and after 1H-non invasive ventilation as well as a visual scale of 100mm in
      order to evaluate variation of breathing discomfort before/after NIV.

      The investigators expect that CCHS patients don't have rest dyspnea but NIV would improve
      breathing discomfort that would mean they have latent rest dyspnea.
observational model: Cohort
time perspective: Prospective
measure: "Immediate unpleasantness" corresponding to A.1 item of the multidimensional dyspnea profile
time frame: Change in MDP score between baseline and after 1 hour of NIV
description: Measure MDP scores before and after NIV
arm group label: CCHS
description: 20 patients presenting with central hypoventilation syndrome during spontaneous breathing and with Non Invasive Ventilation
intervention type: Other
intervention name: MDP
description: Multi dimensional dyspnea profile scores at rest and during non invasive ventilation
arm group label: CCHS
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: MARLENE BRET
phone: 0698674449
email: marlene.bret@aphp.fr
facility: 
country: France
responsible party type: Sponsor
mesh term: Dyspnea
mesh term: Hypoventilation
mesh term: Sleep Apnea, Central
mesh term: Syndrome