<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/NCT03764150</url>
  </required_header>
  <id_info>
    <org_study_id>2009_04/0923</org_study_id>
    <secondary_id>2009-A00588-49</secondary_id>
    <nct_id>NCT03764150</nct_id>
  </id_info>
  <brief_title>Factors Associated With Hypoventilation in the Myotonic Dystrophy, Progressive Profile Over 5 Years</brief_title>
  <acronym>Steinert</acronym>
  <official_title>Factors Associated With Hypoventilation in the Myotonic Dystrophy, Progressive Profile Over 5 Years</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>University Hospital, Lille</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>University Hospital, Lille</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>
      The aim of this study is to determine the factors associated with alveolar hypoventilation in&#xD;
      terms of cognitive impairment, daytime sleepiness, respiratory function, nocturnal&#xD;
      respiratory events This evaluation will clarify the clinical phenotypes of respiratory&#xD;
      disease in myotonic steinert dystrophy.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Recruiting</last_known_status>
  <start_date type="Actual">June 30, 2010</start_date>
  <completion_date type="Anticipated">September 2023</completion_date>
  <primary_completion_date type="Anticipated">September 2023</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 of measurement of spirometry</measure>
    <time_frame>every year during 5 years</time_frame>
    <description>composite predictive factors of alveolar hypoventilation</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Change respiratory force</measure>
    <time_frame>every year during 5 years</time_frame>
    <description>composite predictive factors of alveolar hypoventilation to change Inspiratory and expiratory force</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Change CO2 response (Read test)</measure>
    <time_frame>every year during 5 years</time_frame>
    <description>composite predictive factors of alveolar hypoventilation</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Change subjective sleepiness (Epworth)</measure>
    <time_frame>every year during 5 years</time_frame>
    <description>composite predictive factors of alveolar hypoventilation</description>
  </primary_outcome>
  <primary_outcome>
    <measure>change Krupp's Fatigue Severity Scale (KFSS)</measure>
    <time_frame>every year during 5 years</time_frame>
    <description>composite predictive factors of alveolar hypoventilation This is a short questionnaire of 9 questions to which the patient responds on a Likert scale ranging from 1 to 7</description>
  </primary_outcome>
  <primary_outcome>
    <measure>change cognitive impairment by Trail Making Test A &amp; B</measure>
    <time_frame>every year during 5 years</time_frame>
    <description>composite predictive factors of alveolar hypoventilation Trail Making Test is a neuropsychological test of visual attention and task switching. It consists of two parts in which the subject is instructed to connect a set of 25 dots as quickly as possible while still maintaining accuracy.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>change Oximetry</measure>
    <time_frame>every year during 5 years</time_frame>
    <description>composite predictive factors of alveolar hypoventilation</description>
  </primary_outcome>
  <number_of_groups>2</number_of_groups>
  <enrollment type="Anticipated">160</enrollment>
  <condition>Myotonic Dystrophy, Steinert</condition>
  <arm_group>
    <arm_group_label>PaCO2&gt; 45 mmHg</arm_group_label>
    <description>Diurnal hypercapnia defined by PaCO2&gt; 45 mmHg</description>
  </arm_group>
  <arm_group>
    <arm_group_label>PaCO2 &lt;45 mmHg</arm_group_label>
    <description>PaCO2 diurnal within the limits of normal (PaCO2 &lt;45 mmHg)</description>
  </arm_group>
  <eligibility>
    <study_pop>
      <textblock>
        patient with a myotonic dystrophy of Steinert who consults in pneumology and who will have&#xD;
        Respiratory functional exploration&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Myotonic dystrophy of Steinert genetically confirmed (number of known CTG repeats), in&#xD;
             its classical form of adult&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Inability to perform EFR assessment or polysomnography&#xD;
&#xD;
          -  Moderate to severe asthma according to the GINA classification&#xD;
&#xD;
          -  moderate to severe COPD (&gt; stage 1 of the GOLD recommendations)&#xD;
&#xD;
          -  Other significant respiratory pathology&#xD;
&#xD;
          -  Patient already on NIV, CPAP or under oxygen therapy&#xD;
&#xD;
          -  Impossibility of receiving enlightened information&#xD;
&#xD;
          -  Inability to participate in the entire study&#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_official>
    <last_name>Thierry Perez, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>University Hospital, Lille</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Thierry Perez, MD</last_name>
    <email>thierry.perez@chru-lille.fr</email>
  </overall_contact>
  <location>
    <facility>
      <name>Centre Hospitalier Regional D' Angers</name>
      <address>
        <city>Angers</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
  </location>
  <location>
    <facility>
      <name>Ap-Hp Hu Paris Site Raymond Poincare</name>
      <address>
        <city>Garches</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
  </location>
  <location>
    <facility>
      <name>Hôpital Calmette, CHRU</name>
      <address>
        <city>Lille</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Thierry Perez, MD</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Chu Montpellier</name>
      <address>
        <city>Montpellier</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
  </location>
  <location>
    <facility>
      <name>Chu de Nantes :Site Hôtel-Dieu- Hme - Nantes 1</name>
      <address>
        <city>Nantes</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
  </location>
  <location>
    <facility>
      <name>Ap-Hp Hu Pitie Salpetriere</name>
      <address>
        <city>Paris</city>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
  </location>
  <location_countries>
    <country>France</country>
  </location_countries>
  <verification_date>January 2021</verification_date>
  <study_first_submitted>November 19, 2018</study_first_submitted>
  <study_first_submitted_qc>December 3, 2018</study_first_submitted_qc>
  <study_first_posted type="Actual">December 4, 2018</study_first_posted>
  <last_update_submitted>January 6, 2021</last_update_submitted>
  <last_update_submitted_qc>January 6, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">January 7, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Sleep Apnea Syndrome</keyword>
  <keyword>Muscular Diseases</keyword>
  <keyword>non invasive ventilation</keyword>
  <keyword>Diaphragmatic Disorder</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Myotonic Dystrophy</mesh_term>
    <mesh_term>Hypoventilation</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/NCT03764150
org study id: 2009_04/0923
secondary id: 2009-A00588-49
nct id: NCT03764150
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
The aim of this study is to determine the factors associated with alveolar hypoventilation in
      terms of cognitive impairment, daytime sleepiness, respiratory function, nocturnal
      respiratory events This evaluation will clarify the clinical phenotypes of respiratory
      disease in myotonic steinert dystrophy.
observational model: Cohort
time perspective: Prospective
measure: Change of measurement of spirometry
time frame: every year during 5 years
description: composite predictive factors of alveolar hypoventilation
measure: Change respiratory force
time frame: every year during 5 years
description: composite predictive factors of alveolar hypoventilation to change Inspiratory and expiratory force
measure: Change CO2 response (Read test)
time frame: every year during 5 years
description: composite predictive factors of alveolar hypoventilation
measure: Change subjective sleepiness (Epworth)
time frame: every year during 5 years
description: composite predictive factors of alveolar hypoventilation
measure: change Krupp's Fatigue Severity Scale (KFSS)
time frame: every year during 5 years
description: composite predictive factors of alveolar hypoventilation This is a short questionnaire of 9 questions to which the patient responds on a Likert scale ranging from 1 to 7
measure: change cognitive impairment by Trail Making Test A & B
time frame: every year during 5 years
description: composite predictive factors of alveolar hypoventilation Trail Making Test is a neuropsychological test of visual attention and task switching. It consists of two parts in which the subject is instructed to connect a set of 25 dots as quickly as possible while still maintaining accuracy.
measure: change Oximetry
time frame: every year during 5 years
description: composite predictive factors of alveolar hypoventilation
arm group label: PaCO2> 45 mmHg
description: Diurnal hypercapnia defined by PaCO2> 45 mmHg
arm group label: PaCO2 <45 mmHg
description: PaCO2 diurnal within the limits of normal (PaCO2 <45 mmHg)
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Thierry Perez, MD
role: Principal Investigator
affiliation: University Hospital, Lille
last name: Thierry Perez, MD
email: thierry.perez@chru-lille.fr
facility: 
status: Recruiting
facility: 
status: Recruiting
facility: 
status: Recruiting
investigator: 
facility: 
status: Recruiting
facility: 
status: Recruiting
facility: 
status: Recruiting
country: France
responsible party type: Sponsor
mesh term: Myotonic Dystrophy
mesh term: Hypoventilation
sharing ipd: No