<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/NCT04491747</url>
  </required_header>
  <id_info>
    <org_study_id>GO 19/97</org_study_id>
    <nct_id>NCT04491747</nct_id>
  </id_info>
  <brief_title>Assessment of Factors That Affected Respiratory Parameters in Mucopolysaccharidoses Patients</brief_title>
  <official_title>Assessment of Factors That Affected Respiratory Parameters in Mucopolysaccharidoses Patients</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Hacettepe University</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Hacettepe University</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>
      Respiratory system affects in mucopolysaccharidosis (MPS) disease. Respiratory system&#xD;
      symptoms are seen in almost every MPS patients and respiratory failure is one of the most&#xD;
      common causes of death in this population. The aim of the study was to evaluate respiratory&#xD;
      muscle strength and endurance, cough effectiveness and functional capacity in MPS patients&#xD;
      and to defined the factors that affected the respiratory problem in this.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This study was participated MPS patients. Patients' pulmonary function test, functional&#xD;
      capacity, respiratory muscle strength and endurance, peak cough flow was assessed and&#xD;
      recorded.&#xD;
&#xD;
      Pulmonary function test was assessed with spirometry, functional capacity was evaluated with&#xD;
      3-minute step test and six minute walk test (MWT). Respiratory muscle strength was evaluated&#xD;
      mouth pressure measurement (MIP- maximal inspiratory pressure and MEP- maximal expiratory&#xD;
      pressure) and Sniff nasal inspiratory pressure (SNIP). Respiratory muscle endurance was&#xD;
      assessed Powerbreathe. And cough effectiveness was evaluated peak cough flow.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">February 1, 2019</start_date>
  <completion_date type="Actual">February 1, 2020</completion_date>
  <primary_completion_date type="Actual">February 1, 2020</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>functional capacity</measure>
    <time_frame>15 minutes</time_frame>
    <description>Six minute walk test: Distance walked in six minute was recorded</description>
  </primary_outcome>
  <primary_outcome>
    <measure>3 minute step test</measure>
    <time_frame>5 minutes</time_frame>
    <description>number of completed step in 3 minute was recorded</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Respiratory muscle strength assessment</measure>
    <time_frame>15 minutes</time_frame>
    <description>Respiratory muscle strength was evaluated by measuring maximal inspiratory pressure (MIP) and maximal expiratory pressure (MEP) and sniff nasal inspiratory pressure(SNIP). Also dynamic inspiratory muscle strength was evaluated Powerbreathe K5 device.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Respiratory muscle endurance</measure>
    <time_frame>10 minutes</time_frame>
    <description>Pressure time unit: A constant load test was used to evaluate respiratory muscle endurance. Using a PowerBreathe device (HaB International Ltd., Southam, UK), the participants were asked to breathe maintaining a constant threshold (60% of MIP) until task failure, and the test duration was recorded. Results were obtained by multiplying the set pressure by the test duration</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Pulmonary functions</measure>
    <time_frame>15 minutes</time_frame>
    <description>FVC (Forced vital capacity) assessment via spirometry</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Pulmonary functions</measure>
    <time_frame>15 minutes</time_frame>
    <description>FEV 1(forced expiratory volume in first second) assessment via spirometry</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Pulmonary functions</measure>
    <time_frame>15 minutes</time_frame>
    <description>PEF (peak expiratory flow) assessment via spirometry</description>
  </primary_outcome>
  <primary_outcome>
    <measure>MVV (Maximal ventilatory assesssment)</measure>
    <time_frame>5 minutes</time_frame>
    <description>Maximize ventilation by inhaling and exhaling as quickly and deeply as possible for 15 s via spirometry</description>
  </primary_outcome>
  <primary_outcome>
    <measure>cough effectiveness</measure>
    <time_frame>5 minutes</time_frame>
    <description>cough effectiveness assessment was evaluated peak cough flow meter.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>heart rate</measure>
    <time_frame>6 minutes</time_frame>
    <description>difference pre and post test heart beat number</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>oxygen saturation</measure>
    <time_frame>6 minutes</time_frame>
    <description>difference pre and post test percentage of oxygen saturation</description>
  </secondary_outcome>
  <enrollment type="Actual">24</enrollment>
  <condition>Mucopolysaccharidoses</condition>
  <intervention>
    <intervention_type>Diagnostic Test</intervention_type>
    <intervention_name>functional capacity</intervention_name>
    <description>Six minute walk test used assessment of functional capacity.</description>
    <other_name>Exercise capacity</other_name>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        People who diagnosed Mucopolysaccharidoses disease in Hacettepe University were recruited&#xD;
        the study.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  People who diagnosed Mucopolysaccharidoses disease in Hacettepe University.&#xD;
&#xD;
          -  Cooperation&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Patients who were not walking ability.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>7 Years</minimum_age>
    <maximum_age>30 Years</maximum_age>
  </eligibility>
  <overall_official>
    <last_name>Deniz Dogru- Ersoz, Professor</last_name>
    <role>Study Director</role>
    <affiliation>Hacettepe University</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Naciye Vardar- Yagli, PhD</last_name>
    <role>Study Chair</role>
    <affiliation>Hacettepe University</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Dilber Ademhan- Tural, MD</last_name>
    <role>Study Chair</role>
    <affiliation>Hacettepe University</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Nural Kiper, Professor</last_name>
    <role>Study Chair</role>
    <affiliation>Hacettepe University</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Ugur Ozcelik, Professor</last_name>
    <role>Study Chair</role>
    <affiliation>Hacettepe University</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Ebru Yalcin, Professor</last_name>
    <role>Study Chair</role>
    <affiliation>Hacettepe University</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Deniz Inal-Ince, Professor</last_name>
    <role>Study Chair</role>
    <affiliation>Hacettepe University</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Melda Saglam, PhD</last_name>
    <role>Study Chair</role>
    <affiliation>Hacettepe University</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Serap Sivri, Professor</last_name>
    <role>Study Chair</role>
    <affiliation>Hacettepe University</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Aysegul Tokatlı, Professor</last_name>
    <role>Study Chair</role>
    <affiliation>Hacettepe University</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Hacettepe University</name>
      <address>
        <city>Ankara</city>
        <country>Turkey</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Turkey</country>
  </location_countries>
  <verification_date>July 2020</verification_date>
  <study_first_submitted>July 19, 2020</study_first_submitted>
  <study_first_submitted_qc>July 27, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">July 29, 2020</study_first_posted>
  <last_update_submitted>July 27, 2020</last_update_submitted>
  <last_update_submitted_qc>July 27, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">July 29, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Hacettepe University</investigator_affiliation>
    <investigator_full_name>Kübra Kılıç</investigator_full_name>
    <investigator_title>Physiotherapist</investigator_title>
  </responsible_party>
  <keyword>Respiratory muscles</keyword>
  <keyword>exercise test</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Mucopolysaccharidoses</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/NCT04491747
org study id: GO 19/97
nct id: NCT04491747
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
Respiratory system affects in mucopolysaccharidosis (MPS) disease. Respiratory system
      symptoms are seen in almost every MPS patients and respiratory failure is one of the most
      common causes of death in this population. The aim of the study was to evaluate respiratory
      muscle strength and endurance, cough effectiveness and functional capacity in MPS patients
      and to defined the factors that affected the respiratory problem in this.
This study was participated MPS patients. Patients' pulmonary function test, functional
      capacity, respiratory muscle strength and endurance, peak cough flow was assessed and
      recorded.

      Pulmonary function test was assessed with spirometry, functional capacity was evaluated with
      3-minute step test and six minute walk test (MWT). Respiratory muscle strength was evaluated
      mouth pressure measurement (MIP- maximal inspiratory pressure and MEP- maximal expiratory
      pressure) and Sniff nasal inspiratory pressure (SNIP). Respiratory muscle endurance was
      assessed Powerbreathe. And cough effectiveness was evaluated peak cough flow.
observational model: Cohort
time perspective: Prospective
measure: functional capacity
time frame: 15 minutes
description: Six minute walk test: Distance walked in six minute was recorded
measure: 3 minute step test
time frame: 5 minutes
description: number of completed step in 3 minute was recorded
measure: Respiratory muscle strength assessment
time frame: 15 minutes
description: Respiratory muscle strength was evaluated by measuring maximal inspiratory pressure (MIP) and maximal expiratory pressure (MEP) and sniff nasal inspiratory pressure(SNIP). Also dynamic inspiratory muscle strength was evaluated Powerbreathe K5 device.
measure: Respiratory muscle endurance
time frame: 10 minutes
description: Pressure time unit: A constant load test was used to evaluate respiratory muscle endurance. Using a PowerBreathe device (HaB International Ltd., Southam, UK), the participants were asked to breathe maintaining a constant threshold (60% of MIP) until task failure, and the test duration was recorded. Results were obtained by multiplying the set pressure by the test duration
measure: Pulmonary functions
time frame: 15 minutes
description: FVC (Forced vital capacity) assessment via spirometry
measure: Pulmonary functions
time frame: 15 minutes
description: FEV 1(forced expiratory volume in first second) assessment via spirometry
measure: Pulmonary functions
time frame: 15 minutes
description: PEF (peak expiratory flow) assessment via spirometry
measure: MVV (Maximal ventilatory assesssment)
time frame: 5 minutes
description: Maximize ventilation by inhaling and exhaling as quickly and deeply as possible for 15 s via spirometry
measure: cough effectiveness
time frame: 5 minutes
description: cough effectiveness assessment was evaluated peak cough flow meter.
measure: heart rate
time frame: 6 minutes
description: difference pre and post test heart beat number
measure: oxygen saturation
time frame: 6 minutes
description: difference pre and post test percentage of oxygen saturation
intervention type: Diagnostic Test
intervention name: functional capacity
description: Six minute walk test used assessment of functional capacity.
other name: Exercise capacity
study pop: 
sampling method: Probability Sample
criteria: 
gender: All
minimum age: 7 Years
maximum age: 30 Years
last name: Deniz Dogru- Ersoz, Professor
role: Study Director
affiliation: Hacettepe University
last name: Naciye Vardar- Yagli, PhD
role: Study Chair
affiliation: Hacettepe University
last name: Dilber Ademhan- Tural, MD
role: Study Chair
affiliation: Hacettepe University
last name: Nural Kiper, Professor
role: Study Chair
affiliation: Hacettepe University
last name: Ugur Ozcelik, Professor
role: Study Chair
affiliation: Hacettepe University
last name: Ebru Yalcin, Professor
role: Study Chair
affiliation: Hacettepe University
last name: Deniz Inal-Ince, Professor
role: Study Chair
affiliation: Hacettepe University
last name: Melda Saglam, PhD
role: Study Chair
affiliation: Hacettepe University
last name: Serap Sivri, Professor
role: Study Chair
affiliation: Hacettepe University
last name: Aysegul Tokatlı, Professor
role: Study Chair
affiliation: Hacettepe University
facility: 
country: Turkey
responsible party type: Principal Investigator
investigator affiliation: Hacettepe University
investigator full name: Kübra Kılıç
investigator title: Physiotherapist
mesh term: Mucopolysaccharidoses