<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/NCT04473612</url>
  </required_header>
  <id_info>
    <org_study_id>DF0091UG</org_study_id>
    <nct_id>NCT04473612</nct_id>
  </id_info>
  <brief_title>Physical Status and Upper Airway Function in Neuromuscular Patients</brief_title>
  <official_title>Degree Physical Status and Upper Airway Function in Neuromuscular Patients</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Universidad de Granada</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Universidad de Granada</source>
  <oversight_info>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      Inside of studies that report an upper airway function impaired, the upper limb strength has&#xD;
      been demonstrated to be determinant in breath, speech and swallow performance. Nevertheless,&#xD;
      no previous studies in neuromuscular disease have explored the relation between general&#xD;
      disability and upper airway function.&#xD;
&#xD;
      There is a suspicion of upper airway which is associated with physical deterioration, however&#xD;
      the extent to which if feeds back has not been explored.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">July 20, 2020</start_date>
  <completion_date type="Actual">September 1, 2020</completion_date>
  <primary_completion_date type="Actual">August 15, 2020</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Case-Control</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Hand grip strength</measure>
    <time_frame>Baseline</time_frame>
    <description>Hand grip strength will be evaluated with a pressure dynamometer according to the protocol, to their maximum ability in a seated position . The average of three attempts for the dominant hand will be recorded.&#xD;
It has been proven that it can be used as an indicator of general health and as a strong predictor of future mortality, disability, falls, complications and resource utilization.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Respiratory function</measure>
    <time_frame>Baseline</time_frame>
    <description>Respiratory function will be evaluated with spirometry following standardized protocols. Collected variables will be Forced Expiratory Volume in the First Second (FEV1), Forced Vital Capacity (FVC) and Maximal Voluntary Ventilation (MVV).</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Peak expiratory flow</measure>
    <time_frame>Baseline</time_frame>
    <description>Peak expiratory flow is a person's maximum speed of expiration, as measured with a peak flow meter, a small, hand-held device used to monitor a person's ability to breathe out air. It will be measured following standardized protocols.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>European Quality of Life-5 Dimensions (EQ-5D)</measure>
    <time_frame>Baseline</time_frame>
    <description>The perception of the level of health and the quality of life were evaluated with the questionnaire European Quality of Life-5 Dimensions (EQ-5D). It is composed of 2 parts in which health status is assessed:&#xD;
Descriptive system: It consists on 5 dimensions that include mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Patients classify their health status in 3 severity levels: having no problems, having some or moderate problems, being unable to do/having extreme problems.&#xD;
Visual Analog Scale, by which respondents can report their perceived health status with a grade ranging from 0 (the worst possible health status) to 100 (the best possible health status).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Fatigue Severity Scale</measure>
    <time_frame>Baseline</time_frame>
    <description>Fatigue severity scale (FSS) will be used to measure fatigue. It consists of 9 items with answers that the patient scores between 1 and 7. The total is calculated by adding all the items. For children will be used The PedsQL™ Multidimensional Fatigue Scale, which is composed of 18 items comprising 3 dimensions (general fatigue, sleep/rest fatigue, cognitive fatigue), evaluated thorough a Liker scale with values from 0 (never) to 4 (almost always), referring to difficulty presented.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>World Health Organization Disability Assessment Scale (WHODAS) 2.0 (WD2)</measure>
    <time_frame>Baseline</time_frame>
    <description>To measure the degree of disability, the World Health Organization Disability Assessment Scale, WHODAS 2.0 (WD2) will be used. It measures changes in functioning and levels of difficulty in carrying out and carrying out its activities . The WD2 scale is composed of 36 items divided into 6 domains, evaluated through a Likert scale with values from 1 to 5, referring to the difficulty presented. Higher scores show a greater degree of disability.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Activity Limitations in Children and Adults With Neuromuscular Disorders (ACTIVLIM)</measure>
    <time_frame>Baseline</time_frame>
    <description>The Activity Limitations in Children and Adults With Neuromuscular Disorders (ACTIVLIM) questionnaire is an instrument to measure functional limitation and has been calibrated in children and adults with neuromuscular diseases, according to the difficulty of patients to perform daily activities that require the use of the upper extremities and / or lower.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Brief pain inventory</measure>
    <time_frame>Baseline</time_frame>
    <description>For pain assessment we will use a specific brief pain questionnaire, Brief Pain Inventory , which consists of three parts. In the first, the patient will indicate in a drawing the area in which he presents this pain. The second part refers to the pain felt in the last days, where it is scored, as in the previous questionnaire, from 0 to 10 (0 being no pain and 10 being the maximum pain felt). Finally, in a third part we see how that pain influences the different activities that the patient performs in his day to day.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Hospital Anxiety and Depression Scale</measure>
    <time_frame>Baseline</time_frame>
    <description>Anxiety and depression will be assessed with the Hospital Anxiety and Depression Scale (HADS) . This scale is composed of 14 items divided into two subscales, 7 items of anxiety and 7 items of depression, involving higher values worse psycho-emotional state.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Muscle strength of the neck</measure>
    <time_frame>Baseline</time_frame>
    <description>To evaluate the muscle strength of the neck in neuromuscular patients, neck dynamometry will be performed. With subtests in which front bending, extension, pure lateral flexion, lateral flexion with rotation and pure rotation were measures.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Eating Assessment Tool-10 (EAT-10)</measure>
    <time_frame>Baseline</time_frame>
    <description>The EAT-10 is a self-administered instrument that evaluate specific symptoms of dysphagia with 10 items and each item have a range from zero (no problem to swallow) to four (a serious problem for life).&#xD;
In the case of patients pediatric with dysphagia it used Pediatric Eating Assessment Tool (PEDI-EAT-10)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Swallowing quality of life questionnaire (SWAL-QOL)</measure>
    <time_frame>Baseline</time_frame>
    <description>To measure the possible swallowing problems in daily life we will used the SWAL-QOL, it contains 44 items and each item was scored from zero (the worst state) to four (the best state) . This questionnaire consists of 44 items, consisting of 11 subscales. With a Likert type score in which the patient scores between 1 and 5. Where 1 indicates the worst state and 5 the most favorable.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Functional Oral Intake Scale (FOIS) questionnaire</measure>
    <time_frame>Baseline</time_frame>
    <description>The severity of swallowing will be assessed using the Functional Oral Intake Scale (FOIS) questionnaire . This scale consists of 7 levels, with 1 being the most severe and 7 being the least severe, the patient has to select the level with which he is.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Karaduman Chewing Performance Scale (KCPS)</measure>
    <time_frame>Baseline</time_frame>
    <description>There are also questionnaires like Karaduman Chewing Performance Scale (KCPS) , that permitted us evaluate the function of chewing, this test has not been used in previous studies in adults, so in this study we have only used it in the childrens patients.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Orofacial Myofunctional Evaluation with Scores (OMES)</measure>
    <time_frame>Baseline</time_frame>
    <description>Clinical evaluation of the orofacial myofunctional condition will be evaluated using the Orofacial Myofunctional Evaluation with Scores (OMES) . It consisting of 4 subscales: appearance and posture, mobility and functions (breathing, deglutition, mastication). The examiner attributed scores on a 3 point scale: 3=normal, 2= insufficient ability; and 1= absence of ability or being unable to perform the task.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Volume Viscosity Clinical Exploration Method (MECV-V)</measure>
    <time_frame>Baseline</time_frame>
    <description>Volume Viscosity Clinical Exploration Method (MECV-V) , this test provides us with information about the volume to which the 5ml, 10ml and 20 ml patients are able to feed and tells us what viscosity, nectar, liquid and pudding. It is used in the diagnosis of oropharyngeal dysphagia.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Voice handicap index (VHI-30)</measure>
    <time_frame>Baseline</time_frame>
    <description>The phonatory function and it´s repercussion on daily life will be evaluated by the Voice handicap index (VHI-30) . This scale consists of 30 items, consisting of 3 subscales, functional, physical and emotional. In which the patient must indicate the presence or symptoms of dysphonia with a Likert type score of 5 points, in which 0 indicates never and 4 always indicates.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Grade, Roughness, Breathing, Asthenia, Effort (GRBAS)</measure>
    <time_frame>Baseline</time_frame>
    <description>Perceptual scale of Grade, Roughness, Breathing, Asthenia, Effort (GRBAS) . Will be used to analyze qualitative aspects of the voice, it is completed by the evaluator who qualifies as: 0 normal, 1 mild, 2 moderate, 3 severe, each quality of the voice (Degree of dysphonia, scratchy, blown, weak or asthenic, tight or strangled).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Functional evaluation of the voice</measure>
    <time_frame>Baseline</time_frame>
    <description>The functional evaluation of the voice will allow us to measure the intensity, frequency and duration of the voice: To evaluate the intensity of the voice, measured in decibels (dB), we will use a sound level meter; To evaluate the frequency of the voice , measured in Hz (Hz), we use the PRAAT software (Praat from the Dutch &quot;speak&quot;). In addition, the duration of the phonation and breathing times , measured in Sg, will be evaluated, for this we use an audio recorder.</description>
  </secondary_outcome>
  <number_of_groups>3</number_of_groups>
  <enrollment type="Actual">41</enrollment>
  <condition>Neuromuscular Diseases</condition>
  <condition>Physical Disability</condition>
  <condition>Airway Disease</condition>
  <arm_group>
    <arm_group_label>Neuromuscular low physical status group</arm_group_label>
    <description>Patients diagnosed of Neuromuscular disease with low physical status</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Neuromuscular high physical status group</arm_group_label>
    <description>Patients diagnosed of Neuromuscular disease with high physical status</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Control group</arm_group_label>
    <description>Healthy subjects</description>
  </arm_group>
  <eligibility>
    <study_pop>
      <textblock>
        Patients diagnosed of neuromuscular disease aged 6-65 years with an impairment of the upper&#xD;
        airway, whether in ventilatory, deglutory or communication capacity.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Diagnosis of Neuromuscular disease&#xD;
&#xD;
          -  Between 6 and 65 years&#xD;
&#xD;
          -  Impairment of the upper airway&#xD;
&#xD;
          -  Accepted to sign the informed consent&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Cognitive impairment.&#xD;
&#xD;
          -  Diagnose of autism spectrum disorder or severe intellectual disability&#xD;
&#xD;
          -  Physical or functional impairment that limits the performance of evaluation&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>6 Years</minimum_age>
    <maximum_age>65 Years</maximum_age>
    <healthy_volunteers>Accepts Healthy Volunteers</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>University of Granada</name>
      <address>
        <city>Granada</city>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Spain</country>
  </location_countries>
  <verification_date>October 2020</verification_date>
  <study_first_submitted>July 8, 2020</study_first_submitted>
  <study_first_submitted_qc>July 13, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">July 16, 2020</study_first_posted>
  <last_update_submitted>October 9, 2020</last_update_submitted>
  <last_update_submitted_qc>October 9, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">October 14, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Universidad de Granada</investigator_affiliation>
    <investigator_full_name>Marie Carmen Valenza</investigator_full_name>
    <investigator_title>Principal Investigator; PT; PhD</investigator_title>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Neuromuscular Diseases</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/NCT04473612
org study id: DF0091UG
nct id: NCT04473612
lead sponsor: 
is fda regulated drug: No
is fda regulated device: No
Inside of studies that report an upper airway function impaired, the upper limb strength has
      been demonstrated to be determinant in breath, speech and swallow performance. Nevertheless,
      no previous studies in neuromuscular disease have explored the relation between general
      disability and upper airway function.

      There is a suspicion of upper airway which is associated with physical deterioration, however
      the extent to which if feeds back has not been explored.
observational model: Case-Control
time perspective: Prospective
measure: Hand grip strength
time frame: Baseline
description: Hand grip strength will be evaluated with a pressure dynamometer according to the protocol, to their maximum ability in a seated position . The average of three attempts for the dominant hand will be recorded.
It has been proven that it can be used as an indicator of general health and as a strong predictor of future mortality, disability, falls, complications and resource utilization.
measure: Respiratory function
time frame: Baseline
description: Respiratory function will be evaluated with spirometry following standardized protocols. Collected variables will be Forced Expiratory Volume in the First Second (FEV1), Forced Vital Capacity (FVC) and Maximal Voluntary Ventilation (MVV).
measure: Peak expiratory flow
time frame: Baseline
description: Peak expiratory flow is a person's maximum speed of expiration, as measured with a peak flow meter, a small, hand-held device used to monitor a person's ability to breathe out air. It will be measured following standardized protocols.
measure: European Quality of Life-5 Dimensions (EQ-5D)
time frame: Baseline
description: The perception of the level of health and the quality of life were evaluated with the questionnaire European Quality of Life-5 Dimensions (EQ-5D). It is composed of 2 parts in which health status is assessed:
Descriptive system: It consists on 5 dimensions that include mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Patients classify their health status in 3 severity levels: having no problems, having some or moderate problems, being unable to do/having extreme problems.
Visual Analog Scale, by which respondents can report their perceived health status with a grade ranging from 0 (the worst possible health status) to 100 (the best possible health status).
measure: Fatigue Severity Scale
time frame: Baseline
description: Fatigue severity scale (FSS) will be used to measure fatigue. It consists of 9 items with answers that the patient scores between 1 and 7. The total is calculated by adding all the items. For children will be used The PedsQL™ Multidimensional Fatigue Scale, which is composed of 18 items comprising 3 dimensions (general fatigue, sleep/rest fatigue, cognitive fatigue), evaluated thorough a Liker scale with values from 0 (never) to 4 (almost always), referring to difficulty presented.
measure: World Health Organization Disability Assessment Scale (WHODAS) 2.0 (WD2)
time frame: Baseline
description: To measure the degree of disability, the World Health Organization Disability Assessment Scale, WHODAS 2.0 (WD2) will be used. It measures changes in functioning and levels of difficulty in carrying out and carrying out its activities . The WD2 scale is composed of 36 items divided into 6 domains, evaluated through a Likert scale with values from 1 to 5, referring to the difficulty presented. Higher scores show a greater degree of disability.
measure: Activity Limitations in Children and Adults With Neuromuscular Disorders (ACTIVLIM)
time frame: Baseline
description: The Activity Limitations in Children and Adults With Neuromuscular Disorders (ACTIVLIM) questionnaire is an instrument to measure functional limitation and has been calibrated in children and adults with neuromuscular diseases, according to the difficulty of patients to perform daily activities that require the use of the upper extremities and / or lower.
measure: Brief pain inventory
time frame: Baseline
description: For pain assessment we will use a specific brief pain questionnaire, Brief Pain Inventory , which consists of three parts. In the first, the patient will indicate in a drawing the area in which he presents this pain. The second part refers to the pain felt in the last days, where it is scored, as in the previous questionnaire, from 0 to 10 (0 being no pain and 10 being the maximum pain felt). Finally, in a third part we see how that pain influences the different activities that the patient performs in his day to day.
measure: Hospital Anxiety and Depression Scale
time frame: Baseline
description: Anxiety and depression will be assessed with the Hospital Anxiety and Depression Scale (HADS) . This scale is composed of 14 items divided into two subscales, 7 items of anxiety and 7 items of depression, involving higher values worse psycho-emotional state.
measure: Muscle strength of the neck
time frame: Baseline
description: To evaluate the muscle strength of the neck in neuromuscular patients, neck dynamometry will be performed. With subtests in which front bending, extension, pure lateral flexion, lateral flexion with rotation and pure rotation were measures.
measure: Eating Assessment Tool-10 (EAT-10)
time frame: Baseline
description: The EAT-10 is a self-administered instrument that evaluate specific symptoms of dysphagia with 10 items and each item have a range from zero (no problem to swallow) to four (a serious problem for life).
In the case of patients pediatric with dysphagia it used Pediatric Eating Assessment Tool (PEDI-EAT-10)
measure: Swallowing quality of life questionnaire (SWAL-QOL)
time frame: Baseline
description: To measure the possible swallowing problems in daily life we will used the SWAL-QOL, it contains 44 items and each item was scored from zero (the worst state) to four (the best state) . This questionnaire consists of 44 items, consisting of 11 subscales. With a Likert type score in which the patient scores between 1 and 5. Where 1 indicates the worst state and 5 the most favorable.
measure: Functional Oral Intake Scale (FOIS) questionnaire
time frame: Baseline
description: The severity of swallowing will be assessed using the Functional Oral Intake Scale (FOIS) questionnaire . This scale consists of 7 levels, with 1 being the most severe and 7 being the least severe, the patient has to select the level with which he is.
measure: Karaduman Chewing Performance Scale (KCPS)
time frame: Baseline
description: There are also questionnaires like Karaduman Chewing Performance Scale (KCPS) , that permitted us evaluate the function of chewing, this test has not been used in previous studies in adults, so in this study we have only used it in the childrens patients.
measure: Orofacial Myofunctional Evaluation with Scores (OMES)
time frame: Baseline
description: Clinical evaluation of the orofacial myofunctional condition will be evaluated using the Orofacial Myofunctional Evaluation with Scores (OMES) . It consisting of 4 subscales: appearance and posture, mobility and functions (breathing, deglutition, mastication). The examiner attributed scores on a 3 point scale: 3=normal, 2= insufficient ability; and 1= absence of ability or being unable to perform the task.
measure: Volume Viscosity Clinical Exploration Method (MECV-V)
time frame: Baseline
description: Volume Viscosity Clinical Exploration Method (MECV-V) , this test provides us with information about the volume to which the 5ml, 10ml and 20 ml patients are able to feed and tells us what viscosity, nectar, liquid and pudding. It is used in the diagnosis of oropharyngeal dysphagia.
measure: Voice handicap index (VHI-30)
time frame: Baseline
description: The phonatory function and it´s repercussion on daily life will be evaluated by the Voice handicap index (VHI-30) . This scale consists of 30 items, consisting of 3 subscales, functional, physical and emotional. In which the patient must indicate the presence or symptoms of dysphonia with a Likert type score of 5 points, in which 0 indicates never and 4 always indicates.
measure: Grade, Roughness, Breathing, Asthenia, Effort (GRBAS)
time frame: Baseline
description: Perceptual scale of Grade, Roughness, Breathing, Asthenia, Effort (GRBAS) . Will be used to analyze qualitative aspects of the voice, it is completed by the evaluator who qualifies as: 0 normal, 1 mild, 2 moderate, 3 severe, each quality of the voice (Degree of dysphonia, scratchy, blown, weak or asthenic, tight or strangled).
measure: Functional evaluation of the voice
time frame: Baseline
description: The functional evaluation of the voice will allow us to measure the intensity, frequency and duration of the voice: To evaluate the intensity of the voice, measured in decibels (dB), we will use a sound level meter; To evaluate the frequency of the voice , measured in Hz (Hz), we use the PRAAT software (Praat from the Dutch "speak"). In addition, the duration of the phonation and breathing times , measured in Sg, will be evaluated, for this we use an audio recorder.
arm group label: Neuromuscular low physical status group
description: Patients diagnosed of Neuromuscular disease with low physical status
arm group label: Neuromuscular high physical status group
description: Patients diagnosed of Neuromuscular disease with high physical status
arm group label: Control group
description: Healthy subjects
study pop: 
sampling method: Probability Sample
criteria: 
gender: All
minimum age: 6 Years
maximum age: 65 Years
healthy volunteers: Accepts Healthy Volunteers
facility: 
country: Spain
responsible party type: Principal Investigator
investigator affiliation: Universidad de Granada
investigator full name: Marie Carmen Valenza
investigator title: Principal Investigator; PT; PhD
mesh term: Neuromuscular Diseases
sharing ipd: No