<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/NCT02198430</url>
  </required_header>
  <id_info>
    <org_study_id>Hemo-HOLISTIC</org_study_id>
    <secondary_id>HOLISTIC</secondary_id>
    <nct_id>NCT02198430</nct_id>
  </id_info>
  <brief_title>Multidisciplinary Evaluation of Patients With Hemophilia</brief_title>
  <acronym>HOLISTIC</acronym>
  <official_title>Multidisciplinary Assessment of the Physical, Functional and Psychosocial Alterations in Patients With Hemophilia. An Observational Study.</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Universidad de Murcia</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Universidad de Murcia</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      Multidisciplinary assessment of the physical, functional and psychosocial alterations in&#xD;
      patients with hemophilia around the country. The aims of this study are:&#xD;
&#xD;
        -  Descriptive study of joint involvement in hemophilia patients with and without a history&#xD;
           of hemarthrosis.&#xD;
&#xD;
        -  Descriptive study of the alterations of periarticular muscle strength as a result of&#xD;
           acute processes or the development of chronic articular sequelae.&#xD;
&#xD;
        -  Descriptive study of biomechanical changes in lower limb joints and their impact on gait&#xD;
           in patients with hemophilia.&#xD;
&#xD;
        -  Descriptive study of the joint space, regarding hemarthrosis and synovitis,&#xD;
           intraarticular by ultrasonography.&#xD;
&#xD;
        -  Descriptive study of the relationship between skeletal muscle pathology in patients with&#xD;
           hemophilia and their perceptions of it and their quality of life.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Observational study that will allow us:&#xD;
&#xD;
      Analyze and evaluate the skeletal muscle of patients with hemophilia involvement.&#xD;
&#xD;
      Describe the periarticular muscular deficit, depending on the degree of injury, age, presence&#xD;
      of inhibitors and the development of physical activity.&#xD;
&#xD;
      Identify the relationship between the functional deficit by clinical assessment performed&#xD;
      with the use of validated scales measure force production with patients.&#xD;
&#xD;
      Observe the presence of joint bleeds in children with hemophilia and synovial hypertrophy&#xD;
      developing therein.&#xD;
&#xD;
      Detect biomechanical abnormalities of the lower limbs and their impact on gait in patients&#xD;
      with hemophilia.&#xD;
&#xD;
      Assess the factors that influence the perception of illness and quality of life of patients&#xD;
      with hemophilia.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date>May 2014</start_date>
  <completion_date type="Actual">February 2016</completion_date>
  <primary_completion_date type="Actual">November 2015</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>Assess the Joint Damage</measure>
    <time_frame>Screening visit</time_frame>
    <description>Measurement with Haemophilia Joint Health Score 2.1 (HJHS)</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Assess the Perception of Quality of Life</measure>
    <time_frame>Screening visit</time_frame>
    <description>Measurement through the Child health profile (Childhood Health and Illness Perception; CHIP-CE).</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Assessment of Clinical Patient Variables</measure>
    <time_frame>Screening visit</time_frame>
    <description>Hemophilia type measuring (A or B)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Measure of Weight</measure>
    <time_frame>Screening visit (pretreatment assessment)</time_frame>
    <description>Measure of weight</description>
  </secondary_outcome>
  <number_of_groups>2</number_of_groups>
  <enrollment type="Actual">104</enrollment>
  <condition>Haemophilia</condition>
  <arm_group>
    <arm_group_label>Patients</arm_group_label>
    <description>Patients with haemophilia recruited for multidisciplinary assessment of the main physical, functional and psychosocial variables.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Control group</arm_group_label>
    <description>Children without hemophilia</description>
  </arm_group>
  <eligibility>
    <study_pop>
      <textblock>
        Patients with haemophilia attending hemophilia shelters organized by the Federación&#xD;
        Española de Hemofilia (Fedhemo) in the Centro de Formación Permanente en Hemofilia La&#xD;
        Charca (Murcia, Spain).&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Patients with hemophilia A and B.&#xD;
&#xD;
          -  Patients of all ages (pediatric, adolescents, youth and adults).&#xD;
&#xD;
          -  With or without joint involvement clinically diagnosed, and with or without a previous&#xD;
             history of hemarthrosis joint loading.&#xD;
&#xD;
          -  Patients with or without inhibitors.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Patients without prior walking capacity.&#xD;
&#xD;
          -  Patients diagnosed with other congenital coagulopathy (von Willebrand disease, etc..).&#xD;
&#xD;
          -  Patients with neurological or cognitive impairments that prevent understanding the&#xD;
             questionnaires and physical tests.&#xD;
      </textblock>
    </criteria>
    <gender>Male</gender>
    <minimum_age>8 Years</minimum_age>
    <maximum_age>60 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Rubén Cuesta-Barriuso, PhD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Catholic University San Antonio of Murcia</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Universidad de Murcia</name>
      <address>
        <city>Murcia</city>
        <zip>30100</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Spain</country>
  </location_countries>
  <verification_date>June 2016</verification_date>
  <study_first_submitted>July 22, 2014</study_first_submitted>
  <study_first_submitted_qc>July 22, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">July 23, 2014</study_first_posted>
  <results_first_submitted>June 29, 2016</results_first_submitted>
  <results_first_submitted_qc>August 26, 2016</results_first_submitted_qc>
  <results_first_posted type="Estimate">October 20, 2016</results_first_posted>
  <last_update_submitted>August 26, 2016</last_update_submitted>
  <last_update_submitted_qc>August 26, 2016</last_update_submitted_qc>
  <last_update_posted type="Estimate">October 20, 2016</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Universidad de Murcia</investigator_affiliation>
    <investigator_full_name>ANA TORRES-ORTUÑO</investigator_full_name>
    <investigator_title>PhD</investigator_title>
  </responsible_party>
  <keyword>Haemophilia</keyword>
  <keyword>Arthropathy</keyword>
  <keyword>Range of motion</keyword>
  <keyword>Strength</keyword>
  <keyword>Proprioception</keyword>
  <keyword>Gait</keyword>
  <keyword>Quality of Life.</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Hemophilia A</mesh_term>
  </condition_browse>

  <clinical_results>

    <participant_flow>
      <group_list>
        <group group_id="P1">
          <title>Patients</title>
          <description>Patients with haemophilia recruited for multidisciplinary assessment of the main physical, functional and psychosocial variables.</description>
        </group>
        <group group_id="P2">
          <title>Control Group</title>
          <description>Children without hemophilia</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="53"/>
                <participants group_id="P2" count="51"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="53"/>
                <participants group_id="P2" count="51"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
                <participants group_id="P2" count="0"/>
              </participants_list>
            </milestone>
          </milestone_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <group_list>
        <group group_id="B1">
          <title>Patients</title>
          <description>Patients with haemophilia recruited for multidisciplinary assessment of the main physical, functional and psychosocial variables.</description>
        </group>
        <group group_id="B2">
          <title>Control Group</title>
          <description>Children without hemophilia</description>
        </group>
        <group group_id="B3">
          <title>Total</title>
          <description>Total of all reporting groups</description>
        </group>
      </group_list>
      <analyzed_list>
        <analyzed>
          <units>Participants</units>
          <scope>Overall</scope>
          <count_list>
            <count group_id="B1" value="53"/>
            <count group_id="B2" value="51"/>
            <count group_id="B3" value="104"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>years</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="10.08" spread="1.36"/>
                    <measurement group_id="B2" value="9.78" spread="1.22"/>
                    <measurement group_id="B3" value="10.04" spread="1.31"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Sex: Female, Male</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Female</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="53"/>
                    <measurement group_id="B2" value="51"/>
                    <measurement group_id="B3" value="104"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Region of Enrollment</title>
          <units>participants</units>
          <param>Number</param>
          <class_list>
            <class>
              <title>Spain</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="53"/>
                    <measurement group_id="B2" value="51"/>
                    <measurement group_id="B3" value="104"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Assess the Joint Damage</title>
        <description>Measurement with Haemophilia Joint Health Score 2.1 (HJHS)</description>
        <time_frame>Screening visit</time_frame>
        <population>Haemophilia Joint Health Score assesses joint health in patients with hemophilia. It consists of eight dimensions: swelling, muscular atrophy, crepitation and range of motion, joint pain, strength, motion and axial alignment. The score range is from 0 to 24 points (a score of 0 indicates no joint damage. The higher the score, the higher).</population>
        <group_list>
          <group group_id="O1">
            <title>Patients</title>
            <description>Patients with haemophilia recruited for multidisciplinary assessment of the main physical, functional and psychosocial variables.</description>
          </group>
          <group group_id="O2">
            <title>Control Group</title>
            <description>Children without hemophilia</description>
          </group>
        </group_list>
        <measure>
          <title>Assess the Joint Damage</title>
          <description>Measurement with Haemophilia Joint Health Score 2.1 (HJHS)</description>
          <population>Haemophilia Joint Health Score assesses joint health in patients with hemophilia. It consists of eight dimensions: swelling, muscular atrophy, crepitation and range of motion, joint pain, strength, motion and axial alignment. The score range is from 0 to 24 points (a score of 0 indicates no joint damage. The higher the score, the higher).</population>
          <units>points</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="53"/>
                <count group_id="O2" value="51"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.36" spread="0.84"/>
                    <measurement group_id="O2" value="0" spread="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Assess the Perception of Quality of Life</title>
        <description>Measurement through the Child health profile (Childhood Health and Illness Perception; CHIP-CE).</description>
        <time_frame>Screening visit</time_frame>
        <population>The score ranges from 0 (poor QoL) to 100 points (good perception of QoL).</population>
        <group_list>
          <group group_id="O1">
            <title>Patients</title>
            <description>Patients with haemophilia recruited for multidisciplinary assessment of the main physical, functional and psychosocial variables.</description>
          </group>
          <group group_id="O2">
            <title>Control Group</title>
            <description>Children without hemophilia</description>
          </group>
        </group_list>
        <measure>
          <title>Assess the Perception of Quality of Life</title>
          <description>Measurement through the Child health profile (Childhood Health and Illness Perception; CHIP-CE).</description>
          <population>The score ranges from 0 (poor QoL) to 100 points (good perception of QoL).</population>
          <units>points</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="53"/>
                <count group_id="O2" value="51"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="38.32" spread="4.72"/>
                    <measurement group_id="O2" value="37.55" spread="5.91"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Assessment of Clinical Patient Variables</title>
        <description>Hemophilia type measuring (A or B)</description>
        <time_frame>Screening visit</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Patients</title>
            <description>Patients with haemophilia recruited for multidisciplinary assessment of the main physical, functional and psychosocial variables.</description>
          </group>
          <group group_id="O2">
            <title>Control Group</title>
            <description>Children without hemophilia</description>
          </group>
        </group_list>
        <measure>
          <title>Assessment of Clinical Patient Variables</title>
          <description>Hemophilia type measuring (A or B)</description>
          <units>Percentage of Participants with Hemophil</units>
          <param>Number</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="53"/>
                <count group_id="O2" value="51"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="92.5"/>
                    <measurement group_id="O2" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Measure of Weight</title>
        <description>Measure of weight</description>
        <time_frame>Screening visit (pretreatment assessment)</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Patients</title>
            <description>Patients with haemophilia recruited for multidisciplinary assessment of the main physical, functional and psychosocial variables.</description>
          </group>
          <group group_id="O2">
            <title>Control Group</title>
            <description>Children without hemophilia</description>
          </group>
        </group_list>
        <measure>
          <title>Measure of Weight</title>
          <description>Measure of weight</description>
          <units>Kg</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="53"/>
                <count group_id="O2" value="51"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="41.59" spread="11.82"/>
                    <measurement group_id="O2" value="39.36" spread="9.64"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>Serious and Other (Not Including Serious) Adverse Events were not collected/assessed</time_frame>
      <group_list>
        <group group_id="E1">
          <title>Patients</title>
          <description>Patients with haemophilia recruited for multidisciplinary assessment of the main physical, functional and psychosocial variables.</description>
        </group>
        <group group_id="E2">
          <title>Control Group</title>
          <description>Children without hemophilia</description>
        </group>
      </group_list>
      <serious_events>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="0"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="0"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>0</frequency_threshold>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, other adverse events</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="0"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="0"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </other_events>
    </reported_events>

    <certain_agreements>
      <pi_employee>Principal Investigators are NOT employed by the organization sponsoring the study.</pi_employee>
      <restrictive_agreement>The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.</restrictive_agreement>
    </certain_agreements>
    <point_of_contact>
      <name_or_title>Dr. Rubén Cuesta-Barriuso</name_or_title>
      <organization>Real Fundación Victoria Eugenia</organization>
      <phone>+34913146508</phone>
      <email>ruben.cuestab@gmail.com</email>
    </point_of_contact>
  </clinical_results>
</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/NCT02198430
org study id: Hemo-HOLISTIC
secondary id: HOLISTIC
nct id: NCT02198430
lead sponsor: 
has dmc: No
Multidisciplinary assessment of the physical, functional and psychosocial alterations in
      patients with hemophilia around the country. The aims of this study are:

        -  Descriptive study of joint involvement in hemophilia patients with and without a history
           of hemarthrosis.

        -  Descriptive study of the alterations of periarticular muscle strength as a result of
           acute processes or the development of chronic articular sequelae.

        -  Descriptive study of biomechanical changes in lower limb joints and their impact on gait
           in patients with hemophilia.

        -  Descriptive study of the joint space, regarding hemarthrosis and synovitis,
           intraarticular by ultrasonography.

        -  Descriptive study of the relationship between skeletal muscle pathology in patients with
           hemophilia and their perceptions of it and their quality of life.
Observational study that will allow us:

      Analyze and evaluate the skeletal muscle of patients with hemophilia involvement.

      Describe the periarticular muscular deficit, depending on the degree of injury, age, presence
      of inhibitors and the development of physical activity.

      Identify the relationship between the functional deficit by clinical assessment performed
      with the use of validated scales measure force production with patients.

      Observe the presence of joint bleeds in children with hemophilia and synovial hypertrophy
      developing therein.

      Detect biomechanical abnormalities of the lower limbs and their impact on gait in patients
      with hemophilia.

      Assess the factors that influence the perception of illness and quality of life of patients
      with hemophilia.
observational model: Cohort
time perspective: Prospective
measure: Assess the Joint Damage
time frame: Screening visit
description: Measurement with Haemophilia Joint Health Score 2.1 (HJHS)
measure: Assess the Perception of Quality of Life
time frame: Screening visit
description: Measurement through the Child health profile (Childhood Health and Illness Perception; CHIP-CE).
measure: Assessment of Clinical Patient Variables
time frame: Screening visit
description: Hemophilia type measuring (A or B)
measure: Measure of Weight
time frame: Screening visit (pretreatment assessment)
description: Measure of weight
arm group label: Patients
description: Patients with haemophilia recruited for multidisciplinary assessment of the main physical, functional and psychosocial variables.
arm group label: Control group
description: Children without hemophilia
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: Male
minimum age: 8 Years
maximum age: 60 Years
healthy volunteers: No
last name: Rubén Cuesta-Barriuso, PhD
role: Principal Investigator
affiliation: Catholic University San Antonio of Murcia
facility: 
country: Spain
responsible party type: Principal Investigator
investigator affiliation: Universidad de Murcia
investigator full name: ANA TORRES-ORTUÑO
investigator title: PhD
mesh term: Hemophilia A
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
point of contact: 