xml: <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/NCT06022380</url>
    </required_header>
    <id_info>
      <org_study_id>B00446 - COHERE</org_study_id>
      <secondary_id>261752</secondary_id>
      <nct_id>NCT06022380</nct_id>
    </id_info>
    <brief_title>Core Outcome Set for Head, Neck and Respiratory Disease in Mucopolysaccharidosis II</brief_title>
    <acronym>COHERE</acronym>
    <official_title>COHERE - Core Outcome Set for Head, Neck and Respiratory Disease in Mucopolysaccharidosis II</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Manchester University NHS Foundation Trust</agency>
        <agency_class>Other</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>Manchester University NHS Foundation Trust</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 Mucopolysaccharidoses (MPS) are a family of genetic metabolic disorders, caused by&#xD;
        specific enzyme deficiencies which result in accumulation of partially degraded&#xD;
        glycosaminoglycans (GAGs) within various tissues. As GAGs are common in the body a number of&#xD;
        different organ systems can be affected. Involvement of the upper and lower respiratory tract&#xD;
        in MPS Type II results in significant airway compromise, with progressive airway obstruction&#xD;
        being responsible for a significant proportion of the morbidity and mortality associated with&#xD;
        this condition. Hearing loss is a universal finding in MPS, with a third of patients&#xD;
        suffering with severe profound hearing loss. There is an unmet need for strong clinical&#xD;
        evidence to guide treatment of head, neck and respiratory disease in MPS disorders. A Core&#xD;
        Outcome Set (COS) describes the minimum outcome data that should be measured in a clinical&#xD;
        study for a particular condition. The lack of an agreed COS for MPS II in general, and&#xD;
        specifically head, neck and respiratory disease, makes comparison between studies difficult.&#xD;
        There is also a lack of information detailing patient and parent perspectives on the MPS&#xD;
        disorders. The ideal COS for head, neck and respiratory disease associated with MPS II would&#xD;
        combine both patient/parent and clinician opinion and could be used in the design of all&#xD;
        subsequent clinical studies. Following literature review the investigators have created a&#xD;
        list of outcomes previously reported for qualitative and quantitative studies investigating&#xD;
        head, neck and respiratory disease in MPS II. For the proposed research the investigators&#xD;
        will seek opinions of patients, parents, clinicians and scientists to rate these outcomes via&#xD;
        the Delphi method. Outcomes scored highest by patients, parents, clinicians and scientists&#xD;
        will form a COS for head, neck and respiratory disease in MPS II. The development of a COS&#xD;
        can help limit variability outcomes in studies investigating different interventions in MPS&#xD;
        II.&#xD;
      </textblock>
    </brief_summary>
    <overall_status>Completed</overall_status>
    <start_date type="Actual">September 28, 2020</start_date>
    <completion_date type="Actual">June 30, 2021</completion_date>
    <primary_completion_date type="Actual">June 30, 2021</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>What are the most important outcomes that should be measured in trials examining ENT and respiratory disease in MPS Type II populations, aged 0-11 and 12-25 years old?</measure>
      <time_frame>8 months</time_frame>
      <description>Step 1. A systematic review of the literature and clinical trials registries (including the Hunter Outcome Survey) was undertaken to identify a comprehensive list of head, neck and respiratory disease outcomes reported in literature and trials of MPS Type II population. Additionally, qualitative research investigated perspectives of patients with MPS Type II.&#xD;
  Step 2. A Delphi survey of 3 key stakeholder groups (Patients (only for COS for the age group 12-25 years old), parents/ care givers, MPS Type II healthcare professionals), will be undertaken to try to achieve consensus about the outcomes to include in a COS.&#xD;
  Step 3, This will be followed by a consensus meeting of key stakeholders to ratify the COS.</description>
    </primary_outcome>
    <number_of_groups>2</number_of_groups>
    <enrollment type="Actual">46</enrollment>
    <condition>Mucopolysaccharidosis Type II</condition>
    <arm_group>
      <arm_group_label>Round 1</arm_group_label>
      <description>Participants will be asked to score each outcome domain on a 9 point scale proposed by the GRADE group [http://www.gradeworkinggroup.org], in which 1 to 3 signifies an outcome of 'limited importance', 4 to 6 'important but not critical', and 7 to 9 'critical'. Round 1 will also provide participants with the opportunity to add further outcomes which they think may be important. Any suggested outcomes deemed to represent a new outcome domain by the study group (following discussion within study advisory group and a majority decision) will be added to the list for consideration in round two. Round 1 will last for approximately 2 months.</description>
    </arm_group>
    <arm_group>
      <arm_group_label>Round 2</arm_group_label>
      <description>All items (in addition to further new outcome domains identified by participants in round 1) will be carried forward for consideration in round 2. Descriptive statistics (graphs) will be used to summarize the scores from round 1 and presented to participants. Participants will see the results of their individual score for each outcome in addition to the median score of each stakeholder group. The rationale for showing participants the scores from other groups is that it may improve consensus between the stakeholder groups. In addition, by carrying all items forward from round 1, it may be possible to identify changes in scoring patterns as a result of viewing other scores. Participants will be asked to score all items once again using the 9-point scale. Round 2 will last for approximately 2 month</description>
    </arm_group>
    <eligibility>
      <study_pop>
        <textblock>
          Patients and parents, clinicians and scientists&#xD;
        </textblock>
      </study_pop>
      <sampling_method>Non-Probability Sample</sampling_method>
      <criteria>
        <textblock>
          Inclusion criteria:&#xD;
  &#xD;
            -  Confirmed MPS II diagnosis.&#xD;
  &#xD;
            -  Aged between 12 and 25 years old.&#xD;
  &#xD;
            -  Parents/ main care givers of child with MPS Type II.&#xD;
  &#xD;
            -  Adults aged 18 or over.&#xD;
  &#xD;
            -  Clinicians responsible for direct care and management of at least 2 pediatric MPS Type&#xD;
               II patients in the last 12 months.&#xD;
  &#xD;
            -  Scientists who have worked with MPS Type II within the last 5 years&#xD;
  &#xD;
          Exclusion criteria:&#xD;
  &#xD;
            -  Unconfirmed MPS Type II diagnosis.&#xD;
  &#xD;
            -  Aged below 12 and above 25 years old.&#xD;
  &#xD;
            -  Not directly involved in a day to day care.&#xD;
  &#xD;
            -  Clinicians who only occasionally look after pediatric MPS Type II patients.&#xD;
  &#xD;
            -  Scientists who have not worked with MPS Type II within the last 5 years&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>0 Years</minimum_age>
      <maximum_age>100 Years</maximum_age>
      <healthy_volunteers>No</healthy_volunteers>
    </eligibility>
    <location>
      <facility>
        <name>Manchester University NHS Foundation Trust</name>
        <address>
          <city>Manchester</city>
          <state>Lancashire</state>
          <zip>M13 9WL</zip>
          <country>United Kingdom</country>
        </address>
      </facility>
    </location>
    <location_countries>
      <country>United Kingdom</country>
    </location_countries>
    <verification_date>August 2023</verification_date>
    <study_first_submitted>August 24, 2023</study_first_submitted>
    <study_first_submitted_qc>August 31, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">September 1, 2023</study_first_posted>
    <last_update_submitted>August 31, 2023</last_update_submitted>
    <last_update_submitted_qc>August 31, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">September 1, 2023</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>Respiratory Tract Diseases</mesh_term>
      <mesh_term>Respiration Disorders</mesh_term>
      <mesh_term>Mucopolysaccharidosis II</mesh_term>
      <mesh_term>Mucopolysaccharidoses</mesh_term>
    </condition_browse>
    <!-- Results have not yet been posted for this study                                          -->
  </clinical_study>
  
  
text: 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/NCT06022380
  org study id: B00446 - COHERE
  secondary id: 261752
  nct id: NCT06022380
  lead sponsor: 
  has dmc: No
  is fda regulated drug: No
  is fda regulated device: No
  The Mucopolysaccharidoses (MPS) are a family of genetic metabolic disorders, caused by
        specific enzyme deficiencies which result in accumulation of partially degraded
        glycosaminoglycans (GAGs) within various tissues. As GAGs are common in the body a number of
        different organ systems can be affected. Involvement of the upper and lower respiratory tract
        in MPS Type II results in significant airway compromise, with progressive airway obstruction
        being responsible for a significant proportion of the morbidity and mortality associated with
        this condition. Hearing loss is a universal finding in MPS, with a third of patients
        suffering with severe profound hearing loss. There is an unmet need for strong clinical
        evidence to guide treatment of head, neck and respiratory disease in MPS disorders. A Core
        Outcome Set (COS) describes the minimum outcome data that should be measured in a clinical
        study for a particular condition. The lack of an agreed COS for MPS II in general, and
        specifically head, neck and respiratory disease, makes comparison between studies difficult.
        There is also a lack of information detailing patient and parent perspectives on the MPS
        disorders. The ideal COS for head, neck and respiratory disease associated with MPS II would
        combine both patient/parent and clinician opinion and could be used in the design of all
        subsequent clinical studies. Following literature review the investigators have created a
        list of outcomes previously reported for qualitative and quantitative studies investigating
        head, neck and respiratory disease in MPS II. For the proposed research the investigators
        will seek opinions of patients, parents, clinicians and scientists to rate these outcomes via
        the Delphi method. Outcomes scored highest by patients, parents, clinicians and scientists
        will form a COS for head, neck and respiratory disease in MPS II. The development of a COS
        can help limit variability outcomes in studies investigating different interventions in MPS
        II.
  observational model: Cohort
  time perspective: Prospective
  measure: What are the most important outcomes that should be measured in trials examining ENT and respiratory disease in MPS Type II populations, aged 0-11 and 12-25 years old?
  time frame: 8 months
  description: Step 1. A systematic review of the literature and clinical trials registries (including the Hunter Outcome Survey) was undertaken to identify a comprehensive list of head, neck and respiratory disease outcomes reported in literature and trials of MPS Type II population. Additionally, qualitative research investigated perspectives of patients with MPS Type II.
  Step 2. A Delphi survey of 3 key stakeholder groups (Patients (only for COS for the age group 12-25 years old), parents/ care givers, MPS Type II healthcare professionals), will be undertaken to try to achieve consensus about the outcomes to include in a COS.
  Step 3, This will be followed by a consensus meeting of key stakeholders to ratify the COS.
  arm group label: Round 1
  description: Participants will be asked to score each outcome domain on a 9 point scale proposed by the GRADE group [http://www.gradeworkinggroup.org], in which 1 to 3 signifies an outcome of 'limited importance', 4 to 6 'important but not critical', and 7 to 9 'critical'. Round 1 will also provide participants with the opportunity to add further outcomes which they think may be important. Any suggested outcomes deemed to represent a new outcome domain by the study group (following discussion within study advisory group and a majority decision) will be added to the list for consideration in round two. Round 1 will last for approximately 2 months.
  arm group label: Round 2
  description: All items (in addition to further new outcome domains identified by participants in round 1) will be carried forward for consideration in round 2. Descriptive statistics (graphs) will be used to summarize the scores from round 1 and presented to participants. Participants will see the results of their individual score for each outcome in addition to the median score of each stakeholder group. The rationale for showing participants the scores from other groups is that it may improve consensus between the stakeholder groups. In addition, by carrying all items forward from round 1, it may be possible to identify changes in scoring patterns as a result of viewing other scores. Participants will be asked to score all items once again using the 9-point scale. Round 2 will last for approximately 2 month
  study pop: 
  sampling method: Non-Probability Sample
  criteria: 
  gender: All
  minimum age: 0 Years
  maximum age: 100 Years
  healthy volunteers: No
  facility: 
  country: United Kingdom
  responsible party type: Sponsor
  mesh term: Respiratory Tract Diseases
  mesh term: Respiration Disorders
  mesh term: Mucopolysaccharidosis II
  mesh term: Mucopolysaccharidoses