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/NCT05995834</url>
    </required_header>
    <id_info>
      <org_study_id>ISBUS</org_study_id>
      <nct_id>NCT05995834</nct_id>
    </id_info>
    <brief_title>Producing a Novel Symptom Burden Scale for People Living With Idiopathic Multicentric Castleman Disease (ISBUS)</brief_title>
    <official_title>Producing a Novel Symptom Burden Scale for People Living With Idiopathic Multicentric Castleman Disease (ISBUS)</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>EusaPharma (UK) Limited</agency>
        <agency_class>Industry</agency_class>
      </lead_sponsor>
      <collaborator>
        <agency>University of Sheffield (ScHARR)</agency>
        <agency_class>Other</agency_class>
      </collaborator>
      <collaborator>
        <agency>KMC Health Care</agency>
        <agency_class>Other</agency_class>
      </collaborator>
      <collaborator>
        <agency>Castleman Disease Collaborative Network (CDCN)</agency>
        <agency_class>Other</agency_class>
      </collaborator>
    </sponsors>
    <source>EusaPharma (UK) Limited</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>
        Development, validation of a novel symptom burden scale to assess and quantify the burden&#xD;
        experienced by people living with Idiopathic Multicentric Castleman Disease (iMCD).&#xD;
      </textblock>
    </brief_summary>
    <detailed_description>
      <textblock>
        Idiopathic Multicentric Castleman Disease (iMCD) is a rare lymphoproliferative disorder&#xD;
        associated with systemic inflammation and organ dysfunction. The condition has an unknown&#xD;
        aetiology and diagnostic criteria were established recently in 2017. It can be distinguished&#xD;
        from other forms of Castleman Disease, including unicentric presentations and Multicentric&#xD;
        Castleman Disease (MCD) that is associated with the Kaposi sarcoma herpesvirus. iMCD has an&#xD;
        estimated prevalence of between 6.9 and 9.7 people per million. While clinical presentations&#xD;
        vary, iMCD can be associated with a high degree of symptom burden, including constitutional,&#xD;
        gastrointestinal, neuropsychiatric, dermatologic, respiratory, and hematologic or&#xD;
        lymphoreticular problems. Both symptoms and the side-effects of treatment can impact the&#xD;
        health-related quality of life (HRQoL) of people with iMCD&#xD;
  &#xD;
        This international mixed-methods study will be conducted in four stages:&#xD;
  &#xD;
        Stage 1: Item generation&#xD;
  &#xD;
          -  Drawing on the content, data, and analysis from a previous international patient online&#xD;
             survey in iMCD with 65 patients, generate draft PROM content (including a longlist of&#xD;
             candidate draft items under existing themes) to assess symptom burden in iMCD.&#xD;
  &#xD;
          -  Incorporate expert opinion and lived experience to achieve consensus on which items will&#xD;
             be tested for inclusion in the symptom burden scale via an online multi-stakeholder&#xD;
             workshop involving the funder, research team, patient and public involvement and&#xD;
             engagement (PPIE) representative(s) (i.e., patients with iMCD and/or their carers),&#xD;
             healthcare professionals, and any other relevant stakeholders.&#xD;
  &#xD;
        Stage 2: Item testing and refinement&#xD;
  &#xD;
          -  Cognitive debriefing interviews with about 10 people living with iMCD to assess the&#xD;
             content validity of the items (and associated PROM content) in terms of relevance,&#xD;
             comprehensibility, and comprehensiveness. This will include assessing whether they are&#xD;
             important/meaningful to patients.&#xD;
  &#xD;
          -  Analyse and summarise qualitative evidence on content validity, make recommendations on&#xD;
             revisions to the draft PROM, and agree this with the multi-stakeholder advisory group&#xD;
             (as described above) and a separate group of PPIE collaborators.&#xD;
  &#xD;
        Stage 3: Final item selection&#xD;
  &#xD;
          -  Administer the refined PROM (from Stage 2) to as wide a sample as possible of people&#xD;
             living with iMCD. The estimated sample size is 100 patients. The PROM will be&#xD;
             administered alongside additional sociodemographic and clinical questions and measures&#xD;
             of burden and/or health-related quality of life (HRQoL).&#xD;
  &#xD;
          -  Taking into account the sample size, conduct appropriate psychometric analyses in order&#xD;
             to provide evidence for final item selection and to provide preliminary psychometric&#xD;
             evidence on the reliability and validity of the final measure.&#xD;
  &#xD;
          -  Taking into account all of the available qualitative and quantitative evidence, and a&#xD;
             consultation with PPIE collaborators, agree (with the multi-stakeholder advisory group)&#xD;
             on final item selection, with the goal of obtaining an 8-10 item measure of symptom&#xD;
             burden.&#xD;
  &#xD;
        Stage 4: Preliminary measures of change&#xD;
  &#xD;
          -  Stage 4a: Re-administer the refined PROM (from Stage 2) to participants (from Stage 3)&#xD;
             alongside measure(s) of perceived clinical change.&#xD;
  &#xD;
          -  Stage 4b: Conduct qualitative interviews with about 10 participants who have completed&#xD;
             the PROM at both time points to understand what constitutes a meaningful difference from&#xD;
             their perspective.&#xD;
  &#xD;
          -  Triangulate quantitative and qualitative data to estimate a minimally important clinical&#xD;
             difference (MCID) for the new PROM, agreed with the multi-stakeholder advisory group.&#xD;
  &#xD;
        Recruitment for Stages 2-4 will take place in USA, Canada, Brazil, Australia, New Zealand and&#xD;
        the United Kingdom. Interviews will be conducted online for Stages 2 and 4 and the&#xD;
        psychometric survey will be hosted and administered online.&#xD;
  &#xD;
        Governance: The results of each stage will be discussed and final decisions will be made with&#xD;
        a multi-stakeholder group consisting of the wider research team, clinicians and other&#xD;
        healthcare professionals and PPIE.&#xD;
      </textblock>
    </detailed_description>
    <overall_status>Not yet recruiting</overall_status>
    <start_date type="Anticipated">October 2023</start_date>
    <completion_date type="Anticipated">December 2024</completion_date>
    <primary_completion_date type="Anticipated">December 2024</primary_completion_date>
    <study_type>Observational</study_type>
    <has_expanded_access>No</has_expanded_access>
    <study_design_info>
      <observational_model>Other</observational_model>
      <time_perspective>Prospective</time_perspective>
    </study_design_info>
    <primary_outcome>
      <measure>Long list of candidate items for the scale (stage 1)</measure>
      <time_frame>Sept 2023 - Nov 2023</time_frame>
      <description>completion of data analysis from a previous survey</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Completion of interviews (stage 2)</measure>
      <time_frame>Within 6 months from start (With this being a rare condition, data from all patients will be collected at various levels of treatment and time from diagnosis)</time_frame>
      <description>completion of interviews by 10 patients from all recruiting sites</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Completion of questionnaire (stage 3)</measure>
      <time_frame>Within 9 months from start (With this being a rare condition, data from all patients will be collected at various levels of treatment and time from diagnosis). NB: Advisory comments noted.</time_frame>
      <description>completion of questionnaire which includes extra questions about treatments, time of diagnosis.&#xD;
  Completion of EQ-5D for external validity of PROM. NB: EQ-5D is the full name of the instrument and not an acronym.</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Completion of follow-up questionnaire (stage 4a)</measure>
      <time_frame>About 8 weeks after Stage 3 questionnaire. Note: Advisory comments noted.</time_frame>
      <description>completion of follow-up questionnaire from Stage 3 by same patients who participated in Stage 3. Note: The full name of the scale will be discussed and confirmed. At the moment, the working title is Idiopathic Multicentric Castleman Disease Symptom Burden Scale (ISBUS).</description>
    </primary_outcome>
    <primary_outcome>
      <measure>Completion of interviews (stage 4b)</measure>
      <time_frame>About 4 weeks after Stage 4a</time_frame>
      <description>completion of interviews by 10 patients from all recruiting sites</description>
    </primary_outcome>
    <enrollment type="Anticipated">100</enrollment>
    <condition>Idiopathic Multicentric Castleman's Disease</condition>
    <intervention>
      <intervention_type>Other</intervention_type>
      <intervention_name>Research questions</intervention_name>
      <description>Stage 1: Secondary analysis of a previous international survey to identify items&#xD;
  Stage 2: Cognitive debriefing interviews with about 10 people living with iMCD to assess the content validity of the items&#xD;
  Stage 3: Completion of questionnaire to finalise scale&#xD;
  Stage 4a: Re-administering of follow-up questionnaire to Stage 3 patients to assess minimally important clinical difference. Test-retest will be calculated only in patients exhibiting no change.&#xD;
  Stage 4b: Qualitative interviews with a subset of participants from Stages and 4 to help with interpretation of meaningful difference from their perspective</description>
    </intervention>
    <eligibility>
      <study_pop>
        <textblock>
          Adult patients diagnosed with idiopathic Multicentric Castleman Disease (iMCD).&#xD;
        </textblock>
      </study_pop>
      <sampling_method>Non-Probability Sample</sampling_method>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
            -  Diagnosis of idiopathic Multicentric Castleman Disease (iMCD)&#xD;
  &#xD;
            -  Adults (aged 18+ years)&#xD;
  &#xD;
            -  Fluent in English&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            -  No diagnosis of iMCD&#xD;
  &#xD;
            -  Children (aged &lt; 18 years)&#xD;
  &#xD;
            -  Not able to understand or communicate in English&#xD;
  &#xD;
            -  People lacking the capacity to consent&#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>Anju Keetharuth, PhD</last_name>
      <role>Principal Investigator</role>
      <affiliation>University of Sheffield</affiliation>
    </overall_official>
    <overall_official>
      <last_name>Philip Powell, PhD</last_name>
      <role>Principal Investigator</role>
      <affiliation>University of Sheffield</affiliation>
    </overall_official>
    <overall_contact>
      <last_name>Anju Keetharuth, PhD</last_name>
      <phone>+44 (0) 114 222 0884</phone>
      <email>d.keetharuth@sheffield.ac.uk</email>
    </overall_contact>
    <overall_contact_backup>
      <last_name>Philip Powell, PhD</last_name>
      <phone>+44 (0) 114 222 0794</phone>
      <email>p.a.powell@sheffield.ac.uk</email>
    </overall_contact_backup>
    <link>
      <url>https://clinicaltrials.gov/study/NCT03052283?term=Development%20PROM&amp;rank=3</url>
      <description>example of registration of PROMs</description>
    </link>
    <link>
      <url>https://clinicaltrials.gov/study/NCT05274867?term=Development%20PROM&amp;rank=6</url>
      <description>example of registration of PROMs</description>
    </link>
    <link>
      <url>https://clinicaltrials.gov/study/NCT05315765?term=Development%20PROM&amp;rank=8</url>
      <description>example of registration of PROMs</description>
    </link>
    <verification_date>August 2023</verification_date>
    <study_first_submitted>July 26, 2023</study_first_submitted>
    <study_first_submitted_qc>August 10, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">August 16, 2023</study_first_posted>
    <last_update_submitted>August 17, 2023</last_update_submitted>
    <last_update_submitted_qc>August 17, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">August 22, 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>Castleman Disease</mesh_term>
    </condition_browse>
    <patient_data>
      <sharing_ipd>No</sharing_ipd>
      <ipd_description>Deanonymized transcriptions of stages 2 and 4 interviews will be securely stored in the University of Sheffield repository with access restricted to investigators only. Quantitative data from stages 3 and 4a will be processed, stored and accessed similarly in the same location.</ipd_description>
    </patient_data>
    <!-- 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/NCT05995834
  org study id: ISBUS
  nct id: NCT05995834
  lead sponsor: 
  collaborator: 
  collaborator: 
  collaborator: 
  has dmc: No
  is fda regulated drug: No
  is fda regulated device: No
  Development, validation of a novel symptom burden scale to assess and quantify the burden
        experienced by people living with Idiopathic Multicentric Castleman Disease (iMCD).
  Idiopathic Multicentric Castleman Disease (iMCD) is a rare lymphoproliferative disorder
        associated with systemic inflammation and organ dysfunction. The condition has an unknown
        aetiology and diagnostic criteria were established recently in 2017. It can be distinguished
        from other forms of Castleman Disease, including unicentric presentations and Multicentric
        Castleman Disease (MCD) that is associated with the Kaposi sarcoma herpesvirus. iMCD has an
        estimated prevalence of between 6.9 and 9.7 people per million. While clinical presentations
        vary, iMCD can be associated with a high degree of symptom burden, including constitutional,
        gastrointestinal, neuropsychiatric, dermatologic, respiratory, and hematologic or
        lymphoreticular problems. Both symptoms and the side-effects of treatment can impact the
        health-related quality of life (HRQoL) of people with iMCD
  
        This international mixed-methods study will be conducted in four stages:
  
        Stage 1: Item generation
  
          -  Drawing on the content, data, and analysis from a previous international patient online
             survey in iMCD with 65 patients, generate draft PROM content (including a longlist of
             candidate draft items under existing themes) to assess symptom burden in iMCD.
  
          -  Incorporate expert opinion and lived experience to achieve consensus on which items will
             be tested for inclusion in the symptom burden scale via an online multi-stakeholder
             workshop involving the funder, research team, patient and public involvement and
             engagement (PPIE) representative(s) (i.e., patients with iMCD and/or their carers),
             healthcare professionals, and any other relevant stakeholders.
  
        Stage 2: Item testing and refinement
  
          -  Cognitive debriefing interviews with about 10 people living with iMCD to assess the
             content validity of the items (and associated PROM content) in terms of relevance,
             comprehensibility, and comprehensiveness. This will include assessing whether they are
             important/meaningful to patients.
  
          -  Analyse and summarise qualitative evidence on content validity, make recommendations on
             revisions to the draft PROM, and agree this with the multi-stakeholder advisory group
             (as described above) and a separate group of PPIE collaborators.
  
        Stage 3: Final item selection
  
          -  Administer the refined PROM (from Stage 2) to as wide a sample as possible of people
             living with iMCD. The estimated sample size is 100 patients. The PROM will be
             administered alongside additional sociodemographic and clinical questions and measures
             of burden and/or health-related quality of life (HRQoL).
  
          -  Taking into account the sample size, conduct appropriate psychometric analyses in order
             to provide evidence for final item selection and to provide preliminary psychometric
             evidence on the reliability and validity of the final measure.
  
          -  Taking into account all of the available qualitative and quantitative evidence, and a
             consultation with PPIE collaborators, agree (with the multi-stakeholder advisory group)
             on final item selection, with the goal of obtaining an 8-10 item measure of symptom
             burden.
  
        Stage 4: Preliminary measures of change
  
          -  Stage 4a: Re-administer the refined PROM (from Stage 2) to participants (from Stage 3)
             alongside measure(s) of perceived clinical change.
  
          -  Stage 4b: Conduct qualitative interviews with about 10 participants who have completed
             the PROM at both time points to understand what constitutes a meaningful difference from
             their perspective.
  
          -  Triangulate quantitative and qualitative data to estimate a minimally important clinical
             difference (MCID) for the new PROM, agreed with the multi-stakeholder advisory group.
  
        Recruitment for Stages 2-4 will take place in USA, Canada, Brazil, Australia, New Zealand and
        the United Kingdom. Interviews will be conducted online for Stages 2 and 4 and the
        psychometric survey will be hosted and administered online.
  
        Governance: The results of each stage will be discussed and final decisions will be made with
        a multi-stakeholder group consisting of the wider research team, clinicians and other
        healthcare professionals and PPIE.
  observational model: Other
  time perspective: Prospective
  measure: Long list of candidate items for the scale (stage 1)
  time frame: Sept 2023 - Nov 2023
  description: completion of data analysis from a previous survey
  measure: Completion of interviews (stage 2)
  time frame: Within 6 months from start (With this being a rare condition, data from all patients will be collected at various levels of treatment and time from diagnosis)
  description: completion of interviews by 10 patients from all recruiting sites
  measure: Completion of questionnaire (stage 3)
  time frame: Within 9 months from start (With this being a rare condition, data from all patients will be collected at various levels of treatment and time from diagnosis). NB: Advisory comments noted.
  description: completion of questionnaire which includes extra questions about treatments, time of diagnosis.
  Completion of EQ-5D for external validity of PROM. NB: EQ-5D is the full name of the instrument and not an acronym.
  measure: Completion of follow-up questionnaire (stage 4a)
  time frame: About 8 weeks after Stage 3 questionnaire. Note: Advisory comments noted.
  description: completion of follow-up questionnaire from Stage 3 by same patients who participated in Stage 3. Note: The full name of the scale will be discussed and confirmed. At the moment, the working title is Idiopathic Multicentric Castleman Disease Symptom Burden Scale (ISBUS).
  measure: Completion of interviews (stage 4b)
  time frame: About 4 weeks after Stage 4a
  description: completion of interviews by 10 patients from all recruiting sites
  intervention type: Other
  intervention name: Research questions
  description: Stage 1: Secondary analysis of a previous international survey to identify items
  Stage 2: Cognitive debriefing interviews with about 10 people living with iMCD to assess the content validity of the items
  Stage 3: Completion of questionnaire to finalise scale
  Stage 4a: Re-administering of follow-up questionnaire to Stage 3 patients to assess minimally important clinical difference. Test-retest will be calculated only in patients exhibiting no change.
  Stage 4b: Qualitative interviews with a subset of participants from Stages and 4 to help with interpretation of meaningful difference from their perspective
  study pop: 
  sampling method: Non-Probability Sample
  criteria: 
  gender: All
  minimum age: 18 Years
  maximum age: N/A
  healthy volunteers: No
  last name: Anju Keetharuth, PhD
  role: Principal Investigator
  affiliation: University of Sheffield
  last name: Philip Powell, PhD
  role: Principal Investigator
  affiliation: University of Sheffield
  last name: Anju Keetharuth, PhD
  phone: +44 (0) 114 222 0884
  email: d.keetharuth@sheffield.ac.uk
  last name: Philip Powell, PhD
  phone: +44 (0) 114 222 0794
  email: p.a.powell@sheffield.ac.uk
  url: https://clinicaltrials.gov/study/NCT03052283?term=Development%20PROM&rank=3
  description: example of registration of PROMs
  url: https://clinicaltrials.gov/study/NCT05274867?term=Development%20PROM&rank=6
  description: example of registration of PROMs
  url: https://clinicaltrials.gov/study/NCT05315765?term=Development%20PROM&rank=8
  description: example of registration of PROMs
  responsible party type: Sponsor
  mesh term: Castleman Disease
  sharing ipd: No
  ipd description: Deanonymized transcriptions of stages 2 and 4 interviews will be securely stored in the University of Sheffield repository with access restricted to investigators only. Quantitative data from stages 3 and 4a will be processed, stored and accessed similarly in the same location.