<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/NCT04438538</url>
  </required_header>
  <id_info>
    <org_study_id>275749</org_study_id>
    <nct_id>NCT04438538</nct_id>
  </id_info>
  <brief_title>Ménière's Disease Registry</brief_title>
  <acronym>MDR</acronym>
  <official_title>Risk Factors for the Development of Bilateral Ménière's Disease and the Establishment of a National Ménière's Disease Registry.</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Norfolk and Norwich University Hospitals NHS Foundation Trust</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>University of East Anglia</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Ménière's Society</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Norfolk and Norwich University Hospitals NHS Foundation Trust</source>
  <oversight_info>
    <has_dmc>Yes</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>
      Recent research has suggested that Ménière's disease may be a consequence of a number of&#xD;
      individual conditions rather than developing from a single cause. This means that determining&#xD;
      the different conditions that cause Ménière's disease will help the investigators to provide&#xD;
      effective treatments. Experience from other similar medical conditions has taught the&#xD;
      investigators that the best method to identify different causes of a condition is via a&#xD;
      process called 'clinical subtyping'.&#xD;
&#xD;
      The investigators intend to set up a large Ménière's disease database in order to allow then&#xD;
      to subtype Ménière's disease. More specifically, the proposed project aspires to achieve two&#xD;
      aims. The investigators intend to investigate a sub-type of Ménière's disease, bilateral&#xD;
      disease, i.e. both ears affected. The study hopes to identify what features predict an&#xD;
      individual developing bilateral Ménière's disease. Secondly, to test the feasibility of&#xD;
      expanding the database across the whole of the UK to involve all Ménière's disease patients.&#xD;
      This will allow many other features of Ménière's disease to be used to establish sub-types&#xD;
      and help predict the best treatment for individual patients.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Ménière's disease is an inner ear disorder characterised by recurrent episodes of spontaneous&#xD;
      vertigo, fluctuating hearing loss and tinnitus, often with a feeling of fullness in the ear.&#xD;
      The exact aetiology remains unknown. Ménière's disease is estimated to have a prevalence of&#xD;
      0.25% in the UK (around 162,000 individuals) (Tyrrell 2016) and is associated with&#xD;
      significant physical, psychological and socioeconomic morbidity (Anderson 2001, Tyrrell&#xD;
      2014).&#xD;
&#xD;
      There is a substantial lack of knowledge regarding many aspects of Ménière's disease,&#xD;
      including a fuller understanding of epidemiological aspects of the disease, aetiological&#xD;
      factors, pathogenesis, clinical course and treatment outcomes. The James Lind Alliance cites&#xD;
      Ménière's disease as the theme for four of their top ten priorities for addressing&#xD;
      uncertainties in the field of balance disorders (JLA 2018).&#xD;
&#xD;
      There is mounting unease regarding the definition of Ménière's disease as a single clinical&#xD;
      entity. Many consider it to represent the final pathway of a number of individual disease&#xD;
      processes. This is reflected by the progressive and varied development of diagnostic criteria&#xD;
      over the last few decades (Gurkov 2016). It is in the context of these observations that&#xD;
      Ménière's disease is likely to represent a heterogeneous clinical condition defined only by&#xD;
      small groups of common, but not always mandatory, symptoms. Understanding how Ménière's&#xD;
      disease may exist as a cluster of clinical sub-types is key to allowing further research into&#xD;
      its underlying pathophysiological mechanisms, and the targeting of specific treatment&#xD;
      strategies; as well as allowing a better understanding of the physiology of the inner ear&#xD;
      micro-environment (Phillips 2018).&#xD;
&#xD;
      This project will provide the initial steps necessary to set-up a UK Ménière's disease&#xD;
      registry by primarily investigating a single clinical subtype of Ménière's disease: bilateral&#xD;
      disease. The development of bilateral Ménière's disease has significant implications for&#xD;
      short and long-term opportunities for treatment and rehabilitation; this in turn has&#xD;
      significant implications for communication and employment. Previous literature on bilateral&#xD;
      disease has offered widely ranging estimates of the likelihood that it will occur in cases&#xD;
      where unilateral disease is confirmed, and no clear predictors have been identified (Huppert&#xD;
      2010). However, it is a sub-type which is relatively well-defined with standard&#xD;
      internationally agreed criteria for diagnosis.&#xD;
&#xD;
      In areas of research such as this, the implementation of bespoke data collection platforms&#xD;
      and national registries have been demonstrated to be both effective and efficient (Mandavia&#xD;
      2017). The use of national registries has many advantages over conventional data collection&#xD;
      methods. When implemented correctly, national registries can allow the appraisal of a more&#xD;
      representative sample of the target population. Furthermore, a national approach broadens the&#xD;
      opportunities both for participation among the patient population to be served by such a&#xD;
      venture, and for public engagement and dissemination activities.&#xD;
&#xD;
      A national registry would allow the identification of features at presentation that predict&#xD;
      disease course and long-term outcome, improving the understanding of disease progression,&#xD;
      treatment response, and underlying aetiology. A UK Ménière's disease registry would also&#xD;
      provide a resource for broad and far reaching research into Ménière's disease beyond that of&#xD;
      defining clinical sub-types. Registries offer other benefits to patients, carers (including&#xD;
      partners and family), researchers, clinicians and other individuals that are involved with&#xD;
      the provision of services for those affected by the condition being studied. Individual&#xD;
      hospitals and the NHS as a whole can use registry data to improve the services they offer.&#xD;
      Individual patients and carers can use registries to learn more about their condition and&#xD;
      this can lead to a better understanding of how people's lives are affected. It is anticipated&#xD;
      that this work will lead to further registry development and expansion with a broad remit of&#xD;
      aims via further awards from major research funders.&#xD;
&#xD;
      To develop the methods and infrastructure for a national registry by recruiting participants&#xD;
      from three distinct urban and rural regions within the UK (Norfolk, Leicestershire and&#xD;
      London). This will include an appraisal of a number of practical processes of recruitment,&#xD;
      such as data acquisition, data processing and data management. This will inform future stages&#xD;
      to expand the infrastructure to support a registry for widespread national use.&#xD;
&#xD;
      This project will require the retrospective collection of data from existing Ménière's&#xD;
      patients.&#xD;
&#xD;
      Potential participants with a diagnosis of probable or definite unilateral or bilateral&#xD;
      Ménière's Disease as defined by the 2015 edition of the American Academy of&#xD;
      Otolaryngology-Head and Neck Surgery (AAO-HNS) criteria (Goebel 2016) in their hospital&#xD;
      records will be identified at ENT secondary care and private clinics. Potential participants&#xD;
      must have received a diagnosis of Ménière's Disease within the previous 10 years or have&#xD;
      received a new diagnosis during the recruitment window of the study. Unilateral and bilateral&#xD;
      Ménière's disease will be defined as follows:&#xD;
&#xD;
      Unilateral Ménière's Disease&#xD;
&#xD;
      At least two episodes of vertigo each lasting at least 20 minutes, where each episode is&#xD;
      associated with aural symptoms in a single ear.&#xD;
&#xD;
      Bilateral Ménière's Disease&#xD;
&#xD;
      At least two episodes of vertigo each lasting at least 20 minutes, where each episode is&#xD;
      associated with aural symptoms in both ears.&#xD;
&#xD;
      Data will include contemporaneous and historical information regarding medical and relevant&#xD;
      social/occupational history, audiometric data, vestibular testing data, radiological data and&#xD;
      symptom specific and health related quality of life questionnaires. Clinical data will be&#xD;
      requested, collected and extracted locally and entered directly into the Study Dataset&#xD;
      managed by Norwich Clinical Trials Unit at the University of East Anglia.&#xD;
&#xD;
      Participants will be offered the option of completing the study questionnaires online or on&#xD;
      paper (for entry by a member of the research team). No additional outpatient appointments&#xD;
      outside normal care pathways will be required.&#xD;
&#xD;
      The full dataset will be composed of three components:&#xD;
&#xD;
        1. Medical history. This will be completed by the participant onto a predefined data&#xD;
           collection form - see additional document: MDR_NVPQ&#xD;
&#xD;
        2. Symptom questionnaires. These will be completed by the participant&#xD;
&#xD;
        3. Audiometric, Vestibular and Radiological test results. This will be completed by the&#xD;
           local investigator team.&#xD;
&#xD;
      The investigators anticipate that participants will spend no longer than 60 minutes to&#xD;
      complete the necessary forms and questionnaires.&#xD;
&#xD;
      Participants will be asked to complete a non-validated Ménière's disease questionnaire about&#xD;
      their Ménière's disease including demographics, history, symptoms including triggers of&#xD;
      Ménière's disease related vertigo attacks, comorbidities, current treatment and past&#xD;
      treatments tried for Ménière's disease.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">June 20, 2020</start_date>
  <completion_date type="Actual">December 31, 2021</completion_date>
  <primary_completion_date type="Actual">July 31, 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>Retrospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>To acquire data on existing Ménière's disease patients from three UK regions.</measure>
    <time_frame>20 minutes</time_frame>
    <description>Participants will be asked to complete a non-validated Ménière's disease questionnaire about their Ménière's disease including demographics, history, symptoms including triggers of Ménière's disease related vertigo attacks, comorbidities, current treatment and past treatments tried for Ménière's disease</description>
  </primary_outcome>
  <primary_outcome>
    <measure>An estimation of the prevalence and incidence of bilateral disease:</measure>
    <time_frame>through study completion, an average of 1 year</time_frame>
    <description>The investigators should identify candidate risk factors that would increase or decrease the chances of developing bilateral disease. These could be further tested in a study of incident (i.e. newly occurring) cases of Ménière's disease followed prospectively in time. However, the initial set of risk factors should allow us to start the process of characterising the bilateral subtype and further understanding it aetiology.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Putative risk factors for the conversion from unilateral to bilateral disease:</measure>
    <time_frame>through study completion, an average of 1 year</time_frame>
    <description>The proportion of individuals with unilateral Ménière's disease that develop bilateral Ménière's disease after defined periods of time. Ultimately, it would be useful if the investigators could inform patients that, for example, if they haven't developed bilateral disease after a defined number of years from referral to secondary care, their chance of developing bilateral disease is less than a defined percentage.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Development of methods and infrastructure for a national registry by recruiting participants from three distinct urban and rural regions within the UK (Norfolk, Leicestershire and London).</measure>
    <time_frame>1 year</time_frame>
    <description>This will include an appraisal of a number of practical processes of recruitment, such as data acquisition, data processing and data management. This will inform future stages to expand the infrastructure to support a registry for widespread national use.&#xD;
More generally, the process of patient identification, recruitment, data collection and management will be the start of a much larger and broader national registry.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>In conjunction with the Ménière's disease community, identify research questions which could be answered either using, or in collaboration with, a national registry.</measure>
    <time_frame>through study completion, an average of 1 year</time_frame>
    <description>In conjunction with the Ménière's disease community, identify research questions which could be answered either using, or in collaboration with, a national registry.</description>
  </secondary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Actual">410</enrollment>
  <condition>Ménière's Disease</condition>
  <arm_group>
    <arm_group_label>Patients with Dizziness</arm_group_label>
    <description>All trial participants are within this group. All trial participants will either have a diagnosis or a suspected diagnosis of Ménière's Disease in order to take part.</description>
  </arm_group>
  <eligibility>
    <study_pop>
      <textblock>
        Patients aged 18 or over with a definite or probable diagnosis of Ménière's disease. These&#xD;
        will be collated from NHS hospitals and the corresponding clinician's private clinics.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Adults aged 18 and over.&#xD;
&#xD;
          -  A probable or definite diagnosis of unilateral or bilateral Meniere's disease as&#xD;
             defined by the 2015 edition of the American Academy of Otolaryngology-Head and Neck&#xD;
             Surgery (Goebel 2016)&#xD;
&#xD;
          -  A willingness to provide consent for data from health records to be used for research&#xD;
             purposes.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Unable to provide consent&#xD;
&#xD;
          -  Unable/unwilling to complete questionnaires&#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>John Phillips, Consultant</last_name>
    <role>Principal Investigator</role>
    <affiliation>Norfolk &amp; Norwich University Hospitals NHS Foundation Trust</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Norfolk &amp; Norwich University Hospital</name>
      <address>
        <city>Norwich</city>
        <state>Norfolk</state>
        <zip>NR4 7UY</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United Kingdom</country>
  </location_countries>
  <verification_date>July 2021</verification_date>
  <study_first_submitted>February 26, 2020</study_first_submitted>
  <study_first_submitted_qc>June 16, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 19, 2020</study_first_posted>
  <last_update_submitted>December 31, 2021</last_update_submitted>
  <last_update_submitted_qc>December 31, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">January 4, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>patient reported questionnaires</keyword>
  <keyword>audiometric</keyword>
  <keyword>vestibular</keyword>
  <keyword>radiological tests</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Meniere Disease</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/NCT04438538
org study id: 275749
nct id: NCT04438538
lead sponsor: 
collaborator: 
collaborator: 
has dmc: Yes
is fda regulated drug: No
is fda regulated device: No
Recent research has suggested that Ménière's disease may be a consequence of a number of
      individual conditions rather than developing from a single cause. This means that determining
      the different conditions that cause Ménière's disease will help the investigators to provide
      effective treatments. Experience from other similar medical conditions has taught the
      investigators that the best method to identify different causes of a condition is via a
      process called 'clinical subtyping'.

      The investigators intend to set up a large Ménière's disease database in order to allow then
      to subtype Ménière's disease. More specifically, the proposed project aspires to achieve two
      aims. The investigators intend to investigate a sub-type of Ménière's disease, bilateral
      disease, i.e. both ears affected. The study hopes to identify what features predict an
      individual developing bilateral Ménière's disease. Secondly, to test the feasibility of
      expanding the database across the whole of the UK to involve all Ménière's disease patients.
      This will allow many other features of Ménière's disease to be used to establish sub-types
      and help predict the best treatment for individual patients.
Ménière's disease is an inner ear disorder characterised by recurrent episodes of spontaneous
      vertigo, fluctuating hearing loss and tinnitus, often with a feeling of fullness in the ear.
      The exact aetiology remains unknown. Ménière's disease is estimated to have a prevalence of
      0.25% in the UK (around 162,000 individuals) (Tyrrell 2016) and is associated with
      significant physical, psychological and socioeconomic morbidity (Anderson 2001, Tyrrell
      2014).

      There is a substantial lack of knowledge regarding many aspects of Ménière's disease,
      including a fuller understanding of epidemiological aspects of the disease, aetiological
      factors, pathogenesis, clinical course and treatment outcomes. The James Lind Alliance cites
      Ménière's disease as the theme for four of their top ten priorities for addressing
      uncertainties in the field of balance disorders (JLA 2018).

      There is mounting unease regarding the definition of Ménière's disease as a single clinical
      entity. Many consider it to represent the final pathway of a number of individual disease
      processes. This is reflected by the progressive and varied development of diagnostic criteria
      over the last few decades (Gurkov 2016). It is in the context of these observations that
      Ménière's disease is likely to represent a heterogeneous clinical condition defined only by
      small groups of common, but not always mandatory, symptoms. Understanding how Ménière's
      disease may exist as a cluster of clinical sub-types is key to allowing further research into
      its underlying pathophysiological mechanisms, and the targeting of specific treatment
      strategies; as well as allowing a better understanding of the physiology of the inner ear
      micro-environment (Phillips 2018).

      This project will provide the initial steps necessary to set-up a UK Ménière's disease
      registry by primarily investigating a single clinical subtype of Ménière's disease: bilateral
      disease. The development of bilateral Ménière's disease has significant implications for
      short and long-term opportunities for treatment and rehabilitation; this in turn has
      significant implications for communication and employment. Previous literature on bilateral
      disease has offered widely ranging estimates of the likelihood that it will occur in cases
      where unilateral disease is confirmed, and no clear predictors have been identified (Huppert
      2010). However, it is a sub-type which is relatively well-defined with standard
      internationally agreed criteria for diagnosis.

      In areas of research such as this, the implementation of bespoke data collection platforms
      and national registries have been demonstrated to be both effective and efficient (Mandavia
      2017). The use of national registries has many advantages over conventional data collection
      methods. When implemented correctly, national registries can allow the appraisal of a more
      representative sample of the target population. Furthermore, a national approach broadens the
      opportunities both for participation among the patient population to be served by such a
      venture, and for public engagement and dissemination activities.

      A national registry would allow the identification of features at presentation that predict
      disease course and long-term outcome, improving the understanding of disease progression,
      treatment response, and underlying aetiology. A UK Ménière's disease registry would also
      provide a resource for broad and far reaching research into Ménière's disease beyond that of
      defining clinical sub-types. Registries offer other benefits to patients, carers (including
      partners and family), researchers, clinicians and other individuals that are involved with
      the provision of services for those affected by the condition being studied. Individual
      hospitals and the NHS as a whole can use registry data to improve the services they offer.
      Individual patients and carers can use registries to learn more about their condition and
      this can lead to a better understanding of how people's lives are affected. It is anticipated
      that this work will lead to further registry development and expansion with a broad remit of
      aims via further awards from major research funders.

      To develop the methods and infrastructure for a national registry by recruiting participants
      from three distinct urban and rural regions within the UK (Norfolk, Leicestershire and
      London). This will include an appraisal of a number of practical processes of recruitment,
      such as data acquisition, data processing and data management. This will inform future stages
      to expand the infrastructure to support a registry for widespread national use.

      This project will require the retrospective collection of data from existing Ménière's
      patients.

      Potential participants with a diagnosis of probable or definite unilateral or bilateral
      Ménière's Disease as defined by the 2015 edition of the American Academy of
      Otolaryngology-Head and Neck Surgery (AAO-HNS) criteria (Goebel 2016) in their hospital
      records will be identified at ENT secondary care and private clinics. Potential participants
      must have received a diagnosis of Ménière's Disease within the previous 10 years or have
      received a new diagnosis during the recruitment window of the study. Unilateral and bilateral
      Ménière's disease will be defined as follows:

      Unilateral Ménière's Disease

      At least two episodes of vertigo each lasting at least 20 minutes, where each episode is
      associated with aural symptoms in a single ear.

      Bilateral Ménière's Disease

      At least two episodes of vertigo each lasting at least 20 minutes, where each episode is
      associated with aural symptoms in both ears.

      Data will include contemporaneous and historical information regarding medical and relevant
      social/occupational history, audiometric data, vestibular testing data, radiological data and
      symptom specific and health related quality of life questionnaires. Clinical data will be
      requested, collected and extracted locally and entered directly into the Study Dataset
      managed by Norwich Clinical Trials Unit at the University of East Anglia.

      Participants will be offered the option of completing the study questionnaires online or on
      paper (for entry by a member of the research team). No additional outpatient appointments
      outside normal care pathways will be required.

      The full dataset will be composed of three components:

        1. Medical history. This will be completed by the participant onto a predefined data
           collection form - see additional document: MDR_NVPQ

        2. Symptom questionnaires. These will be completed by the participant

        3. Audiometric, Vestibular and Radiological test results. This will be completed by the
           local investigator team.

      The investigators anticipate that participants will spend no longer than 60 minutes to
      complete the necessary forms and questionnaires.

      Participants will be asked to complete a non-validated Ménière's disease questionnaire about
      their Ménière's disease including demographics, history, symptoms including triggers of
      Ménière's disease related vertigo attacks, comorbidities, current treatment and past
      treatments tried for Ménière's disease.
observational model: Cohort
time perspective: Retrospective
measure: To acquire data on existing Ménière's disease patients from three UK regions.
time frame: 20 minutes
description: Participants will be asked to complete a non-validated Ménière's disease questionnaire about their Ménière's disease including demographics, history, symptoms including triggers of Ménière's disease related vertigo attacks, comorbidities, current treatment and past treatments tried for Ménière's disease
measure: An estimation of the prevalence and incidence of bilateral disease:
time frame: through study completion, an average of 1 year
description: The investigators should identify candidate risk factors that would increase or decrease the chances of developing bilateral disease. These could be further tested in a study of incident (i.e. newly occurring) cases of Ménière's disease followed prospectively in time. However, the initial set of risk factors should allow us to start the process of characterising the bilateral subtype and further understanding it aetiology.
measure: Putative risk factors for the conversion from unilateral to bilateral disease:
time frame: through study completion, an average of 1 year
description: The proportion of individuals with unilateral Ménière's disease that develop bilateral Ménière's disease after defined periods of time. Ultimately, it would be useful if the investigators could inform patients that, for example, if they haven't developed bilateral disease after a defined number of years from referral to secondary care, their chance of developing bilateral disease is less than a defined percentage.
measure: Development of methods and infrastructure for a national registry by recruiting participants from three distinct urban and rural regions within the UK (Norfolk, Leicestershire and London).
time frame: 1 year
description: This will include an appraisal of a number of practical processes of recruitment, such as data acquisition, data processing and data management. This will inform future stages to expand the infrastructure to support a registry for widespread national use.
More generally, the process of patient identification, recruitment, data collection and management will be the start of a much larger and broader national registry.
measure: In conjunction with the Ménière's disease community, identify research questions which could be answered either using, or in collaboration with, a national registry.
time frame: through study completion, an average of 1 year
description: In conjunction with the Ménière's disease community, identify research questions which could be answered either using, or in collaboration with, a national registry.
arm group label: Patients with Dizziness
description: All trial participants are within this group. All trial participants will either have a diagnosis or a suspected diagnosis of Ménière's Disease in order to take part.
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: John Phillips, Consultant
role: Principal Investigator
affiliation: Norfolk & Norwich University Hospitals NHS Foundation Trust
facility: 
country: United Kingdom
responsible party type: Sponsor
mesh term: Meniere Disease