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/NCT06011226</url>
    </required_header>
    <id_info>
      <org_study_id>APHP220733</org_study_id>
      <secondary_id>2022-A01816-37</secondary_id>
      <nct_id>NCT06011226</nct_id>
    </id_info>
    <brief_title>Development of a Patient-reported Outcome Measure for Chiari Malformation and Syringomyelia</brief_title>
    <acronym>BCS-score</acronym>
    <official_title>Development of a Patient-reported Outcome Measure for Chiari Malformation and Syringomyelia</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Assistance Publique - Hôpitaux de Paris</agency>
        <agency_class>Other</agency_class>
      </lead_sponsor>
      <collaborator>
        <agency>Institut National de la Santé Et de la Recherche Médicale, France</agency>
        <agency_class>Other</agency_class>
      </collaborator>
    </sponsors>
    <source>Assistance Publique - Hôpitaux de Paris</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>
        Chiari malformation corresponds to the herniation of cerebellar tonsils into the foramen&#xD;
        magnum resulting in obstruction of cerebrospinal fluid circulation, which may eventually lead&#xD;
        to the formation of an intramedullary cavity called syringomyelia.&#xD;
  &#xD;
        Chiari and syringomyelia can be responsible of variable symptoms, based on which&#xD;
        neurosurgeons might propose surgical treatment. Yet, there is no properly developped and&#xD;
        validated patient reported outcome measure (PROM) to assess the clinical severity of Chiari&#xD;
        malformation and/or syringomyelia. The lack of such evaluation tool is a major issue to&#xD;
        determine the optimal therapeutic strategy and to achieve a standardized and reproducible&#xD;
        follow-up.&#xD;
      </textblock>
    </brief_summary>
    <detailed_description>
      <textblock>
        Chiari malformation corresponds to the herniation of cerebellar tonsils into the foramen&#xD;
        magnum resulting in obstruction of cerebrospinal fluid circulation, which may eventually lead&#xD;
        to the formation of an intramedullary cavity called syringomyelia.&#xD;
  &#xD;
        Chiari and syringomyelia can be responsible of variable symptoms, based on which&#xD;
        neurosurgeons might propose surgical treatment. Yet, there is no properly developped and&#xD;
        validated patient reported outcome measure (PROM) to assess the clinical severity of Chiari&#xD;
        malformation and/or syringomyelia. The lack of such evaluation tool is a major issue to&#xD;
        determine the optimal therapeutic strategy and to achieve a standardized and reproducible&#xD;
        follow-up. The project will consist of two successive phases: a survey design of the&#xD;
        questionnaire with small selected groups of patients; a monocentric pilot study on a limited&#xD;
        population; The purpose of the development study is to produce a first version of the PROM&#xD;
        based on proposed items written by experts and tested with volunteer patients (n = 10-20)&#xD;
        within the framework of &quot; focus groups&quot; followed by cognitive debriefings. These items will&#xD;
        explore various dimensions of the functional impact of Chiari malformation and syringomyelia&#xD;
        (pain, motor disability, sphincter disorders, quality of life, etc.). The pilot study will&#xD;
        test this 1st version of the questionnaire on a limited population of patients (n = 40)&#xD;
        within the CRMR C-MAVEM of Bicêtre hospital to produce a final version. This work will be&#xD;
        supplemented subsequently by a multicenter study allowing to validate a simple and&#xD;
        reproducible evaluation tool in order to ensure the follow-up of patients with a Chiari&#xD;
        malformation and/or syringomyelia and to measure surgical outcome.&#xD;
      </textblock>
    </detailed_description>
    <overall_status>Not yet recruiting</overall_status>
    <start_date type="Anticipated">September 2023</start_date>
    <completion_date type="Anticipated">December 2026</completion_date>
    <primary_completion_date type="Anticipated">December 2025</primary_completion_date>
    <study_type>Observational</study_type>
    <has_expanded_access>No</has_expanded_access>
    <study_design_info>
      <observational_model>Case-Only</observational_model>
      <time_perspective>Prospective</time_perspective>
    </study_design_info>
    <primary_outcome>
      <measure>development of metrologic and psychometric features of a patient-reported outcome measure (Bicêtre Chiari and Syringomyelia score - BCS score)</measure>
      <time_frame>throughout the study (an average of 26 months)</time_frame>
      <description>Development of metrologic and psychometric features of a patient-reported outcome measure (Bicêtre Chiari and Syringomyelia score - BCS score) for evaluating functional severity of Chiari malformation and/or syringomyelia</description>
    </primary_outcome>
    <primary_outcome>
      <measure>validation of metrologic and psychometric features of a patient-reported outcome</measure>
      <time_frame>throughout the study (an average of 26 months)</time_frame>
      <description>validation of metrologic and psychometric features of a patient-reported outcome measure (Bicêtre Chiari and Syringomyelia score - BCS score) for evaluating functional severity of Chiari malformation and/or syringomyelia</description>
    </primary_outcome>
    <secondary_outcome>
      <measure>validation of sub-scores</measure>
      <time_frame>throughout the study (an average of 26 months)</time_frame>
      <description>Validation of two sub-scores for isolated Chiari malformation and isolated syringomyelia</description>
    </secondary_outcome>
    <number_of_groups>3</number_of_groups>
    <enrollment type="Anticipated">60</enrollment>
    <condition>Syringomyelia</condition>
    <condition>Chiari Malformation</condition>
    <arm_group>
      <arm_group_label>Phase 1: development of the questionnaire</arm_group_label>
      <description>development of the patient reported outcome: The strategy consists of four phases:&#xD;
  structured litterature review&#xD;
  Group of experts: identification of the main areas to be covered by the questionnaire in order to draw up the focus group moderation guide&#xD;
  focus groups: A discussion session will explore patient symptomatology, the functional impact of the disease, quality of life and the problems faced by patients from their point of view.</description>
    </arm_group>
    <arm_group>
      <arm_group_label>Phase 2: pilot phase</arm_group_label>
      <description>Once the initial version of the self-questionnaire has been developed, it will be tested on a group of 40 patients with either Chiari malformation with syringomyelia (n = 20), isolated Chiari malformation (n = 10) or isolated syringomyelia (n = 10), managed by the CRMR C-MAVEM at Bicêtre Hospital.</description>
    </arm_group>
    <arm_group>
      <arm_group_label>Phase 3: national testing of the questionnaire</arm_group_label>
      <description>the 3rd phase will be submitted to the Human Subjects Protection Review Board according to the progression of the first two phases.</description>
    </arm_group>
    <intervention>
      <intervention_type>Other</intervention_type>
      <intervention_name>focus groups</intervention_name>
      <description>A dozen patients with Chiari malformation and syringomyelia (n = 4) or isolated Chiari malformation (n = 4) or isolated syringomyelia (n = 4) will be brought together in at least two focus groups. Each focus group will include 5 to 8 patients and will last approximately 1 to 2 hours. The focus groups will be recorded (audio only, no personal data will be collected during these sessions) and will be conducted in the presence of a neurosurgeon and a psychologist, who will moderate the session.</description>
      <arm_group_label>Phase 1: development of the questionnaire</arm_group_label>
    </intervention>
    <intervention>
      <intervention_type>Other</intervention_type>
      <intervention_name>questionnaire</intervention_name>
      <description>developed questionnaire will be proposed to the patients in phase 2 and 3</description>
      <arm_group_label>Phase 2: pilot phase</arm_group_label>
      <arm_group_label>Phase 3: national testing of the questionnaire</arm_group_label>
    </intervention>
    <eligibility>
      <study_pop>
        <textblock>
          The development of a questionnaire for self-assessment of the clinical impact of a&#xD;
          pathology necessarily requires the involvement of the patients concerned. In particular, it&#xD;
          is important that the clinical heterogeneity of the patients concerned is represented. The&#xD;
          study population will therefore be patients with Chiari malformation, whether or not they&#xD;
          have associated syringomyelia, and whether or not they have foraminal syringomyelia. They&#xD;
          will be recruited via neurosurgical centers belonging to the C-MAVEM (Chiari, syringomyelia&#xD;
          and vertebro-medullary malformations) network of rare disease reference centers for Chiari&#xD;
          malformations and syringomyelia, and with the help of the main patient association&#xD;
          (APAISER).&#xD;
        </textblock>
      </study_pop>
      <sampling_method>Probability Sample</sampling_method>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
          Criteria common to all subjects included in the study&#xD;
  &#xD;
            -  Age &gt; 18 years&#xD;
  &#xD;
            -  Subject understanding French Patients included in Phase I&#xD;
  &#xD;
            -  Subject with signed informed consent Patients included in phase II&#xD;
  &#xD;
            -  Patient not opposed to study participation&#xD;
  &#xD;
          Patients Chiari only group:&#xD;
  &#xD;
            -  Isolated descent of cerebellar tonsils &gt; 5 mm below McRae's line (no associated&#xD;
               syringomyelia)&#xD;
  &#xD;
          Patients syringomyelia only group:&#xD;
  &#xD;
            -  Presence of an intramedullary fluid cavity secondary to circulatory disturbance of&#xD;
               cerebrospinal fluid of non foraminal origin (no Chiari)&#xD;
  &#xD;
          Patients Chiari with Syringomyelia group:&#xD;
  &#xD;
            -  Presence of Chiari malformation (tonsils &gt; 5 mm below McRae's line) AND foraminal&#xD;
               syringomyelia.&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            -  Insufficient command of French&#xD;
  &#xD;
            -  Minor or protected adult (guardianship, curatorship, safeguard of justice)&#xD;
  &#xD;
            -  Diagnosis of Chiari and/or syringomyelia not proven by an MRI scan&#xD;
  &#xD;
            -  Pregnant or breast-feeding&#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>Steven KNAFO</last_name>
      <role>Principal Investigator</role>
      <affiliation>Assistance Publique - Hôpitaux de Paris, Hôpital Bicêtre</affiliation>
    </overall_official>
    <overall_contact>
      <last_name>Steven KNAFO, MD</last_name>
      <phone>01 45 21 22 88</phone>
      <phone_ext>+33</phone_ext>
      <email>steven.knafo@aphp.fr</email>
    </overall_contact>
    <location>
      <facility>
        <name>Hôpital Bicêtre - Service de Neurochirurgie - CRMR C-MAVEM</name>
        <address>
          <city>Le Kremlin-Bicêtre</city>
          <zip>94270</zip>
          <country>France</country>
        </address>
      </facility>
      <contact>
        <last_name>Steven KNAFO</last_name>
        <phone>01 45 21 22 88</phone>
        <phone_ext>+33</phone_ext>
        <email>steven.knafo@aphp.fr</email>
      </contact>
    </location>
    <location_countries>
      <country>France</country>
    </location_countries>
    <verification_date>July 2023</verification_date>
    <study_first_submitted>August 21, 2023</study_first_submitted>
    <study_first_submitted_qc>August 21, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">August 25, 2023</study_first_posted>
    <last_update_submitted>August 21, 2023</last_update_submitted>
    <last_update_submitted_qc>August 21, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">August 25, 2023</last_update_posted>
    <responsible_party>
      <responsible_party_type>Sponsor</responsible_party_type>
    </responsible_party>
    <keyword>Syringomyelia</keyword>
    <keyword>Chiari Malformation</keyword>
    <keyword>patient-reported outcome measure</keyword>
    <condition_browse>
      <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
      <mesh_term>Arnold-Chiari Malformation</mesh_term>
      <mesh_term>Syringomyelia</mesh_term>
      <mesh_term>Congenital Abnormalities</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/NCT06011226
  org study id: APHP220733
  secondary id: 2022-A01816-37
  nct id: NCT06011226
  lead sponsor: 
  collaborator: 
  has dmc: No
  is fda regulated drug: No
  is fda regulated device: No
  Chiari malformation corresponds to the herniation of cerebellar tonsils into the foramen
        magnum resulting in obstruction of cerebrospinal fluid circulation, which may eventually lead
        to the formation of an intramedullary cavity called syringomyelia.
  
        Chiari and syringomyelia can be responsible of variable symptoms, based on which
        neurosurgeons might propose surgical treatment. Yet, there is no properly developped and
        validated patient reported outcome measure (PROM) to assess the clinical severity of Chiari
        malformation and/or syringomyelia. The lack of such evaluation tool is a major issue to
        determine the optimal therapeutic strategy and to achieve a standardized and reproducible
        follow-up.
  Chiari malformation corresponds to the herniation of cerebellar tonsils into the foramen
        magnum resulting in obstruction of cerebrospinal fluid circulation, which may eventually lead
        to the formation of an intramedullary cavity called syringomyelia.
  
        Chiari and syringomyelia can be responsible of variable symptoms, based on which
        neurosurgeons might propose surgical treatment. Yet, there is no properly developped and
        validated patient reported outcome measure (PROM) to assess the clinical severity of Chiari
        malformation and/or syringomyelia. The lack of such evaluation tool is a major issue to
        determine the optimal therapeutic strategy and to achieve a standardized and reproducible
        follow-up. The project will consist of two successive phases: a survey design of the
        questionnaire with small selected groups of patients; a monocentric pilot study on a limited
        population; The purpose of the development study is to produce a first version of the PROM
        based on proposed items written by experts and tested with volunteer patients (n = 10-20)
        within the framework of " focus groups" followed by cognitive debriefings. These items will
        explore various dimensions of the functional impact of Chiari malformation and syringomyelia
        (pain, motor disability, sphincter disorders, quality of life, etc.). The pilot study will
        test this 1st version of the questionnaire on a limited population of patients (n = 40)
        within the CRMR C-MAVEM of Bicêtre hospital to produce a final version. This work will be
        supplemented subsequently by a multicenter study allowing to validate a simple and
        reproducible evaluation tool in order to ensure the follow-up of patients with a Chiari
        malformation and/or syringomyelia and to measure surgical outcome.
  observational model: Case-Only
  time perspective: Prospective
  measure: development of metrologic and psychometric features of a patient-reported outcome measure (Bicêtre Chiari and Syringomyelia score - BCS score)
  time frame: throughout the study (an average of 26 months)
  description: Development of metrologic and psychometric features of a patient-reported outcome measure (Bicêtre Chiari and Syringomyelia score - BCS score) for evaluating functional severity of Chiari malformation and/or syringomyelia
  measure: validation of metrologic and psychometric features of a patient-reported outcome
  time frame: throughout the study (an average of 26 months)
  description: validation of metrologic and psychometric features of a patient-reported outcome measure (Bicêtre Chiari and Syringomyelia score - BCS score) for evaluating functional severity of Chiari malformation and/or syringomyelia
  measure: validation of sub-scores
  time frame: throughout the study (an average of 26 months)
  description: Validation of two sub-scores for isolated Chiari malformation and isolated syringomyelia
  arm group label: Phase 1: development of the questionnaire
  description: development of the patient reported outcome: The strategy consists of four phases:
  structured litterature review
  Group of experts: identification of the main areas to be covered by the questionnaire in order to draw up the focus group moderation guide
  focus groups: A discussion session will explore patient symptomatology, the functional impact of the disease, quality of life and the problems faced by patients from their point of view.
  arm group label: Phase 2: pilot phase
  description: Once the initial version of the self-questionnaire has been developed, it will be tested on a group of 40 patients with either Chiari malformation with syringomyelia (n = 20), isolated Chiari malformation (n = 10) or isolated syringomyelia (n = 10), managed by the CRMR C-MAVEM at Bicêtre Hospital.
  arm group label: Phase 3: national testing of the questionnaire
  description: the 3rd phase will be submitted to the Human Subjects Protection Review Board according to the progression of the first two phases.
  intervention type: Other
  intervention name: focus groups
  description: A dozen patients with Chiari malformation and syringomyelia (n = 4) or isolated Chiari malformation (n = 4) or isolated syringomyelia (n = 4) will be brought together in at least two focus groups. Each focus group will include 5 to 8 patients and will last approximately 1 to 2 hours. The focus groups will be recorded (audio only, no personal data will be collected during these sessions) and will be conducted in the presence of a neurosurgeon and a psychologist, who will moderate the session.
  arm group label: Phase 1: development of the questionnaire
  intervention type: Other
  intervention name: questionnaire
  description: developed questionnaire will be proposed to the patients in phase 2 and 3
  arm group label: Phase 2: pilot phase
  arm group label: Phase 3: national testing of the questionnaire
  study pop: 
  sampling method: Probability Sample
  criteria: 
  gender: All
  minimum age: 18 Years
  maximum age: N/A
  healthy volunteers: No
  last name: Steven KNAFO
  role: Principal Investigator
  affiliation: Assistance Publique - Hôpitaux de Paris, Hôpital Bicêtre
  last name: Steven KNAFO, MD
  phone: 01 45 21 22 88
  phone ext: +33
  email: steven.knafo@aphp.fr
  facility: 
  contact: 
  country: France
  responsible party type: Sponsor
  mesh term: Arnold-Chiari Malformation
  mesh term: Syringomyelia
  mesh term: Congenital Abnormalities