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/NCT06007521</url>
    </required_header>
    <id_info>
      <org_study_id>69HCL23_0804</org_study_id>
      <nct_id>NCT06007521</nct_id>
    </id_info>
    <brief_title>Validation of a Clinical Assessment Scale Specific to Alternating Hemiplegia</brief_title>
    <acronym>TREAT-AHC</acronym>
    <official_title>Validation of a Clinical Assessment Scale Specific to Alternating Hemiplegia</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Hospices Civils de Lyon</agency>
        <agency_class>Other</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>Hospices Civils de Lyon</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>
        Alternating Hemiplegia of Childhood (AHC) is a very rare neurological disorder of genetic&#xD;
        origin, combining paroxysmal episodes and neurodevelopmental impairment. The disease is&#xD;
        generally sporadic, and its incidence has been estimated at 1 in 100,000 births, with around&#xD;
        500 cases published worldwide. The true prevalence of AHC may be underestimated due to a lack&#xD;
        of understanding of the disease and a high degree of phenotypic heterogeneity.&#xD;
  &#xD;
        It should be noted that, to date, it is difficult to assess the clinical characteristics of&#xD;
        AHC patients in a homogeneous way. The lack of disease-specific clinical outcome measures&#xD;
        therefore constitutes a critical node for advancing AHC research. In addition, recognition of&#xD;
        the pattern of movement disorders and the ability to classify their severity are very&#xD;
        important and useful for the clinician. Homogeneous disease assessment will help plan&#xD;
        rehabilitative and pharmacological interventions, facilitate monitoring of treatment&#xD;
        outcomes, and predict prognosis.&#xD;
  &#xD;
        This is why an AHC assessment scale have been developed in collaboration with clinical teams&#xD;
        from the IAHCRC consortium.&#xD;
  &#xD;
        The scale has already been tested and validated on Italian and Spanish patients. The aim of&#xD;
        this study is therefore to validate the use of this same scale on our French population of&#xD;
        AHC patients. The hypothesis of the study is that the practical use of this scale specific to&#xD;
        alternating hemiplegia is reproducible. To this end, all the items on the scale will be&#xD;
        scored independently for each patient by 4 neuropediatricians with expertise in the disease,&#xD;
        in order to observe the reproducibility of the results obtained with this scale. The scale&#xD;
        will be used for twenty AHC patients of all ages.&#xD;
      </textblock>
    </brief_summary>
    <overall_status>Not yet recruiting</overall_status>
    <start_date type="Anticipated">October 1, 2023</start_date>
    <completion_date type="Anticipated">October 1, 2024</completion_date>
    <primary_completion_date type="Anticipated">October 1, 2024</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>Composite primary endpoint : - missing data from the scale &lt;5 - mean scores compared between the 4 neurologists are close to the scale's mid-point, i.e. floor and ceiling effects are &lt; 20%, and skewness statistics are between 21 and 11</measure>
      <time_frame>At inclusion</time_frame>
      <description>All statistical tests will be performed using SPSS version 24.0 statistical software, and P &lt; 0.05 will be considered significant.&#xD;
  Validation of the use of the scale based on the results obtained independently by the 4 expert neurologists using a composite primary endpoint consisting of three criteria:&#xD;
  missing data from the scale &lt;5&#xD;
  mean scores compared between the 4 neurologists are close to the scale's mid-point, i.e. floor and ceiling effects are &lt; 20%, and skewness statistics are between 21 and 11.</description>
    </primary_outcome>
    <number_of_groups>1</number_of_groups>
    <enrollment type="Anticipated">20</enrollment>
    <condition>Alternating Hemiplegia</condition>
    <arm_group>
      <arm_group_label>Patients with alternating hemiplegia</arm_group_label>
      <description>Patients with alternating hemiplegia, from 0 to 99 years old</description>
    </arm_group>
    <intervention>
      <intervention_type>Other</intervention_type>
      <intervention_name>interview with neurologist</intervention_name>
      <description>interview with neurologist</description>
      <arm_group_label>Patients with alternating hemiplegia</arm_group_label>
    </intervention>
    <eligibility>
      <study_pop>
        <textblock>
          Patients likely to participate in the study will be identified within the ESEFNP Department&#xD;
          - Clinical Epileptology, Sleep Disorders, Functional Child Neurology, Hôpital Femme Mère&#xD;
          Enfant, (HCL) as part of their regular medical follow-up. 20 AHC patients will be included&#xD;
          in this study.&#xD;
        </textblock>
      </study_pop>
      <sampling_method>Probability Sample</sampling_method>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
            -  Patient with confirmed diagnosis of AHC, unrestricted age range 0-99 years&#xD;
  &#xD;
            -  At least one parent and/or legal guardian who understands and speaks the national&#xD;
               language&#xD;
  &#xD;
          Adult patients :&#xD;
  &#xD;
            -  Gathering of the non-opposition of the patient and/or his legal representative&#xD;
  &#xD;
          Minor patients :&#xD;
  &#xD;
            -  Collection of non-opposition from parents/guardians&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            -  Patient's refusal to participate in the study&#xD;
  &#xD;
            -  Patient under court protection, unable to give consent&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>0 Years</minimum_age>
      <maximum_age>99 Years</maximum_age>
    </eligibility>
    <overall_contact>
      <last_name>Eleni PANAGIOTAKAKI, Dr</last_name>
      <phone>04.27.85.60.60</phone>
      <phone_ext>+33</phone_ext>
      <email>eleni.panagiotakaki@chu-lyon.fr</email>
    </overall_contact>
    <overall_contact_backup>
      <last_name>Maria PAPADOPOULOU, Dr</last_name>
      <phone>04.27.85.60.40</phone>
      <phone_ext>+33</phone_ext>
      <email>maria.papadopoulou@chu-lyon.fr</email>
    </overall_contact_backup>
    <location>
      <facility>
        <name>Service d'Epileptologie Clinique, des Troubles du Sommeil et de Neurologie Fonctionnelle de l'Enfant</name>
        <address>
          <city>Bron</city>
          <state>Rhone</state>
          <zip>69500</zip>
          <country>France</country>
        </address>
      </facility>
      <contact>
        <last_name>Eleni PANAGIOTAKAKI, Dr</last_name>
        <phone>04.27.85.60.60</phone>
        <phone_ext>+33</phone_ext>
        <email>eleni.panagiotakaki@chu-lyon.fr</email>
      </contact>
      <contact_backup>
        <last_name>Maria PAPADOPOULOU, Dr</last_name>
        <phone>04.27.85.60.40</phone>
        <phone_ext>+33</phone_ext>
        <email>maria.papadopoulou@chu-lyon.fr</email>
      </contact_backup>
      <investigator>
        <last_name>Eleni PANAGIOTAKAKI, Dr</last_name>
        <role>Principal Investigator</role>
      </investigator>
    </location>
    <location_countries>
      <country>France</country>
    </location_countries>
    <verification_date>August 2023</verification_date>
    <study_first_submitted>August 8, 2023</study_first_submitted>
    <study_first_submitted_qc>August 21, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">August 23, 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 23, 2023</last_update_posted>
    <responsible_party>
      <responsible_party_type>Sponsor</responsible_party_type>
    </responsible_party>
    <keyword>Alternating Hemiplegia of Childhood</keyword>
    <keyword>rating scale</keyword>
    <condition_browse>
      <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
      <mesh_term>Hemiplegia</mesh_term>
    </condition_browse>
    <patient_data>
      <sharing_ipd>No</sharing_ipd>
    </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/NCT06007521
  org study id: 69HCL23_0804
  nct id: NCT06007521
  lead sponsor: 
  has dmc: No
  is fda regulated drug: No
  is fda regulated device: No
  Alternating Hemiplegia of Childhood (AHC) is a very rare neurological disorder of genetic
        origin, combining paroxysmal episodes and neurodevelopmental impairment. The disease is
        generally sporadic, and its incidence has been estimated at 1 in 100,000 births, with around
        500 cases published worldwide. The true prevalence of AHC may be underestimated due to a lack
        of understanding of the disease and a high degree of phenotypic heterogeneity.
  
        It should be noted that, to date, it is difficult to assess the clinical characteristics of
        AHC patients in a homogeneous way. The lack of disease-specific clinical outcome measures
        therefore constitutes a critical node for advancing AHC research. In addition, recognition of
        the pattern of movement disorders and the ability to classify their severity are very
        important and useful for the clinician. Homogeneous disease assessment will help plan
        rehabilitative and pharmacological interventions, facilitate monitoring of treatment
        outcomes, and predict prognosis.
  
        This is why an AHC assessment scale have been developed in collaboration with clinical teams
        from the IAHCRC consortium.
  
        The scale has already been tested and validated on Italian and Spanish patients. The aim of
        this study is therefore to validate the use of this same scale on our French population of
        AHC patients. The hypothesis of the study is that the practical use of this scale specific to
        alternating hemiplegia is reproducible. To this end, all the items on the scale will be
        scored independently for each patient by 4 neuropediatricians with expertise in the disease,
        in order to observe the reproducibility of the results obtained with this scale. The scale
        will be used for twenty AHC patients of all ages.
  observational model: Case-Only
  time perspective: Prospective
  measure: Composite primary endpoint : - missing data from the scale <5 - mean scores compared between the 4 neurologists are close to the scale's mid-point, i.e. floor and ceiling effects are < 20%, and skewness statistics are between 21 and 11
  time frame: At inclusion
  description: All statistical tests will be performed using SPSS version 24.0 statistical software, and P < 0.05 will be considered significant.
  Validation of the use of the scale based on the results obtained independently by the 4 expert neurologists using a composite primary endpoint consisting of three criteria:
  missing data from the scale <5
  mean scores compared between the 4 neurologists are close to the scale's mid-point, i.e. floor and ceiling effects are < 20%, and skewness statistics are between 21 and 11.
  arm group label: Patients with alternating hemiplegia
  description: Patients with alternating hemiplegia, from 0 to 99 years old
  intervention type: Other
  intervention name: interview with neurologist
  description: interview with neurologist
  arm group label: Patients with alternating hemiplegia
  study pop: 
  sampling method: Probability Sample
  criteria: 
  gender: All
  minimum age: 0 Years
  maximum age: 99 Years
  last name: Eleni PANAGIOTAKAKI, Dr
  phone: 04.27.85.60.60
  phone ext: +33
  email: eleni.panagiotakaki@chu-lyon.fr
  last name: Maria PAPADOPOULOU, Dr
  phone: 04.27.85.60.40
  phone ext: +33
  email: maria.papadopoulou@chu-lyon.fr
  facility: 
  contact: 
  contact backup: 
  investigator: 
  country: France
  responsible party type: Sponsor
  mesh term: Hemiplegia
  sharing ipd: No