<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/NCT03766347</url>
  </required_header>
  <id_info>
    <org_study_id>17-005618</org_study_id>
    <nct_id>NCT03766347</nct_id>
  </id_info>
  <brief_title>Pediatric NMOSD Observational Study</brief_title>
  <official_title>Pediatric Neuromyelitis Optica Spectrum Disorder (NMOSD) 1 Year Observational Study</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Mayo Clinic</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Alexion</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
  </sponsors>
  <source>Mayo Clinic</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>
      This study is being done to develop a database of pediatric patients in order to study the&#xD;
      cause, early detection and best treatment for neuromyelitis optica spectrum disorder (NMOSD)&#xD;
      in pediatric patients.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This study is being done to collect information on the natural history of NMOSD in pediatric&#xD;
      AQP4-IgG seropositive patients. A major restriction in performing drug studies in pediatric&#xD;
      patients with NMOSD is limited information on the course of the disease in these patients.&#xD;
      Collecting clinical information over the course of a 1 year observational study would inform&#xD;
      on the natural history of the disease in these patients. A repository of pediatric patients&#xD;
      with rare diseases can increase knowledge on the natural history of the specific disease,&#xD;
      assist in identifying appropriate patients fulfilling specified criteria for drug studies and&#xD;
      potentially serve as a control group.&#xD;
&#xD;
      Timepoints: Baseline, 3mo, 6mo, 9mo, 12mo (+/- 1 mo for each time point).&#xD;
&#xD;
      Baseline data:&#xD;
&#xD;
        -  Demographics [age/sex/ethnicity],&#xD;
&#xD;
        -  Clinical presentation information including date of initial diagnosis,&#xD;
&#xD;
        -  Clinical phenotype&#xD;
&#xD;
        -  Immunotherapy used current and past,&#xD;
&#xD;
        -  Family history of autoimmune diseases,&#xD;
&#xD;
        -  Serological data results&#xD;
&#xD;
        -  Radiologic data as available&#xD;
&#xD;
      Self-Report Assessments will be:&#xD;
&#xD;
        -  Current impairment, as measured by the expanded disability status scale (EDSS) score&#xD;
           self-reported using Ratzker (1997) EDSS Self Report form,&#xD;
&#xD;
        -  Quality of life as measured by the EQ-5D and Varni's (1998) PedsQL over the&#xD;
           phone/mail/email.&#xD;
&#xD;
      At follow-up visits 3mo, 6mo, 9mo, 12mo (+/- 1 mo for each time point):&#xD;
&#xD;
        -  Attacks/relapses&#xD;
&#xD;
        -  Any hospitalizations&#xD;
&#xD;
        -  Confirm medications and update records if changes&#xD;
&#xD;
      Self-Report Assessments at follow-up will be:&#xD;
&#xD;
        -  Current impairment, as measured by the EDSS score self-reported using Ratzker (1997)&#xD;
           EDSS Self Report form,&#xD;
&#xD;
        -  Quality of life as measured by the EQ-5D and PedsQL over the phone/mail/email.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">February 1, 2018</start_date>
  <completion_date type="Anticipated">February 2024</completion_date>
  <primary_completion_date type="Anticipated">February 2024</primary_completion_date>
  <study_type>Observational [Patient Registry]</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>
  <target_duration>1 Year</target_duration>
  <primary_outcome>
    <measure>Time to relapse</measure>
    <time_frame>One year.</time_frame>
    <description>Relapse events of NMOSD defined by clinical worsening of neurological symptoms and treatment.</description>
  </primary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Anticipated">100</enrollment>
  <condition>Neuromyelitis Optica</condition>
  <condition>NMO Spectrum Disorder</condition>
  <arm_group>
    <arm_group_label>Pediatric Neuromyelitis Optica</arm_group_label>
    <description>Participants under the age of 18 that are positive for Aquaporin-4 antibody.</description>
  </arm_group>
  <eligibility>
    <study_pop>
      <textblock>
        Any pediatric patients that have tested positive for AQP4 antibody regardless of geographic&#xD;
        location.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  AQP4 positive&#xD;
&#xD;
          -  Ability to give informed consent by patient or caregiver&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
        - Inability to complete required forms via phone, mail, or email.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>2 Years</minimum_age>
    <maximum_age>17 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Sean J Pittock, M.D.</last_name>
    <role>Principal Investigator</role>
    <affiliation>Mayo Clinic</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Melissa L Bush</last_name>
    <phone>507-538-5418</phone>
    <email>bush.melissa1@mayo.edu</email>
  </overall_contact>
  <location>
    <facility>
      <name>Mayo Clinic</name>
      <address>
        <city>Rochester</city>
        <state>Minnesota</state>
        <zip>55905</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Sean J. Pittock, M.D.</last_name>
      <phone>507-266-3196</phone>
      <email>pittock.sean@mayo.edu</email>
    </contact>
    <investigator>
      <last_name>Sean J. Pittock, M.D.</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <link>
    <url>https://www.mayo.edu/research/clinical-trials</url>
    <description>Mayo Clinic Clinical Trials</description>
  </link>
  <verification_date>March 2023</verification_date>
  <study_first_submitted>December 4, 2018</study_first_submitted>
  <study_first_submitted_qc>December 4, 2018</study_first_submitted_qc>
  <study_first_posted type="Actual">December 6, 2018</study_first_posted>
  <last_update_submitted>March 27, 2023</last_update_submitted>
  <last_update_submitted_qc>March 27, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">March 28, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Mayo Clinic</investigator_affiliation>
    <investigator_full_name>Sean Pittock</investigator_full_name>
    <investigator_title>Prinicpal Investigator</investigator_title>
  </responsible_party>
  <keyword>Pediatric</keyword>
  <keyword>Aquaporin-4</keyword>
  <keyword>AQP4</keyword>
  <keyword>NMOSD</keyword>
  <keyword>NMO</keyword>
  <keyword>Neuromyelitis Optica</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Neuromyelitis Optica</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <!-- 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/NCT03766347
org study id: 17-005618
nct id: NCT03766347
lead sponsor: 
collaborator: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
This study is being done to develop a database of pediatric patients in order to study the
      cause, early detection and best treatment for neuromyelitis optica spectrum disorder (NMOSD)
      in pediatric patients.
This study is being done to collect information on the natural history of NMOSD in pediatric
      AQP4-IgG seropositive patients. A major restriction in performing drug studies in pediatric
      patients with NMOSD is limited information on the course of the disease in these patients.
      Collecting clinical information over the course of a 1 year observational study would inform
      on the natural history of the disease in these patients. A repository of pediatric patients
      with rare diseases can increase knowledge on the natural history of the specific disease,
      assist in identifying appropriate patients fulfilling specified criteria for drug studies and
      potentially serve as a control group.

      Timepoints: Baseline, 3mo, 6mo, 9mo, 12mo (+/- 1 mo for each time point).

      Baseline data:

        -  Demographics [age/sex/ethnicity],

        -  Clinical presentation information including date of initial diagnosis,

        -  Clinical phenotype

        -  Immunotherapy used current and past,

        -  Family history of autoimmune diseases,

        -  Serological data results

        -  Radiologic data as available

      Self-Report Assessments will be:

        -  Current impairment, as measured by the expanded disability status scale (EDSS) score
           self-reported using Ratzker (1997) EDSS Self Report form,

        -  Quality of life as measured by the EQ-5D and Varni's (1998) PedsQL over the
           phone/mail/email.

      At follow-up visits 3mo, 6mo, 9mo, 12mo (+/- 1 mo for each time point):

        -  Attacks/relapses

        -  Any hospitalizations

        -  Confirm medications and update records if changes

      Self-Report Assessments at follow-up will be:

        -  Current impairment, as measured by the EDSS score self-reported using Ratzker (1997)
           EDSS Self Report form,

        -  Quality of life as measured by the EQ-5D and PedsQL over the phone/mail/email.
observational model: Cohort
time perspective: Prospective
measure: Time to relapse
time frame: One year.
description: Relapse events of NMOSD defined by clinical worsening of neurological symptoms and treatment.
arm group label: Pediatric Neuromyelitis Optica
description: Participants under the age of 18 that are positive for Aquaporin-4 antibody.
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 2 Years
maximum age: 17 Years
healthy volunteers: No
last name: Sean J Pittock, M.D.
role: Principal Investigator
affiliation: Mayo Clinic
last name: Melissa L Bush
phone: 507-538-5418
email: bush.melissa1@mayo.edu
facility: 
status: Recruiting
contact: 
investigator: 
country: United States
url: https://www.mayo.edu/research/clinical-trials
description: Mayo Clinic Clinical Trials
responsible party type: Principal Investigator
investigator affiliation: Mayo Clinic
investigator full name: Sean Pittock
investigator title: Prinicpal Investigator
mesh term: Neuromyelitis Optica
sharing ipd: No