<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/NCT04406636</url>
  </required_header>
  <id_info>
    <org_study_id>TTX N1</org_study_id>
    <nct_id>NCT04406636</nct_id>
  </id_info>
  <brief_title>Natural History Study in Huntington Disease Gene Expansion Carriers (HDGECs) - SHIELD HD</brief_title>
  <acronym>SHIELD HD</acronym>
  <official_title>Natural History Study in Prodromal and Manifest Huntington Disease Gene Expansion Carriers (HDGECs) - SHIELD HD</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>CHDI Foundation, Inc.</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Medpace, Inc.</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
  </sponsors>
  <source>CHDI Foundation, Inc.</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>
      SHIELD HD is an international, multisite, prospective, longitudinal cohort natural history&#xD;
      study to assess the natural history of HD and its biomarkers that are associated with&#xD;
      modulation of the number of cytosine-adenine-guanine (CAG) repeats in the mutant Huntingtin&#xD;
      (HTT) gene.&#xD;
&#xD;
      Approximately 60 patients will be enrolled into the study and followed for up to 24 months at&#xD;
      clinical sites in North America and Europe.&#xD;
&#xD;
      The results of this study will inform assessments for a future interventional treatment&#xD;
      trial.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      The rationale for this study is to obtain longitudinal information related to Somatic&#xD;
      Instability and DNA damage response genes in HDGECs at various stages of the disease.&#xD;
      Established assessments of disease progression will also be recorded.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Active, not recruiting</overall_status>
  <start_date type="Actual">May 19, 2020</start_date>
  <completion_date type="Anticipated">April 2023</completion_date>
  <primary_completion_date type="Anticipated">April 2023</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>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>DDR gene expression</measure>
    <time_frame>2 years</time_frame>
    <description>To assess deoxyribonucleic acid (DNA) damage repair (DDR) gene expression in accessible biofluids and disease trajectories for established and novel biomarkers and clinical outcomes.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Compare rates of change in biomarkers for disease progression</measure>
    <time_frame>2 years</time_frame>
    <description>To compare the rates of change for different outcomes and cytosine adenine guanine (CAG) age product (CAP) Scores.</description>
  </secondary_outcome>
  <other_outcome>
    <measure>Additional biomarkers to be examined</measure>
    <time_frame>2 years</time_frame>
    <description>The exploratory objectives of this study are to be determined and may include the examination of additional biomarkers present in CSF, plasma, and whole blood, including but not limited to mutant HTT (mHTT) protein, cytokines, and others, as well as clinical markers of progression.</description>
  </other_outcome>
  <enrollment type="Actual">70</enrollment>
  <condition>Huntington Disease</condition>
  <eligibility>
    <study_pop>
      <textblock>
        Huntington disease (HD), genetically confirmed by direct DNA testing, either obtained&#xD;
        previously or performed at Screening&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Key Inclusion Criteria&#xD;
&#xD;
        Patients who meet all of the following criteria will be eligible to participate in the&#xD;
        study:&#xD;
&#xD;
          1. Capacity to comprehend the study objectives and procedures&#xD;
&#xD;
          2. Documentation of genetically confirmed disease by direct DNA testing, defined as a CAG&#xD;
             repeat length ≥39 in the HTT gene&#xD;
&#xD;
          3. Ability to undergo and tolerate MRI scans&#xD;
&#xD;
          4. Ability to tolerate blood draws and lumbar punctures&#xD;
&#xD;
        Key Exclusion Criteria&#xD;
&#xD;
        Patients who meet any of the following criteria will be excluded from participation in the&#xD;
        study:&#xD;
&#xD;
          1. Any conditions, including severe chorea and dementia, that would prevent either&#xD;
             writing or performing pen and paper, tablet, or computer based tasks as determined by&#xD;
             the Investigator&#xD;
&#xD;
          2. Treatment with an investigational drug within 30 days prior to screening or within 5&#xD;
             half lives of the investigational drug, whichever is longer&#xD;
&#xD;
          3. History of gene therapy or cell transplantation or any other experimental brain&#xD;
             surgery&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>63 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Anne Rosser, PhD FRCP</last_name>
    <role>Principal Investigator</role>
    <affiliation>Cardiff University</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>University of California, San Diego (UCSD)</name>
      <address>
        <city>San Diego</city>
        <state>California</state>
        <zip>920161</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Rocky Mountain Movement Disorders Center</name>
      <address>
        <city>Englewood</city>
        <state>Colorado</state>
        <zip>80113</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Beth Israel Deaconess</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02215</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Columbia University</name>
      <address>
        <city>New York</city>
        <state>New York</state>
        <zip>10032</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Inland Northwest Research</name>
      <address>
        <city>Spokane</city>
        <state>Washington</state>
        <zip>99202</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Centre for Movement Disorders</name>
      <address>
        <city>Toronto</city>
        <state>Ontario</state>
        <zip>M3B 257</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>North York General Hospital</name>
      <address>
        <city>Toronto</city>
        <state>Ontario</state>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>ICM - Institut du Cerveau et de la Moelle épinière</name>
      <address>
        <city>Paris</city>
        <zip>75013</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>George-Huntington-Institut (GHI)</name>
      <address>
        <city>Muenster</city>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University College London - Institute of Neurology &amp; The National Hospital for Neurology and Neurosurgery</name>
      <address>
        <city>London</city>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Canada</country>
    <country>France</country>
    <country>Germany</country>
    <country>United Kingdom</country>
    <country>United States</country>
  </location_countries>
  <verification_date>September 2022</verification_date>
  <study_first_submitted>May 20, 2020</study_first_submitted>
  <study_first_submitted_qc>May 22, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">May 28, 2020</study_first_posted>
  <last_update_submitted>September 21, 2022</last_update_submitted>
  <last_update_submitted_qc>September 21, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">September 23, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>HD</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Huntington Disease</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>Undecided</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/NCT04406636
org study id: TTX N1
nct id: NCT04406636
lead sponsor: 
collaborator: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
SHIELD HD is an international, multisite, prospective, longitudinal cohort natural history
      study to assess the natural history of HD and its biomarkers that are associated with
      modulation of the number of cytosine-adenine-guanine (CAG) repeats in the mutant Huntingtin
      (HTT) gene.

      Approximately 60 patients will be enrolled into the study and followed for up to 24 months at
      clinical sites in North America and Europe.

      The results of this study will inform assessments for a future interventional treatment
      trial.
The rationale for this study is to obtain longitudinal information related to Somatic
      Instability and DNA damage response genes in HDGECs at various stages of the disease.
      Established assessments of disease progression will also be recorded.
observational model: Cohort
time perspective: Prospective
measure: DDR gene expression
time frame: 2 years
description: To assess deoxyribonucleic acid (DNA) damage repair (DDR) gene expression in accessible biofluids and disease trajectories for established and novel biomarkers and clinical outcomes.
measure: Compare rates of change in biomarkers for disease progression
time frame: 2 years
description: To compare the rates of change for different outcomes and cytosine adenine guanine (CAG) age product (CAP) Scores.
measure: Additional biomarkers to be examined
time frame: 2 years
description: The exploratory objectives of this study are to be determined and may include the examination of additional biomarkers present in CSF, plasma, and whole blood, including but not limited to mutant HTT (mHTT) protein, cytokines, and others, as well as clinical markers of progression.
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 18 Years
maximum age: 63 Years
healthy volunteers: No
last name: Anne Rosser, PhD FRCP
role: Principal Investigator
affiliation: Cardiff University
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: Canada
country: France
country: Germany
country: United Kingdom
country: United States
responsible party type: Sponsor
mesh term: Huntington Disease
sharing ipd: Undecided