<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/NCT04480476</url>
  </required_header>
  <id_info>
    <org_study_id>RGX-381-9102</org_study_id>
    <nct_id>NCT04480476</nct_id>
  </id_info>
  <brief_title>A Retrospective, Natural History Study in Children With CLN2</brief_title>
  <official_title>A Retrospective, Chart Review Study to Evaluate Ocular Disease Progression in Children With Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>REGENXBIO Inc.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>REGENXBIO 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>
      This is a multi center, retrospective, chart review study to document the evolution of ocular&#xD;
      disease progression in pediatric patients with CLN2.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      CLN2 is a rare disease with limited available ocular natural history data. While current&#xD;
      standard of care slows motor degeneration, it is not known to treat the ocular manifestations&#xD;
      of disease. This study is planned to document, through retrospective data collection, ocular&#xD;
      disease progression in children with a clinical presentation consistent with CLN2. No&#xD;
      investigational product is administered in this retrospective, chart review study.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Withdrawn</overall_status>
  <why_stopped>
    study stopped due to company decision&#xD;
  </why_stopped>
  <start_date type="Anticipated">March 31, 2021</start_date>
  <completion_date type="Anticipated">April 2022</completion_date>
  <primary_completion_date type="Anticipated">April 2022</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>Retrospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Characterize retinal structural changes in children with CLN2</measure>
    <time_frame>From first available medical chart through informed consent, an average of 10 years</time_frame>
    <description>As assessed in by SD-OCT measures in ophthalmic records of children with CLN2</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Characterize changes in visual function.</measure>
    <time_frame>From first available medical chart through informed consent, an average of 10 years</time_frame>
    <description>As measured by changes in visual acuity over time in ophthalmic records of children with CLN2.</description>
  </secondary_outcome>
  <enrollment type="Actual">0</enrollment>
  <condition>Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)</condition>
  <eligibility>
    <study_pop>
      <textblock>
        Patients with CLN2 Batten Disease&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
        A participant is eligible to be included in the study only if all of the following criteria&#xD;
        apply:&#xD;
&#xD;
          1. The participant's legal guardian(s) is(are) willing and able to provide them written,&#xD;
             signed informed consent.&#xD;
&#xD;
          2. The participant has a documented diagnosis of CLN2 disease due to TPP1 deficiency, or&#xD;
             has a relative clinically diagnosed with CLN2 disease who has the same CLN2 mutations&#xD;
             as the participant&#xD;
&#xD;
          3. The participant has had one or more eye examinations by an eye care specialist at any&#xD;
             time since birth.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
        No exclusion criteria apply to this study.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>N/A</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <verification_date>October 2021</verification_date>
  <study_first_submitted>July 10, 2020</study_first_submitted>
  <study_first_submitted_qc>July 17, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">July 21, 2020</study_first_posted>
  <last_update_submitted>October 25, 2021</last_update_submitted>
  <last_update_submitted_qc>October 25, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">November 1, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>CLN2, Batten Disease</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Neuronal Ceroid-Lipofuscinoses</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/NCT04480476
org study id: RGX-381-9102
nct id: NCT04480476
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
This is a multi center, retrospective, chart review study to document the evolution of ocular
      disease progression in pediatric patients with CLN2.
CLN2 is a rare disease with limited available ocular natural history data. While current
      standard of care slows motor degeneration, it is not known to treat the ocular manifestations
      of disease. This study is planned to document, through retrospective data collection, ocular
      disease progression in children with a clinical presentation consistent with CLN2. No
      investigational product is administered in this retrospective, chart review study.
observational model: Case-Only
time perspective: Retrospective
measure: Characterize retinal structural changes in children with CLN2
time frame: From first available medical chart through informed consent, an average of 10 years
description: As assessed in by SD-OCT measures in ophthalmic records of children with CLN2
measure: Characterize changes in visual function.
time frame: From first available medical chart through informed consent, an average of 10 years
description: As measured by changes in visual acuity over time in ophthalmic records of children with CLN2.
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: N/A
maximum age: N/A
healthy volunteers: No
responsible party type: Sponsor
mesh term: Neuronal Ceroid-Lipofuscinoses
sharing ipd: No