<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/NCT04462692</url>
  </required_header>
  <id_info>
    <org_study_id>RGX-381-9101</org_study_id>
    <nct_id>NCT04462692</nct_id>
  </id_info>
  <brief_title>An Observational Study in Children With CLN2 Batten Disease</brief_title>
  <official_title>A Prospective, Observational Study to Evaluate Ocular Disease Progression in Children With CLN2 Batten Disease</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 prospective, longitudinal natural history study to document the progression of&#xD;
      ocular manifestations of CLN2 disease among a community-dwelling population of pediatric&#xD;
      participants affected by this disease.&#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 prospective data collection, ocular&#xD;
      disease progression in children with a clinical presentation consistent with CLN2 Batten&#xD;
      disease undergoing current standard of care for their condition. No investigational product&#xD;
      is administered in this observational 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Withdrawn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">October 2023</completion_date>
  <primary_completion_date type="Anticipated">October 2023</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>Change in retinal structure in children with CLN2 Batten disease</measure>
    <time_frame>96 weeks</time_frame>
    <description>As assessed by SD-OCT measures over time.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Change in visual function</measure>
    <time_frame>96 weeks</time_frame>
    <description>As assessed by visual acuity over time.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change in visual function</measure>
    <time_frame>96 weeks</time_frame>
    <description>As assessed by pupillary light reflex over time.</description>
  </secondary_outcome>
  <enrollment type="Actual">0</enrollment>
  <condition>Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)</condition>
  <eligibility>
    <study_pop>
      <textblock>
        Children with CLN2 Batten disease undergoing current standard of care for their condition.&#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. His or her legal guardian(s) is(are) willing and able to provide them written, signed&#xD;
             informed consent.&#xD;
&#xD;
          2. Has documented diagnosis of CLN2 disease due to TPP1 deficiency, or has a relative&#xD;
             clinically diagnosed with CLN2 with the same mutation as the participant&#xD;
&#xD;
          3. Is currently receiving biweekly ERT treatment with cerliponase alfa&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
        A participant is excluded from the study if any of the following criteria apply:&#xD;
&#xD;
          1. Has had prior treatment with an adeno-associated virus-based AAV gene therapy&#xD;
&#xD;
          2. Is currently participating in a clinical trial of investigational product for the&#xD;
             treatment of CLN2 disease&#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 2, 2020</study_first_submitted>
  <study_first_submitted_qc>July 2, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">July 8, 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/NCT04462692
org study id: RGX-381-9101
nct id: NCT04462692
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
This is a prospective, longitudinal natural history study to document the progression of
      ocular manifestations of CLN2 disease among a community-dwelling population of pediatric
      participants affected by this disease.
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 prospective data collection, ocular
      disease progression in children with a clinical presentation consistent with CLN2 Batten
      disease undergoing current standard of care for their condition. No investigational product
      is administered in this observational study.
observational model: Case-Only
time perspective: Prospective
measure: Change in retinal structure in children with CLN2 Batten disease
time frame: 96 weeks
description: As assessed by SD-OCT measures over time.
measure: Change in visual function
time frame: 96 weeks
description: As assessed by visual acuity over time.
measure: Change in visual function
time frame: 96 weeks
description: As assessed by pupillary light reflex over time.
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