<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/NCT02994368</url>
  </required_header>
  <id_info>
    <org_study_id>4D-CHM-001-NH-0001</org_study_id>
    <nct_id>NCT02994368</nct_id>
  </id_info>
  <brief_title>&quot;Natural History&quot; Study of Choroideremia</brief_title>
  <official_title>A Multicenter Prospective Observational &quot;Natural History&quot; Study in Patients With Choroideremia</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>4D Molecular Therapeutics</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>4D Molecular Therapeutics</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this study is to understand the rate of progression of all stages of&#xD;
      choroideremia using a variety of assessments performed in the clinic including visual field&#xD;
      measures, specialized photography of the eye and participant-reported visual problems&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Terminated</overall_status>
  <start_date>December 2016</start_date>
  <completion_date type="Actual">October 2021</completion_date>
  <primary_completion_date type="Actual">October 2021</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>Rate of progression of disease</measure>
    <time_frame>4 years</time_frame>
    <description>A variety of psychophysical, anatomical and image-based endpoints</description>
  </primary_outcome>
  <number_of_groups>2</number_of_groups>
  <enrollment type="Actual">57</enrollment>
  <condition>Choroideremia</condition>
  <arm_group>
    <arm_group_label>Primary Cohort</arm_group_label>
    <description>No intervention</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Expansion Cohort</arm_group_label>
    <description>No intervention</description>
  </arm_group>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Observation</intervention_name>
    <arm_group_label>Expansion Cohort</arm_group_label>
    <arm_group_label>Primary Cohort</arm_group_label>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        Individuals with a clinical diagnosis of choroideremia&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria (Primary Cohort):&#xD;
&#xD;
          -  Healthy individuals (at least 14 years) with choroideremia (20/200 or better vision)&#xD;
             willing to participate in an observational study and meeting the eligibility criteria.&#xD;
&#xD;
        Inclusion Criteria (Expansion Cohort):&#xD;
&#xD;
          -  Healthy individuals (at least 18 years) with choroideremia (20/200 or better vision in&#xD;
             both eyes) willing to participate in an observational study and meeting the&#xD;
             eligibility criteria.&#xD;
&#xD;
        Exclusion Criteria (Primary and Expansion Cohort):&#xD;
&#xD;
          1. Prior therapy with an AAV vector-based treatment&#xD;
&#xD;
          2. Pre-existing eye conditions that would: (1) preclude future planned treatment in a&#xD;
             therapeutic intent clinical trial (i.e. intravitreal injection), (2) interfere with&#xD;
             the interpretation of study endpoints, and/ or put patient at risk for surgical&#xD;
             complications&#xD;
&#xD;
          3. Complicating systemic diseases that would preclude future enrollment in a therapeutic&#xD;
             intent clinical trial&#xD;
&#xD;
          4. Any other condition that would not allow the potential subject to complete follow-up&#xD;
             examinations during the course of the study and, in the opinion of the investigator,&#xD;
             makes the potential subject unsuitable for the study.&#xD;
      </textblock>
    </criteria>
    <gender>Male</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Schonmei Lee, MD</last_name>
    <role>Study Director</role>
    <affiliation>4D Molecular Therapeutics</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Retina Foundation of the Southwest</name>
      <address>
        <city>Dallas</city>
        <state>Texas</state>
        <zip>75251</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Baylor College of Medicine, Ophthalmology-Cullen Eye Center</name>
      <address>
        <city>Houston</city>
        <state>Texas</state>
        <zip>77030</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Moran Eye Center, University of Utah</name>
      <address>
        <city>Salt Lake City</city>
        <state>Utah</state>
        <zip>84132</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Alberta</name>
      <address>
        <city>Edmonton</city>
        <state>Alberta</state>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Canada</country>
    <country>United States</country>
  </location_countries>
  <verification_date>January 2022</verification_date>
  <study_first_submitted>December 13, 2016</study_first_submitted>
  <study_first_submitted_qc>December 14, 2016</study_first_submitted_qc>
  <study_first_posted type="Estimate">December 15, 2016</study_first_posted>
  <last_update_submitted>January 4, 2022</last_update_submitted>
  <last_update_submitted_qc>January 4, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">January 6, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>retinal degeneration</keyword>
  <keyword>eye</keyword>
  <keyword>REP-1 gene</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Choroideremia</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/NCT02994368
org study id: 4D-CHM-001-NH-0001
nct id: NCT02994368
lead sponsor: 
has dmc: No
The purpose of this study is to understand the rate of progression of all stages of
      choroideremia using a variety of assessments performed in the clinic including visual field
      measures, specialized photography of the eye and participant-reported visual problems
observational model: Cohort
time perspective: Prospective
measure: Rate of progression of disease
time frame: 4 years
description: A variety of psychophysical, anatomical and image-based endpoints
arm group label: Primary Cohort
description: No intervention
arm group label: Expansion Cohort
description: No intervention
intervention type: Other
intervention name: Observation
arm group label: Expansion Cohort
arm group label: Primary Cohort
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: Male
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Schonmei Lee, MD
role: Study Director
affiliation: 4D Molecular Therapeutics
facility: 
facility: 
facility: 
facility: 
country: Canada
country: United States
responsible party type: Sponsor
mesh term: Choroideremia
sharing ipd: No