<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/NCT03033108</url>
  </required_header>
  <id_info>
    <org_study_id>4429-204</org_study_id>
    <nct_id>NCT03033108</nct_id>
  </id_info>
  <brief_title>Pharmacodynamic Study of Emixustat Hydrochloride in Subjects With Macular Atrophy Secondary to Stargardt Disease</brief_title>
  <official_title>A Phase 2a Multicenter, Randomized, Masked Study Evaluating the Pharmacodynamics of Emixustat Hydrochloride in Subjects With Macular Atrophy Secondary to Stargardt Disease</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Kubota Vision Inc.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Kubota Vision Inc.</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      This is a pharmacodynamics study of emixustat hydrochloride in subjects with macular atrophy&#xD;
      secondary to Stargardt disease.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This is a multicenter, randomized, masked study to characterize the pharmacodynamics, safety&#xD;
      and tolerability of emixustat in subjects with macular atrophy secondary to Stargardt&#xD;
      disease.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">January 2017</start_date>
  <completion_date type="Actual">December 2017</completion_date>
  <primary_completion_date type="Actual">November 2017</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Change in Electrical Response of the Retina to a Flash of Light, as Measured by Electroretinogram</measure>
    <time_frame>Baseline and 1 month</time_frame>
    <description>Percent suppression compared to baseline of rod b-wave amplitude recovery after a photobleaching light.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Percentage of Subjects With Adverse Events, by Severity and Seriousness</measure>
    <time_frame>1 month</time_frame>
    <description>Assessment of safety profile</description>
  </secondary_outcome>
  <number_of_arms>3</number_of_arms>
  <enrollment type="Actual">23</enrollment>
  <condition>Stargardt Disease</condition>
  <condition>Macular Atrophy</condition>
  <arm_group>
    <arm_group_label>Emixustat Dose 1</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>lowest dose of once-daily oral emixustat</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Emixustat Dose 2</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>middle dose of once-daily oral emixustat</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Emixustat Dose 3</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>highest dose of once-daily oral emixustat</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Emixustat</intervention_name>
    <description>Once daily, tablet for oral administration</description>
    <arm_group_label>Emixustat Dose 1</arm_group_label>
    <arm_group_label>Emixustat Dose 2</arm_group_label>
    <arm_group_label>Emixustat Dose 3</arm_group_label>
    <other_name>emixustat hydrochloride</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria, including, but not limited to:&#xD;
&#xD;
          -  Clinical diagnosis of macular atrophy (MA) secondary to Stargardt disease (STGD) in&#xD;
             one or both eyes&#xD;
&#xD;
          -  At least 2 pathogenic mutations of the ABCA4 gene&#xD;
&#xD;
          -  Early Treatment Diabetic Retinopathy Study BCVA of ≥ 20 letters (approximately ≥&#xD;
             20/400 Snellen) in the study eye&#xD;
&#xD;
          -  Adequate clarity of ocular media and adequate pupillary dilation to permit good&#xD;
             quality imaging of MA in the study eye&#xD;
&#xD;
          -  Able and willing to provide written informed consent before undergoing any&#xD;
             study-related procedures&#xD;
&#xD;
          -  Able to reliably administer oral medication by self or with available assistance&#xD;
&#xD;
        Exclusion Criteria, including, but not limited to:&#xD;
&#xD;
          -  Macular atrophy associated with a condition other than STGD in either eye.&#xD;
&#xD;
          -  Presence in either eye of an active ocular disease that in the opinion of the&#xD;
             Investigator compromises or confounds visual function.&#xD;
&#xD;
          -  History of any intraocular or ocular surface surgery in either eye within 3 months of&#xD;
             screening.&#xD;
&#xD;
          -  Current or previous participation in an interventional study to treat STGD using gene&#xD;
             therapy or stem cell therapy at any time, or participation in an interventional study&#xD;
             of a vitamin A derivative ≤3 months prior to screening.&#xD;
&#xD;
          -  Pre-specified laboratory abnormalities at screening&#xD;
&#xD;
          -  Presence of other medical or ophthalmic disease, physical examination finding, or&#xD;
             clinical laboratory finding that in the opinion of the Investigator may contraindicate&#xD;
             the use of an investigational drug and place the subject at risk&#xD;
&#xD;
          -  Current or history of cancer (except for adequately treated basal cell or squamous&#xD;
             cell carcinoma of the skin) within 1 year of screening&#xD;
&#xD;
          -  History of myocardial infarction, stroke, unstable ischemic heart disease,&#xD;
             uncontrolled cardiac arrhythmia, or hospitalization for congestive heart failure&#xD;
             within 6 months of screening.&#xD;
&#xD;
          -  Anticipated hospitalization for a medical/surgical procedure(s) that could result in&#xD;
             interruption/premature cessation of study treatment or participation.&#xD;
&#xD;
          -  Electrocardiogram with a clinically significant abnormal finding&#xD;
&#xD;
          -  Female subjects who are pregnant or lactating&#xD;
&#xD;
          -  Female subjects of childbearing potential or male subjects who are not surgically&#xD;
             sterile who are not willing to practice a medically accepted method of birth control&#xD;
             with their sexual partner from screening through 30 days after the final dose of study&#xD;
             drug&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Acucela Medical Director, MD</last_name>
    <role>Study Director</role>
    <affiliation>Kubota Vision Inc.</affiliation>
  </overall_official>
  <location>
    <facility>
      <address>
        <city>Dallas</city>
        <state>Texas</state>
        <zip>75231</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>April 2021</verification_date>
  <study_first_submitted>January 20, 2017</study_first_submitted>
  <study_first_submitted_qc>January 24, 2017</study_first_submitted_qc>
  <study_first_posted type="Estimate">January 26, 2017</study_first_posted>
  <results_first_submitted>March 31, 2021</results_first_submitted>
  <results_first_submitted_qc>March 31, 2021</results_first_submitted_qc>
  <results_first_posted type="Actual">April 27, 2021</results_first_posted>
  <last_update_submitted>April 27, 2021</last_update_submitted>
  <last_update_submitted_qc>April 27, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">May 19, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Stargardt Disease</mesh_term>
    <mesh_term>Macular Degeneration</mesh_term>
    <mesh_term>Anetoderma</mesh_term>
    <mesh_term>Atrophy</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <provided_document_section>
    <provided_document>
      <document_type>Study Protocol and Statistical Analysis Plan</document_type>
      <document_has_protocol>Yes</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>Yes</document_has_sap>
      <document_date>July 13, 2017</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/08/NCT03033108/Prot_SAP_000.pdf</document_url>
    </provided_document>
  </provided_document_section>

  <clinical_results>

    <participant_flow>
      <recruitment_details>Subjects were recruited at 6 sites in the United States from January to September 2017</recruitment_details>
      <group_list>
        <group group_id="P1">
          <title>Emixustat Dose 1</title>
          <description>lowest dose of once-daily oral emixustat&#xD;
Emixustat: Once daily, tablet for oral administration</description>
        </group>
        <group group_id="P2">
          <title>Emixustat Dose 2</title>
          <description>middle dose of once-daily oral emixustat&#xD;
Emixustat: Once daily, tablet for oral administration</description>
        </group>
        <group group_id="P3">
          <title>Emixustat Dose 3</title>
          <description>highest dose of once-daily oral emixustat&#xD;
Emixustat: Once daily, tablet for oral administration</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="7"/>
                <participants group_id="P2" count="9"/>
                <participants group_id="P3" count="7"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="7"/>
                <participants group_id="P2" count="8"/>
                <participants group_id="P3" count="7"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
                <participants group_id="P2" count="1"/>
                <participants group_id="P3" count="0"/>
              </participants_list>
            </milestone>
          </milestone_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <group_list>
        <group group_id="B1">
          <title>Emixustat Dose 1</title>
          <description>lowest dose of once-daily oral emixustat&#xD;
Emixustat: Once daily, tablet for oral administration</description>
        </group>
        <group group_id="B2">
          <title>Emixustat Dose 2</title>
          <description>middle dose of once-daily oral emixustat&#xD;
Emixustat: Once daily, tablet for oral administration</description>
        </group>
        <group group_id="B3">
          <title>Emixustat Dose 3</title>
          <description>highest dose of once-daily oral emixustat&#xD;
Emixustat: Once daily, tablet for oral administration</description>
        </group>
        <group group_id="B4">
          <title>Total</title>
          <description>Total of all reporting groups</description>
        </group>
      </group_list>
      <analyzed_list>
        <analyzed>
          <units>Participants</units>
          <scope>Overall</scope>
          <count_list>
            <count group_id="B1" value="7"/>
            <count group_id="B2" value="9"/>
            <count group_id="B3" value="7"/>
            <count group_id="B4" value="23"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>years</units>
          <param>Mean</param>
          <dispersion>Full Range</dispersion>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="53.6" lower_limit="27" upper_limit="77"/>
                    <measurement group_id="B2" value="54.3" lower_limit="32" upper_limit="77"/>
                    <measurement group_id="B3" value="46.3" lower_limit="18" upper_limit="70"/>
                    <measurement group_id="B4" value="51.6" lower_limit="18" upper_limit="77"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Sex: Female, Male</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Female</title>
                  <measurement_list>
                    <measurement group_id="B1" value="4"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="3"/>
                    <measurement group_id="B4" value="8"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="3"/>
                    <measurement group_id="B2" value="8"/>
                    <measurement group_id="B3" value="4"/>
                    <measurement group_id="B4" value="15"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Ethnicity (NIH/OMB)</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="1"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Not Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="6"/>
                    <measurement group_id="B2" value="9"/>
                    <measurement group_id="B3" value="7"/>
                    <measurement group_id="B4" value="22"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Race (NIH/OMB)</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>American Indian or Alaska Native</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Asian</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="1"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Native Hawaiian or Other Pacific Islander</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Black or African American</title>
                  <measurement_list>
                    <measurement group_id="B1" value="2"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="1"/>
                    <measurement group_id="B4" value="4"/>
                  </measurement_list>
                </category>
                <category>
                  <title>White</title>
                  <measurement_list>
                    <measurement group_id="B1" value="5"/>
                    <measurement group_id="B2" value="7"/>
                    <measurement group_id="B3" value="6"/>
                    <measurement group_id="B4" value="18"/>
                  </measurement_list>
                </category>
                <category>
                  <title>More than one race</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Region of Enrollment</title>
          <units>participants</units>
          <param>Number</param>
          <class_list>
            <class>
              <title>United States</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="7"/>
                    <measurement group_id="B2" value="9"/>
                    <measurement group_id="B3" value="7"/>
                    <measurement group_id="B4" value="23"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Number of ABCA4 gene mutations</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>One mutation</title>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                    <measurement group_id="B2" value="2"/>
                    <measurement group_id="B3" value="2"/>
                    <measurement group_id="B4" value="5"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Two mutations</title>
                  <measurement_list>
                    <measurement group_id="B1" value="6"/>
                    <measurement group_id="B2" value="7"/>
                    <measurement group_id="B3" value="5"/>
                    <measurement group_id="B4" value="18"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Change in Electrical Response of the Retina to a Flash of Light, as Measured by Electroretinogram</title>
        <description>Percent suppression compared to baseline of rod b-wave amplitude recovery after a photobleaching light.</description>
        <time_frame>Baseline and 1 month</time_frame>
        <population>Subjects with evaluable ERGs at both Baseline and Month 1</population>
        <group_list>
          <group group_id="O1">
            <title>Emixustat Dose 1</title>
            <description>lowest dose of once-daily oral emixustat&#xD;
Emixustat: Once daily, tablet for oral administration</description>
          </group>
          <group group_id="O2">
            <title>Emixustat Dose 2</title>
            <description>middle dose of once-daily oral emixustat&#xD;
Emixustat: Once daily, tablet for oral administration</description>
          </group>
          <group group_id="O3">
            <title>Emixustat Dose 3</title>
            <description>highest dose of once-daily oral emixustat&#xD;
Emixustat: Once daily, tablet for oral administration</description>
          </group>
        </group_list>
        <measure>
          <title>Change in Electrical Response of the Retina to a Flash of Light, as Measured by Electroretinogram</title>
          <description>Percent suppression compared to baseline of rod b-wave amplitude recovery after a photobleaching light.</description>
          <population>Subjects with evaluable ERGs at both Baseline and Month 1</population>
          <units>percent suppression</units>
          <param>Median</param>
          <dispersion>Full Range</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="6"/>
                <count group_id="O2" value="7"/>
                <count group_id="O3" value="6"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-12.23" lower_limit="-41.0" upper_limit="54.7"/>
                    <measurement group_id="O2" value="68.00" lower_limit="1.0" upper_limit="91.5"/>
                    <measurement group_id="O3" value="96.69" lower_limit="64.9" upper_limit="100.0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Percentage of Subjects With Adverse Events, by Severity and Seriousness</title>
        <description>Assessment of safety profile</description>
        <time_frame>1 month</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Emixustat Dose 1</title>
            <description>lowest dose of once-daily oral emixustat&#xD;
Emixustat: Once daily, tablet for oral administration</description>
          </group>
          <group group_id="O2">
            <title>Emixustat Dose 2</title>
            <description>middle dose of once-daily oral emixustat&#xD;
Emixustat: Once daily, tablet for oral administration</description>
          </group>
          <group group_id="O3">
            <title>Emixustat Dose 3</title>
            <description>highest dose of once-daily oral emixustat&#xD;
Emixustat: Once daily, tablet for oral administration</description>
          </group>
        </group_list>
        <measure>
          <title>Percentage of Subjects With Adverse Events, by Severity and Seriousness</title>
          <description>Assessment of safety profile</description>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="7"/>
                <count group_id="O2" value="9"/>
                <count group_id="O3" value="7"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Subjects with 1 or more adverse events</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="6"/>
                    <measurement group_id="O2" value="8"/>
                    <measurement group_id="O3" value="6"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Subjects with 1 or more mild adverse events</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="5"/>
                    <measurement group_id="O2" value="5"/>
                    <measurement group_id="O3" value="4"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Subjects with 1 or more moderate adverse events</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                    <measurement group_id="O2" value="2"/>
                    <measurement group_id="O3" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Subjects with 1 or more severe adverse events</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                    <measurement group_id="O2" value="1"/>
                    <measurement group_id="O3" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Subjects with 1 or more serious adverse events</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                    <measurement group_id="O2" value="0"/>
                    <measurement group_id="O3" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>1 month</time_frame>
      <group_list>
        <group group_id="E1">
          <title>Emixustat Dose 1</title>
          <description>lowest dose of once-daily oral emixustat&#xD;
Emixustat: Once daily, tablet for oral administration</description>
        </group>
        <group group_id="E2">
          <title>Emixustat Dose 2</title>
          <description>middle dose of once-daily oral emixustat&#xD;
Emixustat: Once daily, tablet for oral administration</description>
        </group>
        <group group_id="E3">
          <title>Emixustat Dose 3</title>
          <description>highest dose of once-daily oral emixustat&#xD;
Emixustat: Once daily, tablet for oral administration</description>
        </group>
      </group_list>
      <serious_events>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, all-cause mortality</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="7"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="9"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="7"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="9"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>5</frequency_threshold>
        <default_vocab>MedDRA 20.1</default_vocab>
        <default_assessment>Systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, other adverse events</sub_title>
                <counts group_id="E1" subjects_affected="6" subjects_at_risk="7"/>
                <counts group_id="E2" subjects_affected="8" subjects_at_risk="9"/>
                <counts group_id="E3" subjects_affected="7" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Eye disorders</title>
            <event_list>
              <event>
                <sub_title>Delayed Dark Adaptation</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
                <counts group_id="E2" subjects_affected="6" subjects_at_risk="9"/>
                <counts group_id="E3" subjects_affected="4" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Erythropsia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
                <counts group_id="E2" subjects_affected="3" subjects_at_risk="9"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Vision blurred</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="7"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="9"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Photophobia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
                <counts group_id="E2" subjects_affected="2" subjects_at_risk="9"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Visual impairment</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="9"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Blindness day</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="7"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="9"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Chromatopsia</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="7"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="9"/>
                <counts group_id="E3" subjects_affected="2" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Night blindness</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="7"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="9"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Xanthopsia</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="7"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="9"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Asthenopia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="9"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Eye Pain</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="9"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Eye Pruritis</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="9"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Lenticular opacities</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="7"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="9"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Visual acuity reduced</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="7"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="9"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Upper respiratory tract infections</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="7"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="9"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Investigations</title>
            <event_list>
              <event>
                <sub_title>Blood bilirubin increased</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="7"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="9"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Blood creatine phosphokinase increased</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="7"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="9"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Nervous system disorders</title>
            <event_list>
              <event>
                <sub_title>Headache</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="7"/>
                <counts group_id="E2" subjects_affected="2" subjects_at_risk="9"/>
                <counts group_id="E3" subjects_affected="2" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Visual field defect</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="9"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Respiratory, thoracic and mediastinal disorders</title>
            <event_list>
              <event>
                <sub_title>Cough</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="9"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Rhinorrheoea</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="9"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Skin and subcutaneous tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Dermatitis allergic</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="7"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="9"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Rash</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="7"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="9"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </other_events>
    </reported_events>

    <certain_agreements>
      <pi_employee>Principal Investigators are NOT employed by the organization sponsoring the study.</pi_employee>
      <restrictive_agreement>There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed. </restrictive_agreement>
    </certain_agreements>
    <limitations_and_caveats>Small sample size and inherent, significant intersubject and intrasubject variability observed in electroretinography.</limitations_and_caveats>
    <point_of_contact>
      <name_or_title>Clinical Trials Helpdesk</name_or_title>
      <organization>Kubota Vision Inc.</organization>
      <phone>(206) 805-8310</phone>
      <email>clinicaltrials@kubotavision.com</email>
    </point_of_contact>
  </clinical_results>
</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/NCT03033108
org study id: 4429-204
nct id: NCT03033108
lead sponsor: 
has dmc: No
This is a pharmacodynamics study of emixustat hydrochloride in subjects with macular atrophy
      secondary to Stargardt disease.
This is a multicenter, randomized, masked study to characterize the pharmacodynamics, safety
      and tolerability of emixustat in subjects with macular atrophy secondary to Stargardt
      disease.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
measure: Change in Electrical Response of the Retina to a Flash of Light, as Measured by Electroretinogram
time frame: Baseline and 1 month
description: Percent suppression compared to baseline of rod b-wave amplitude recovery after a photobleaching light.
measure: Percentage of Subjects With Adverse Events, by Severity and Seriousness
time frame: 1 month
description: Assessment of safety profile
arm group label: Emixustat Dose 1
arm group type: Experimental
description: lowest dose of once-daily oral emixustat
arm group label: Emixustat Dose 2
arm group type: Experimental
description: middle dose of once-daily oral emixustat
arm group label: Emixustat Dose 3
arm group type: Experimental
description: highest dose of once-daily oral emixustat
intervention type: Drug
intervention name: Emixustat
description: Once daily, tablet for oral administration
arm group label: Emixustat Dose 1
arm group label: Emixustat Dose 2
arm group label: Emixustat Dose 3
other name: emixustat hydrochloride
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Acucela Medical Director, MD
role: Study Director
affiliation: Kubota Vision Inc.
facility: 
country: United States
responsible party type: Sponsor
mesh term: Stargardt Disease
mesh term: Macular Degeneration
mesh term: Anetoderma
mesh term: Atrophy
sharing ipd: No
provided document: 
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
limitations and caveats: Small sample size and inherent, significant intersubject and intrasubject variability observed in electroretinography.
point of contact: 