<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/NCT02230761</url>
  </required_header>
  <id_info>
    <org_study_id>XOPH5-OINT-2</org_study_id>
    <nct_id>NCT02230761</nct_id>
  </id_info>
  <brief_title>Phase 2 Study of XAF5 (XOPH5) Ointment for Reduction of Excess Eyelid Fat (Steatoblepharon)</brief_title>
  <official_title>A Phase 2, Randomized, Double-masked, Placebo-controlled Study of XOPH5 Ointment for Reduction of Lower Lid Steatoblepharon</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Topokine Therapeutics, Inc.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Topokine Therapeutics, Inc.</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      This study will test whether XAF5 (XOPH5) ointment, applied once daily to the lower eyelids&#xD;
      for 10 weeks, reduces the prominence of lower eyelid fat in adults with moderate to severe&#xD;
      steatoblepharon (excess eyelid fat).&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date>September 2014</start_date>
  <completion_date type="Actual">December 2014</completion_date>
  <primary_completion_date type="Actual">December 2014</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>Lower Eyelid Steatoblepharon Severity (LESS) Score--Clinician-Reported</measure>
    <time_frame>11 weeks</time_frame>
    <description>Photonumeric scale, range 0-4, 0 is absence of steatoblepharon, 4 is very severe steatoblepharon.</description>
  </primary_outcome>
  <other_outcome>
    <measure>Patient Satisfaction Scale 1 (PSS1)</measure>
    <time_frame>0-11 weeks</time_frame>
    <description>Change from Baseline to Week 11 in PSS1 score. PSS1 scale items use an ordinal scale with range 0-10, where 0 is the least favorable and 10 is the most favorable. The composite score is the sum of 6 items and has a possible response range of 0 to 60.</description>
  </other_outcome>
  <other_outcome>
    <measure>Patient Satisfaction Scale 2 (PSS2)</measure>
    <time_frame>11 weeks</time_frame>
    <description>Composite score on PSS2 scale. PSS2 is an ordinal scale with range 1-7, where 1 is least favorable and 7 is most favorable. The composite score is the sum of 3 items and has a possible response range of 3 to 21.</description>
  </other_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">90</enrollment>
  <condition>Lower Eyelid Steatoblepharon (Excess Eyelid Fat)</condition>
  <arm_group>
    <arm_group_label>XOPH5 Ointment</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>XOPH5 Ointment is the investigational drug to be studied.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Placebo Ointment</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Placebo is an ointment that matches XOPH5 Ointment but lacks the active ingredient.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>XOPH5 Ointment</intervention_name>
    <arm_group_label>XOPH5 Ointment</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebo</intervention_name>
    <arm_group_label>Placebo Ointment</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria (abbreviated):&#xD;
&#xD;
          -  Moderate to severe lower eyelid steatoblepharon (LESS score of 2-4 bilaterally)&#xD;
&#xD;
          -  Men and women 40-70 years old&#xD;
&#xD;
          -  Healthy facial skin&#xD;
&#xD;
          -  Must understand and provide informed consent&#xD;
&#xD;
        Exclusion Criteria (abbreviated):&#xD;
&#xD;
          -  Best corrected visual acuity worse than 20/40 in either eye&#xD;
&#xD;
          -  Regular use of ophthalmic medication in either eye&#xD;
&#xD;
          -  Planned use of contact lenses during the study&#xD;
&#xD;
          -  Any history of lower eyelid surgery&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>40 Years</minimum_age>
    <maximum_age>70 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Michael S. Singer, MD, PhD</last_name>
    <role>Study Chair</role>
    <affiliation>Topokine Therapeutics, Inc.</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Topokine Therapeutics Clinical Study Centers</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02111</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>December 2015</verification_date>
  <study_first_submitted>August 28, 2014</study_first_submitted>
  <study_first_submitted_qc>September 2, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">September 3, 2014</study_first_posted>
  <results_first_submitted>November 19, 2015</results_first_submitted>
  <results_first_submitted_qc>January 9, 2016</results_first_submitted_qc>
  <results_first_posted type="Estimate">February 8, 2016</results_first_posted>
  <last_update_submitted>January 9, 2016</last_update_submitted>
  <last_update_submitted_qc>January 9, 2016</last_update_submitted_qc>
  <last_update_posted type="Estimate">February 8, 2016</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>steatoblepharon</keyword>
  <keyword>blepharoptosis</keyword>
  <keyword>dermatochalasis</keyword>
  <keyword>blepharoplasty</keyword>
  <keyword>periorbital</keyword>
  <keyword>orbital</keyword>
  <keyword>eyelid</keyword>
  <keyword>eyebags</keyword>
  <keyword>eye bags</keyword>

  <clinical_results>

    <participant_flow>
      <group_list>
        <group group_id="P1">
          <title>XOPH5 Ointment</title>
          <description>XOPH5 Ointment is the investigational drug to be studied.&#xD;
XOPH5 Ointment</description>
        </group>
        <group group_id="P2">
          <title>Placebo Ointment</title>
          <description>Placebo is an ointment that matches XOPH5 Ointment but lacks the active ingredient.&#xD;
Placebo</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="60"/>
                <participants group_id="P2" count="30"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="60"/>
                <participants group_id="P2" count="29"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
                <participants group_id="P2" count="1"/>
              </participants_list>
            </milestone>
          </milestone_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <group_list>
        <group group_id="B1">
          <title>XOPH5 Ointment</title>
          <description>XOPH5 Ointment is the investigational drug to be studied.&#xD;
XOPH5 Ointment</description>
        </group>
        <group group_id="B2">
          <title>Placebo Ointment</title>
          <description>Placebo is an ointment that matches XOPH5 Ointment but lacks the active ingredient.&#xD;
Placebo</description>
        </group>
        <group group_id="B3">
          <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="60"/>
            <count group_id="B2" value="30"/>
            <count group_id="B3" value="90"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>years</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="54.2" spread="7.4"/>
                    <measurement group_id="B2" value="55.1" spread="7.5"/>
                    <measurement group_id="B3" value="54.5" spread="7.4"/>
                  </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="40"/>
                    <measurement group_id="B2" value="17"/>
                    <measurement group_id="B3" value="57"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="20"/>
                    <measurement group_id="B2" value="13"/>
                    <measurement group_id="B3" value="33"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Lower Eyelid Steatoblepharon Severity (LESS) Score--Clinician-Reported</title>
        <description>Photonumeric scale, range 0-4, 0 is absence of steatoblepharon, 4 is very severe steatoblepharon.</description>
        <time_frame>11 weeks</time_frame>
        <population>intention-to-treat</population>
        <group_list>
          <group group_id="O1">
            <title>XOPH5 Ointment</title>
            <description>XOPH5 Ointment is the investigational drug to be studied.&#xD;
XOPH5 Ointment</description>
          </group>
          <group group_id="O2">
            <title>Placebo Ointment</title>
            <description>Placebo is an ointment that matches XOPH5 Ointment but lacks the active ingredient.&#xD;
Placebo</description>
          </group>
        </group_list>
        <measure>
          <title>Lower Eyelid Steatoblepharon Severity (LESS) Score--Clinician-Reported</title>
          <description>Photonumeric scale, range 0-4, 0 is absence of steatoblepharon, 4 is very severe steatoblepharon.</description>
          <population>intention-to-treat</population>
          <units>participants</units>
          <param>Number</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="60"/>
                <count group_id="O2" value="30"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="27"/>
                    <measurement group_id="O2" value="6"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority or Other</non_inferiority_type>
            <p_value>0.020</p_value>
            <method>Chi-squared</method>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Other Pre-specified</type>
        <title>Patient Satisfaction Scale 1 (PSS1)</title>
        <description>Change from Baseline to Week 11 in PSS1 score. PSS1 scale items use an ordinal scale with range 0-10, where 0 is the least favorable and 10 is the most favorable. The composite score is the sum of 6 items and has a possible response range of 0 to 60.</description>
        <time_frame>0-11 weeks</time_frame>
        <population>intention-to-treat</population>
        <group_list>
          <group group_id="O1">
            <title>XOPH5 Ointment</title>
            <description>XOPH5 Ointment is the investigational drug to be studied.&#xD;
XOPH5 Ointment</description>
          </group>
          <group group_id="O2">
            <title>Placebo Ointment</title>
            <description>Placebo is an ointment that matches XOPH5 Ointment but lacks the active ingredient.&#xD;
Placebo</description>
          </group>
        </group_list>
        <measure>
          <title>Patient Satisfaction Scale 1 (PSS1)</title>
          <description>Change from Baseline to Week 11 in PSS1 score. PSS1 scale items use an ordinal scale with range 0-10, where 0 is the least favorable and 10 is the most favorable. The composite score is the sum of 6 items and has a possible response range of 0 to 60.</description>
          <population>intention-to-treat</population>
          <units>units on a scale</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="60"/>
                <count group_id="O2" value="30"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="17.9" spread="14.4"/>
                    <measurement group_id="O2" value="7.3" spread="13.0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority or Other</non_inferiority_type>
            <p_value>0.001</p_value>
            <method>t-test, 2 sided</method>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Other Pre-specified</type>
        <title>Patient Satisfaction Scale 2 (PSS2)</title>
        <description>Composite score on PSS2 scale. PSS2 is an ordinal scale with range 1-7, where 1 is least favorable and 7 is most favorable. The composite score is the sum of 3 items and has a possible response range of 3 to 21.</description>
        <time_frame>11 weeks</time_frame>
        <population>intention-to-treat</population>
        <group_list>
          <group group_id="O1">
            <title>XOPH5 Ointment</title>
            <description>XOPH5 Ointment is the investigational drug to be studied.&#xD;
XOPH5 Ointment</description>
          </group>
          <group group_id="O2">
            <title>Placebo Ointment</title>
            <description>Placebo is an ointment that matches XOPH5 Ointment but lacks the active ingredient.&#xD;
Placebo</description>
          </group>
        </group_list>
        <measure>
          <title>Patient Satisfaction Scale 2 (PSS2)</title>
          <description>Composite score on PSS2 scale. PSS2 is an ordinal scale with range 1-7, where 1 is least favorable and 7 is most favorable. The composite score is the sum of 3 items and has a possible response range of 3 to 21.</description>
          <population>intention-to-treat</population>
          <units>units on a scale</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="60"/>
                <count group_id="O2" value="30"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="16.3" spread="3.3"/>
                    <measurement group_id="O2" value="14.0" spread="3.0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority or Other</non_inferiority_type>
            <p_value>0.002</p_value>
            <method>t-test, 2 sided</method>
          </analysis>
        </analysis_list>
      </outcome>
    </outcome_list>

    <reported_events>
      <group_list>
        <group group_id="E1">
          <title>XOPH5 Ointment</title>
          <description>XOPH5 Ointment is the investigational drug to be studied.&#xD;
XOPH5 Ointment</description>
        </group>
        <group group_id="E2">
          <title>Placebo Ointment</title>
          <description>Placebo is an ointment that matches XOPH5 Ointment but lacks the active ingredient.&#xD;
Placebo</description>
        </group>
      </group_list>
      <serious_events>
        <default_assessment>Systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="60"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="30"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Nervous system disorders</title>
            <event_list>
              <event>
                <sub_title>Guillan-Barre syndrome</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="60"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="30"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>5</frequency_threshold>
        <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="11" subjects_at_risk="60"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="30"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Skin and subcutaneous tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Hyperemia eyelid (mild, transient)</sub_title>
                <counts group_id="E1" events="9" subjects_affected="9" subjects_at_risk="60"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="30"/>
              </event>
              <event>
                <sub_title>Increase in vellus hair (mild)</sub_title>
                <counts group_id="E1" events="4" subjects_affected="4" subjects_at_risk="60"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="30"/>
              </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>The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.</restrictive_agreement>
    </certain_agreements>
    <point_of_contact>
      <name_or_title>Chief Scientific Officer</name_or_title>
      <organization>Topokine Therapeutics, Inc.</organization>
      <phone>617 203 2034</phone>
      <email>CSO@topokine.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/NCT02230761
org study id: XOPH5-OINT-2
nct id: NCT02230761
lead sponsor: 
has dmc: No
This study will test whether XAF5 (XOPH5) ointment, applied once daily to the lower eyelids
      for 10 weeks, reduces the prominence of lower eyelid fat in adults with moderate to severe
      steatoblepharon (excess eyelid fat).
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
measure: Lower Eyelid Steatoblepharon Severity (LESS) Score--Clinician-Reported
time frame: 11 weeks
description: Photonumeric scale, range 0-4, 0 is absence of steatoblepharon, 4 is very severe steatoblepharon.
measure: Patient Satisfaction Scale 1 (PSS1)
time frame: 0-11 weeks
description: Change from Baseline to Week 11 in PSS1 score. PSS1 scale items use an ordinal scale with range 0-10, where 0 is the least favorable and 10 is the most favorable. The composite score is the sum of 6 items and has a possible response range of 0 to 60.
measure: Patient Satisfaction Scale 2 (PSS2)
time frame: 11 weeks
description: Composite score on PSS2 scale. PSS2 is an ordinal scale with range 1-7, where 1 is least favorable and 7 is most favorable. The composite score is the sum of 3 items and has a possible response range of 3 to 21.
arm group label: XOPH5 Ointment
arm group type: Experimental
description: XOPH5 Ointment is the investigational drug to be studied.
arm group label: Placebo Ointment
arm group type: Placebo Comparator
description: Placebo is an ointment that matches XOPH5 Ointment but lacks the active ingredient.
intervention type: Drug
intervention name: XOPH5 Ointment
arm group label: XOPH5 Ointment
intervention type: Drug
intervention name: Placebo
arm group label: Placebo Ointment
criteria: 
gender: All
minimum age: 40 Years
maximum age: 70 Years
healthy volunteers: No
last name: Michael S. Singer, MD, PhD
role: Study Chair
affiliation: Topokine Therapeutics, Inc.
facility: 
country: United States
responsible party type: Sponsor
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
point of contact: 