<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/NCT04499235</url>
  </required_header>
  <id_info>
    <org_study_id>AKST4290-221</org_study_id>
    <nct_id>NCT04499235</nct_id>
  </id_info>
  <brief_title>A Study to Assess the Therapeutic Effect and Safety of Adjunctive AKST4290 in Subjects With Bullous Pemphigoid</brief_title>
  <official_title>Double-Blind, Randomized, Placebo-Controlled Trial of AKST4290 for Adjunctive Treatment of Mild to Moderate Bullous Pemphigoid</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Alkahest, Inc.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Alkahest, Inc.</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
    <is_us_export>Yes</is_us_export>
  </oversight_info>
  <brief_summary>
    <textblock>
      This study will evaluate the therapeutic effect and safety of adjunctive AKST4290 in subjects&#xD;
      with bullous pemphigoid (BP).&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This is a randomized, double-blind, placebo-controlled study to assess the therapeutic effect&#xD;
      and safety of adjunctive AKST4290 in subjects with bullous pemphigoid (BP). Subjects will&#xD;
      receive topical mometasone furoate cream (MFC) therapy concurrently with study agent (placebo&#xD;
      or AKST4290) in an inpatient setting until disease control is reached (duration of inpatient&#xD;
      stay is dependent upon individual disease course, but is estimated between 1-3 weeks).&#xD;
&#xD;
      Subjects will receive rescue therapy at any time if their clinical condition worsens or if&#xD;
      their clinical condition fails to improve by the completion of Week 1 on study treatment, as&#xD;
      assessed by the investigator. Rescue therapy will consist of whole-body clobetasol propionate&#xD;
      cream (CPC) (15-50g) and/or oral prednisone (0.5 mg/kg per day), as determined by the&#xD;
      investigator. Subjects who receive rescue therapy will remain in the study until disease&#xD;
      control, unless they are withdrawn or withdraw from participation.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Terminated</overall_status>
  <why_stopped>
    The study was prematurely terminated by the Sponsor due to operational challenges stemming from&#xD;
    the coronavirus disease 2019 (COVID-19) pandemic, treatment limitations, rarity of the disease,&#xD;
    and drug supply considerations.&#xD;
  </why_stopped>
  <start_date type="Actual">January 30, 2020</start_date>
  <completion_date type="Actual">April 14, 2021</completion_date>
  <primary_completion_date type="Actual">March 29, 2021</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>Double (Participant, Investigator)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>The Percentage of Subjects Who Achieve Disease Control Without Rescue Therapy</measure>
    <time_frame>Baseline to up to 3 weeks (until disease control)</time_frame>
    <description>Disease control is defined as ≤ 3 new blisters/eczematous lesions/urticarial plaques/day and healing of existing blisters/eczematous lesions/urticarial plaques without requiring rescue therapy.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Number of Participants With TEAEs, Assessed by Seriousness and Severity</measure>
    <time_frame>Baseline to 5 weeks</time_frame>
    <description>Treatment-emergent AEs summarized by MedDRA coding terms; separate tabulations produced for incidence, seriousness and severity of AEs</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Time to Disease Control</measure>
    <time_frame>Baseline to up to 3 weeks (until disease control)</time_frame>
    <description>Time to disease control by treatment day/week. The time to disease control is calculated as the date of disease control minus Date of Visit 2 (Baseline (Day 1)) plus 1.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Time to Rescue Therapy</measure>
    <time_frame>Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.</time_frame>
    <description>Time to rescue therapy by treatment day/week. The time to rescue therapy is calculated as the start date of the first rescue therapy minus Date of Visit 2 (Baseline (Day 1)) plus 1.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The Bullous Pemphigoid Disease Area Index (BPDAI) Score</measure>
    <time_frame>Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.</time_frame>
    <description>Change from baseline in BPDAI score at End of Treatment (EOT). Subscales for the BPDAI include the skin blister score (range 0-120), skin urticarial score (range 0-120), mucosal activity score (range 0-120), and damage score (range 0-12). Higher scores indicate greater disease activity or damage.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The Bullous Pemphigoid Disease Area Index Visual Analog Scale (BPDAI-VAS)</measure>
    <time_frame>Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.</time_frame>
    <description>Change from baseline in pruritus as evaluated by the BPDAI-VAS at End of Treatment (EOT). EOT occurs at disease control (up to 3 weeks) or at Week 3 when the subject is discontinued from treatment due to not reaching disease control. Scores for the BPDAI-VAS can range from 0 to 30, with higher scores indicating a worse condition.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Total Cumulative Steroid Exposure</measure>
    <time_frame>Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.</time_frame>
    <description>Total cumulative steroid exposure (cortisol equivalent/kg) by treatment group</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Maximum Daily Steroid Dose</measure>
    <time_frame>Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.</time_frame>
    <description>Evaluation of maximum daily steroid dose at baseline, by treatment week, and at disease control. Study Day 1 is defined as the initiation of study treatment. 1 mg/kg prednisolon(e) = 5 mg/kg cortisone.</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">6</enrollment>
  <condition>Pemphigoid, Bullous</condition>
  <arm_group>
    <arm_group_label>Mometasone furoate + AKST4290</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Mometasone furoate + Placebo</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Mometasone furoate</intervention_name>
    <description>Topical mometasone furoate</description>
    <arm_group_label>Mometasone furoate + AKST4290</arm_group_label>
    <arm_group_label>Mometasone furoate + Placebo</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>AKST4290</intervention_name>
    <description>Oral AKST4290</description>
    <arm_group_label>Mometasone furoate + AKST4290</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebo</intervention_name>
    <description>Oral placebo</description>
    <arm_group_label>Mometasone furoate + Placebo</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Clinical diagnosis of mild to moderate BP at screening.&#xD;
&#xD;
          -  Treatment naïve or initiation of whole-body high potency topical steroid treatment ≤ 7&#xD;
             days of screening (lesion-only treatment for any amount of time with any topical&#xD;
             steroids prior to screening is allowed without restriction).&#xD;
&#xD;
          -  Provide a signed and dated informed consent form in accordance with local regulations&#xD;
             and/or IRB/IEC guidelines.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Severe BP.&#xD;
&#xD;
          -  Initiation of gliptins and other treatments (e.g., etanercept, sulfasalazine,&#xD;
             furosemide, penicillin) that can trigger BP if this treatment was started within 4&#xD;
             weeks prior to screening and is considered possibly related to the onset of BP.&#xD;
&#xD;
          -  Any concomitant medications in the last 3 months prior to screening and assessed by&#xD;
             the investigator as possibly related to the development of BP.&#xD;
&#xD;
          -  Planned use of intravenous immunoglobulin or other concomitant treatments for BP&#xD;
             (i.e., doxycycline, dapsone) during the study period.&#xD;
&#xD;
          -  Use of systemic immunosuppressants (i.e., mycophenolate, azathioprine, methotrexate)&#xD;
             within 4 weeks prior to screening.&#xD;
&#xD;
          -  Treatment with rituximab within 1 year prior to screening.&#xD;
&#xD;
          -  Subjects taking warfarin.&#xD;
&#xD;
          -  Use of systemic steroids (&gt;10 mg prednisone or equivalent/day) within 14 days of first&#xD;
             dose of study agent or known diseases (other than BP) that could require the use of&#xD;
             systemic steroids within the study period.&#xD;
&#xD;
          -  Clinically relevant abnormal laboratory value at screening, including hematology,&#xD;
             blood chemistry, or urinalysis (laboratory testing may be repeated once during the&#xD;
             screening phase).&#xD;
&#xD;
          -  Participation in studies of investigational drugs must have been discontinued within&#xD;
             30 days or 5 half lives of the drug (whichever was longer) prior to screening.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>60 Years</minimum_age>
    <maximum_age>95 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Alkahest Medical Monitor</last_name>
    <role>Study Director</role>
    <affiliation>Alkahest, Inc.</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Universitätsklinikum Carl Gustav Carus Dresden Klinik und Poliklinik für Dermatologie</name>
      <address>
        <city>Dresden</city>
        <zip>01307</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Universitätsklinikum Düsseldorf Klinik für Dermatologie</name>
      <address>
        <city>Düsseldorf</city>
        <zip>40225</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Universitätsklinikum Erlangen - Hautklinik</name>
      <address>
        <city>Erlangen</city>
        <zip>91054</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Universitätsklinikum Freiburg Klinik für Dermatologie und Venerologie</name>
      <address>
        <city>Freiburg</city>
        <zip>79104</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Universitätsklinikum Schleswig-Holstein Klinik für Dermatologie, Allergologie und Venerologie (Hautklinik) Exzellenzzentrum Entzündungsmedizin</name>
      <address>
        <city>Lübeck</city>
        <zip>23538</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Universitätsklinikum Magdeburg A.ö.R. Universitätshautklinik</name>
      <address>
        <city>Magdeburg</city>
        <zip>39120</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Universitätsmedizin der Johannes Gutenberg-Universität Mainz Hautklinik Clinical Research Center (CRC)</name>
      <address>
        <city>Mainz</city>
        <zip>55131</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Universitätsklinikum Würzburg Klinik und Poliklinik für Dermatologie, Venerologie und Allergologie</name>
      <address>
        <city>Würzburg</city>
        <zip>97080</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Germany</country>
  </location_countries>
  <verification_date>September 2023</verification_date>
  <study_first_submitted>July 31, 2020</study_first_submitted>
  <study_first_submitted_qc>July 31, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">August 5, 2020</study_first_posted>
  <results_first_submitted>March 30, 2022</results_first_submitted>
  <results_first_submitted_qc>September 1, 2023</results_first_submitted_qc>
  <results_first_posted type="Actual">September 14, 2023</results_first_posted>
  <last_update_submitted>September 1, 2023</last_update_submitted>
  <last_update_submitted_qc>September 1, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">September 14, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Autoimmune disease</keyword>
  <keyword>Skin disease</keyword>
  <keyword>Vesiculobullous</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Pemphigoid, Bullous</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Mometasone Furoate</mesh_term>
  </intervention_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <provided_document_section>
    <provided_document>
      <document_type>Study Protocol</document_type>
      <document_has_protocol>Yes</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>No</document_has_sap>
      <document_date>May 15, 2020</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/35/NCT04499235/Prot_000.pdf</document_url>
    </provided_document>
    <provided_document>
      <document_type>Statistical Analysis Plan</document_type>
      <document_has_protocol>No</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>Yes</document_has_sap>
      <document_date>August 10, 2021</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/35/NCT04499235/SAP_001.pdf</document_url>
    </provided_document>
  </provided_document_section>

  <clinical_results>

    <participant_flow>
      <pre_assignment_details>Enrolled participants per the protocol are participants who are randomized to mometasone furoate + AKST4290 or mometasone furoate + placebo. Subjects screened but not randomized are not reflected in Participant Flow. The study enrolled 6 subjects of the planned 30 subjects before study prematurely terminated by the Sponsor.</pre_assignment_details>
      <group_list>
        <group group_id="P1">
          <title>Mometasone Furoate + AKST4290</title>
          <description>Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached.&#xD;
Mometasone furoate: Topical mometasone furoate&#xD;
AKST4290: Oral AKST4290</description>
        </group>
        <group group_id="P2">
          <title>Mometasone Furoate + Placebo</title>
          <description>Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.&#xD;
Mometasone furoate: Topical mometasone furoate&#xD;
Placebo: Oral 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="5"/>
                <participants group_id="P2" count="1"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="5"/>
                <participants group_id="P2" count="1"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
                <participants group_id="P2" count="0"/>
              </participants_list>
            </milestone>
          </milestone_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <group_list>
        <group group_id="B1">
          <title>Mometasone Furoate + AKST4290</title>
          <description>Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached.&#xD;
Mometasone furoate: Topical mometasone furoate&#xD;
AKST4290: Oral AKST4290</description>
        </group>
        <group group_id="B2">
          <title>Mometasone Furoate + Placebo</title>
          <description>Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.&#xD;
Mometasone furoate: Topical mometasone furoate&#xD;
Placebo: Oral 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="5"/>
            <count group_id="B2" value="1"/>
            <count group_id="B3" value="6"/>
          </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="66" spread="7.78"/>
                    <measurement group_id="B2" value="60"/>
                    <measurement group_id="B3" value="65" spread="7.38"/>
                  </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="2"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="2"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="3"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="4"/>
                  </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="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Not Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="5"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="6"/>
                  </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_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_list>
                </category>
                <category>
                  <title>Asian</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </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_list>
                </category>
                <category>
                  <title>Black or African American</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>White</title>
                  <measurement_list>
                    <measurement group_id="B1" value="5"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="6"/>
                  </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_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_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>The Percentage of Subjects Who Achieve Disease Control Without Rescue Therapy</title>
        <description>Disease control is defined as ≤ 3 new blisters/eczematous lesions/urticarial plaques/day and healing of existing blisters/eczematous lesions/urticarial plaques without requiring rescue therapy.</description>
        <time_frame>Baseline to up to 3 weeks (until disease control)</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Mometasone Furoate + AKST4290</title>
            <description>Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached.&#xD;
Mometasone furoate: Topical mometasone furoate&#xD;
AKST4290: Oral AKST4290</description>
          </group>
          <group group_id="O2">
            <title>Mometasone Furoate + Placebo</title>
            <description>Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.&#xD;
Mometasone furoate: Topical mometasone furoate&#xD;
Placebo: Oral placebo</description>
          </group>
        </group_list>
        <measure>
          <title>The Percentage of Subjects Who Achieve Disease Control Without Rescue Therapy</title>
          <description>Disease control is defined as ≤ 3 new blisters/eczematous lesions/urticarial plaques/day and healing of existing blisters/eczematous lesions/urticarial plaques without requiring rescue therapy.</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="5"/>
                <count group_id="O2" value="1"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2"/>
                    <measurement group_id="O2" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Number of Participants With TEAEs, Assessed by Seriousness and Severity</title>
        <description>Treatment-emergent AEs summarized by MedDRA coding terms; separate tabulations produced for incidence, seriousness and severity of AEs</description>
        <time_frame>Baseline to 5 weeks</time_frame>
        <population>The Safety Evaluable Set includes all randomized subjects who receive at least 1 dose of the study agent (AKST4290 or placebo)</population>
        <group_list>
          <group group_id="O1">
            <title>Mometasone Furoate + AKST4290</title>
            <description>Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached.&#xD;
Mometasone furoate: Topical mometasone furoate&#xD;
AKST4290: Oral AKST4290</description>
          </group>
          <group group_id="O2">
            <title>Mometasone Furoate + Placebo</title>
            <description>Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.&#xD;
Mometasone furoate: Topical mometasone furoate&#xD;
Placebo: Oral placebo</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants With TEAEs, Assessed by Seriousness and Severity</title>
          <description>Treatment-emergent AEs summarized by MedDRA coding terms; separate tabulations produced for incidence, seriousness and severity of AEs</description>
          <population>The Safety Evaluable Set includes all randomized subjects who receive at least 1 dose of the study agent (AKST4290 or placebo)</population>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="5"/>
                <count group_id="O2" value="1"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Subjects Reporting at Least One TEAE</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2"/>
                    <measurement group_id="O2" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Subjects Reporting at Least One Serious TEAE</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                    <measurement group_id="O2" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>TEAE by Severity: Mild</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2"/>
                    <measurement group_id="O2" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>TEAE by Severity: Moderate</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                    <measurement group_id="O2" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>TEAE by Severity: Severe</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                    <measurement group_id="O2" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Time to Disease Control</title>
        <description>Time to disease control by treatment day/week. The time to disease control is calculated as the date of disease control minus Date of Visit 2 (Baseline (Day 1)) plus 1.</description>
        <time_frame>Baseline to up to 3 weeks (until disease control)</time_frame>
        <population>Analysis population includes subjects who achieved disease control with and without rescue therapy.</population>
        <group_list>
          <group group_id="O1">
            <title>Mometasone Furoate + AKST4290</title>
            <description>Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached.&#xD;
Mometasone furoate: Topical mometasone furoate&#xD;
AKST4290: Oral AKST4290</description>
          </group>
          <group group_id="O2">
            <title>Mometasone Furoate + Placebo</title>
            <description>Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.&#xD;
Mometasone furoate: Topical mometasone furoate&#xD;
Placebo: Oral placebo</description>
          </group>
        </group_list>
        <measure>
          <title>Time to Disease Control</title>
          <description>Time to disease control by treatment day/week. The time to disease control is calculated as the date of disease control minus Date of Visit 2 (Baseline (Day 1)) plus 1.</description>
          <population>Analysis population includes subjects who achieved disease control with and without rescue therapy.</population>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="5"/>
                <count group_id="O2" value="1"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Time (Days) to Disease Control: 8</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                    <measurement group_id="O2" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Time (Days) to Disease Control: 11</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2"/>
                    <measurement group_id="O2" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Time (Days) to Disease Control: 15</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                    <measurement group_id="O2" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Time (Days) to Disease Control: 16</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                    <measurement group_id="O2" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Time (Days) to Disease Control: 22</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                    <measurement group_id="O2" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Time to Rescue Therapy</title>
        <description>Time to rescue therapy by treatment day/week. The time to rescue therapy is calculated as the start date of the first rescue therapy minus Date of Visit 2 (Baseline (Day 1)) plus 1.</description>
        <time_frame>Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Mometasone Furoate + AKST4290</title>
            <description>Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached.&#xD;
Mometasone furoate: Topical mometasone furoate&#xD;
AKST4290: Oral AKST4290</description>
          </group>
          <group group_id="O2">
            <title>Mometasone Furoate + Placebo</title>
            <description>Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.&#xD;
Mometasone furoate: Topical mometasone furoate&#xD;
Placebo: Oral placebo</description>
          </group>
        </group_list>
        <measure>
          <title>Time to Rescue Therapy</title>
          <description>Time to rescue therapy by treatment day/week. The time to rescue therapy is calculated as the start date of the first rescue therapy minus Date of Visit 2 (Baseline (Day 1)) plus 1.</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="5"/>
                <count group_id="O2" value="1"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Time (Days) to Rescue Therapy: 4</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                    <measurement group_id="O2" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Time (Days) to Rescue Therapy 8</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                    <measurement group_id="O2" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Time (Days) to Rescue Therapy 9</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                    <measurement group_id="O2" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>The Bullous Pemphigoid Disease Area Index (BPDAI) Score</title>
        <description>Change from baseline in BPDAI score at End of Treatment (EOT). Subscales for the BPDAI include the skin blister score (range 0-120), skin urticarial score (range 0-120), mucosal activity score (range 0-120), and damage score (range 0-12). Higher scores indicate greater disease activity or damage.</description>
        <time_frame>Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Mometasone Furoate + AKST4290</title>
            <description>Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached.&#xD;
Mometasone furoate: Topical mometasone furoate&#xD;
AKST4290: Oral AKST4290</description>
          </group>
          <group group_id="O2">
            <title>Mometasone Furoate + Placebo</title>
            <description>Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.&#xD;
Mometasone furoate: Topical mometasone furoate&#xD;
Placebo: Oral placebo</description>
          </group>
        </group_list>
        <measure>
          <title>The Bullous Pemphigoid Disease Area Index (BPDAI) Score</title>
          <description>Change from baseline in BPDAI score at End of Treatment (EOT). Subscales for the BPDAI include the skin blister score (range 0-120), skin urticarial score (range 0-120), mucosal activity score (range 0-120), and damage score (range 0-12). Higher scores indicate greater disease activity or damage.</description>
          <units>score 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="5"/>
                <count group_id="O2" value="1"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Total BPDAI Skin Blister Score: Baseline</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="15" spread="12.83"/>
                    <measurement group_id="O2" value="13.0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Total BPDAI Skin Blister Score: EOT</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="7.4" spread="7.02"/>
                    <measurement group_id="O2" value="8.0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Total BPDAI Skin Urticarial Score: Baseline</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="16.4" spread="9.15"/>
                    <measurement group_id="O2" value="14.0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Total BPDAI Skin Urticarial Score: EOT</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="9.2" spread="6.38"/>
                    <measurement group_id="O2" value="12.0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Total BPDAI Mucosal Activity Score: Baseline</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="3.4" spread="7.60"/>
                    <measurement group_id="O2" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Total BPDAI Mucosal Activity Score: EOT</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1.6" spread="3.58"/>
                    <measurement group_id="O2" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Total BPDAI Damage Score: Baseline</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="5.0" spread="4.18"/>
                    <measurement group_id="O2" value="9.0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Total BPDAI Damage Score: EOT</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="5.0" spread="3.81"/>
                    <measurement group_id="O2" value="8.0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>The Bullous Pemphigoid Disease Area Index Visual Analog Scale (BPDAI-VAS)</title>
        <description>Change from baseline in pruritus as evaluated by the BPDAI-VAS at End of Treatment (EOT). EOT occurs at disease control (up to 3 weeks) or at Week 3 when the subject is discontinued from treatment due to not reaching disease control. Scores for the BPDAI-VAS can range from 0 to 30, with higher scores indicating a worse condition.</description>
        <time_frame>Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Mometasone Furoate + AKST4290</title>
            <description>Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached.&#xD;
Mometasone furoate: Topical mometasone furoate&#xD;
AKST4290: Oral AKST4290</description>
          </group>
          <group group_id="O2">
            <title>Mometasone Furoate + Placebo</title>
            <description>Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.&#xD;
Mometasone furoate: Topical mometasone furoate&#xD;
Placebo: Oral placebo</description>
          </group>
        </group_list>
        <measure>
          <title>The Bullous Pemphigoid Disease Area Index Visual Analog Scale (BPDAI-VAS)</title>
          <description>Change from baseline in pruritus as evaluated by the BPDAI-VAS at End of Treatment (EOT). EOT occurs at disease control (up to 3 weeks) or at Week 3 when the subject is discontinued from treatment due to not reaching disease control. Scores for the BPDAI-VAS can range from 0 to 30, with higher scores indicating a worse condition.</description>
          <units>score 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="5"/>
                <count group_id="O2" value="1"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>BPDAI-VAS Baseline</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="19.4" spread="5.68"/>
                    <measurement group_id="O2" value="15.0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>BPDAI-VAS EOT</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="9.2" spread="3.42"/>
                    <measurement group_id="O2" value="4.0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Total Cumulative Steroid Exposure</title>
        <description>Total cumulative steroid exposure (cortisol equivalent/kg) by treatment group</description>
        <time_frame>Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.</time_frame>
        <population>Total cumulative steroid exposure is calculated based on oral route treatment from Day 1 to EOT/disease control. Subjects with no oral route treatment were excluded from analysis.</population>
        <group_list>
          <group group_id="O1">
            <title>Mometasone Furoate + AKST4290</title>
            <description>Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached.&#xD;
Mometasone furoate: Topical mometasone furoate&#xD;
AKST4290: Oral AKST4290</description>
          </group>
          <group group_id="O2">
            <title>Mometasone Furoate + Placebo</title>
            <description>Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.&#xD;
Mometasone furoate: Topical mometasone furoate&#xD;
Placebo: Oral placebo</description>
          </group>
        </group_list>
        <measure>
          <title>Total Cumulative Steroid Exposure</title>
          <description>Total cumulative steroid exposure (cortisol equivalent/kg) by treatment group</description>
          <population>Total cumulative steroid exposure is calculated based on oral route treatment from Day 1 to EOT/disease control. Subjects with no oral route treatment were excluded from analysis.</population>
          <units>mg cortisol equivalent/kg</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="3"/>
                <count group_id="O2" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="260" spread="210"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Maximum Daily Steroid Dose</title>
        <description>Evaluation of maximum daily steroid dose at baseline, by treatment week, and at disease control. Study Day 1 is defined as the initiation of study treatment. 1 mg/kg prednisolon(e) = 5 mg/kg cortisone.</description>
        <time_frame>Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.</time_frame>
        <population>The analysis population includes subjects who received systemic steroids. In the AKST4290 group, 3 subjects received systemic steroids. In the Placebo group, the single subject did not have systemic steroid exposure.</population>
        <group_list>
          <group group_id="O1">
            <title>Mometasone Furoate + AKST4290</title>
            <description>Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached.&#xD;
Mometasone furoate: Topical mometasone furoate&#xD;
AKST4290: Oral AKST4290</description>
          </group>
          <group group_id="O2">
            <title>Mometasone Furoate + Placebo</title>
            <description>Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.&#xD;
Mometasone furoate: Topical mometasone furoate&#xD;
Placebo: Oral placebo</description>
          </group>
        </group_list>
        <measure>
          <title>Maximum Daily Steroid Dose</title>
          <description>Evaluation of maximum daily steroid dose at baseline, by treatment week, and at disease control. Study Day 1 is defined as the initiation of study treatment. 1 mg/kg prednisolon(e) = 5 mg/kg cortisone.</description>
          <population>The analysis population includes subjects who received systemic steroids. In the AKST4290 group, 3 subjects received systemic steroids. In the Placebo group, the single subject did not have systemic steroid exposure.</population>
          <units>mg cortisol equivalent/kg</units>
          <param>Number</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="3"/>
                <count group_id="O2" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Maximum Daily Steroid Dose Occurred: Day 4</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="1"/>
                    <count group_id="O2" value="0"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="175"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Maximum Daily Steroid Dose Occurred: Day 8</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="1"/>
                    <count group_id="O2" value="0"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="150"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Maximum Daily Steroid Dose Occurred: Day 11</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="1"/>
                    <count group_id="O2" value="0"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="150"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>5 weeks</time_frame>
      <group_list>
        <group group_id="E1">
          <title>Mometasone Furoate + AKST4290</title>
          <description>Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached.&#xD;
Mometasone furoate: Topical mometasone furoate&#xD;
AKST4290: Oral AKST4290</description>
        </group>
        <group group_id="E2">
          <title>Mometasone Furoate + Placebo</title>
          <description>Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.&#xD;
Mometasone furoate: Topical mometasone furoate&#xD;
Placebo: Oral 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, all-cause mortality</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="5"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="1"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="5"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="1"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>COVID-19</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="5"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="1"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>0</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="2" subjects_at_risk="5"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Investigations</title>
            <event_list>
              <event>
                <sub_title>Blood potassium decreased</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="5"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="1"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Skin and subcutaneous tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Rash</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="5"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="1"/>
              </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 Clinical Trial Agreement contains language that restricts the PI from discussing or publishing Sponsor confidential and/or proprietary information. The embargo period may be extended by mutual agreement of the Sponsor and PI.</restrictive_agreement>
    </certain_agreements>
    <point_of_contact>
      <name_or_title>Head of Communications</name_or_title>
      <organization>Alkahest, Inc.</organization>
      <phone>(650) 801-0474</phone>
      <email>info@alkahest.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/NCT04499235
org study id: AKST4290-221
nct id: NCT04499235
lead sponsor: 
has dmc: Yes
is fda regulated drug: No
is fda regulated device: No
is us export: Yes
This study will evaluate the therapeutic effect and safety of adjunctive AKST4290 in subjects
      with bullous pemphigoid (BP).
This is a randomized, double-blind, placebo-controlled study to assess the therapeutic effect
      and safety of adjunctive AKST4290 in subjects with bullous pemphigoid (BP). Subjects will
      receive topical mometasone furoate cream (MFC) therapy concurrently with study agent (placebo
      or AKST4290) in an inpatient setting until disease control is reached (duration of inpatient
      stay is dependent upon individual disease course, but is estimated between 1-3 weeks).

      Subjects will receive rescue therapy at any time if their clinical condition worsens or if
      their clinical condition fails to improve by the completion of Week 1 on study treatment, as
      assessed by the investigator. Rescue therapy will consist of whole-body clobetasol propionate
      cream (CPC) (15-50g) and/or oral prednisone (0.5 mg/kg per day), as determined by the
      investigator. Subjects who receive rescue therapy will remain in the study until disease
      control, unless they are withdrawn or withdraw from participation.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Double (Participant, Investigator)
measure: The Percentage of Subjects Who Achieve Disease Control Without Rescue Therapy
time frame: Baseline to up to 3 weeks (until disease control)
description: Disease control is defined as ≤ 3 new blisters/eczematous lesions/urticarial plaques/day and healing of existing blisters/eczematous lesions/urticarial plaques without requiring rescue therapy.
measure: Number of Participants With TEAEs, Assessed by Seriousness and Severity
time frame: Baseline to 5 weeks
description: Treatment-emergent AEs summarized by MedDRA coding terms; separate tabulations produced for incidence, seriousness and severity of AEs
measure: Time to Disease Control
time frame: Baseline to up to 3 weeks (until disease control)
description: Time to disease control by treatment day/week. The time to disease control is calculated as the date of disease control minus Date of Visit 2 (Baseline (Day 1)) plus 1.
measure: Time to Rescue Therapy
time frame: Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.
description: Time to rescue therapy by treatment day/week. The time to rescue therapy is calculated as the start date of the first rescue therapy minus Date of Visit 2 (Baseline (Day 1)) plus 1.
measure: The Bullous Pemphigoid Disease Area Index (BPDAI) Score
time frame: Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.
description: Change from baseline in BPDAI score at End of Treatment (EOT). Subscales for the BPDAI include the skin blister score (range 0-120), skin urticarial score (range 0-120), mucosal activity score (range 0-120), and damage score (range 0-12). Higher scores indicate greater disease activity or damage.
measure: The Bullous Pemphigoid Disease Area Index Visual Analog Scale (BPDAI-VAS)
time frame: Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.
description: Change from baseline in pruritus as evaluated by the BPDAI-VAS at End of Treatment (EOT). EOT occurs at disease control (up to 3 weeks) or at Week 3 when the subject is discontinued from treatment due to not reaching disease control. Scores for the BPDAI-VAS can range from 0 to 30, with higher scores indicating a worse condition.
measure: Total Cumulative Steroid Exposure
time frame: Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.
description: Total cumulative steroid exposure (cortisol equivalent/kg) by treatment group
measure: Maximum Daily Steroid Dose
time frame: Baseline to up to 3 weeks (EOT). EOT occurs at disease control (assessed every day from Week 1/Day 7 up to Week 3/Day 21 +/- 2 days) or at Week 3/Day 21 +/- 2 days when the subject is discontinued from treatment due to not reaching disease control.
description: Evaluation of maximum daily steroid dose at baseline, by treatment week, and at disease control. Study Day 1 is defined as the initiation of study treatment. 1 mg/kg prednisolon(e) = 5 mg/kg cortisone.
arm group label: Mometasone furoate + AKST4290
arm group type: Experimental
description: Subjects will receive mometasone furoate concurrently with AKST4290, 400 mg twice daily, until disease control is reached.
arm group label: Mometasone furoate + Placebo
arm group type: Placebo Comparator
description: Subjects will receive mometasone furoate concurrently with placebo until disease control is reached.
intervention type: Drug
intervention name: Mometasone furoate
description: Topical mometasone furoate
arm group label: Mometasone furoate + AKST4290
arm group label: Mometasone furoate + Placebo
intervention type: Drug
intervention name: AKST4290
description: Oral AKST4290
arm group label: Mometasone furoate + AKST4290
intervention type: Drug
intervention name: Placebo
description: Oral placebo
arm group label: Mometasone furoate + Placebo
criteria: 
gender: All
minimum age: 60 Years
maximum age: 95 Years
healthy volunteers: No
last name: Alkahest Medical Monitor
role: Study Director
affiliation: Alkahest, Inc.
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: Germany
responsible party type: Sponsor
mesh term: Pemphigoid, Bullous
mesh term: Mometasone Furoate
sharing ipd: No
provided document: 
provided document: 
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
point of contact: 