<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/NCT02978690</url>
  </required_header>
  <id_info>
    <org_study_id>1368.11</org_study_id>
    <secondary_id>2016-001236-35</secondary_id>
    <nct_id>NCT02978690</nct_id>
  </id_info>
  <brief_title>Spesolimab (BI 655130) Single Dose in Generalized Pustular Psoriasis</brief_title>
  <official_title>Multi-centre, Open-label, Single Arm, Phase I Study to Investigate Safety, Tolerability, Pharmacokinetics, Pharmacogenomics and Efficacy of a Single Intravenous Dose of Spesolimab in Patients With Active Generalized Pustular Psoriasis</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Boehringer Ingelheim</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Boehringer Ingelheim</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      This is a phase I, open label, single group study that is being performed to assess the&#xD;
      safety, tolerability, Pharmacokinetics (PK) , Pharmacogenomics (PGx) and efficacy of a single&#xD;
      dose of spesolimab in adult patients with active Generalized Pustular Psoriasis (GPP).&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">December 19, 2016</start_date>
  <completion_date type="Actual">January 10, 2018</completion_date>
  <primary_completion_date type="Actual">September 20, 2017</primary_completion_date>
  <phase>Phase 1</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Percentage of Patients With Adverse Reactions, Defined as Drug-related Adverse Events (AE)</measure>
    <time_frame>Up to 140 days from the administration of spesolimab.</time_frame>
    <description>Percentage of patients with adverse reactions, defined as drug-related Adverse Events is presented.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Percent Change From Baseline in Generalized Pustular Psoriasis Area and Severity Index (GPPASI) Total Score at Week 2</measure>
    <time_frame>At baseline and at Week 2.</time_frame>
    <description>The GPPASI is an adaptation for Generalized Pustular Psoriasis (GPP) patients of the Psoriasis Area and Severity Index (PASI), an established measure of severity and area of psoriatic lesions in patients with psoriasis. It is a tool which provides a numeric scoring for patients overall GPP disease state, ranging from 0 (no disease) to 72 (worse disease state). It is a linear combination of percent of surface area of skin that is affected and the severity of erythema, pustules, and scaling (desquamation) over four body regions. % GPPASI change from baseline=100* (GPPASI at baseline - GPPASI at post-baseline visit)/(GPPASI at baseline). For %GPPASI change, positive numbers show reduction in GPPASI with higher values representing a larger improvement or recovery of disease, while negative numbers show an increase in GPPASI, i.e. worsening of disease.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Proportion of Patients With Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) Total Score of 0 (Clear) or 1 (Almost Clear) at Week 2</measure>
    <time_frame>At Week 2.</time_frame>
    <description>GPPGA relies on clinical assessment of the GPP patient's skin presentation. It is a modified Physician Global Assessment (PGA), a physician's assessment of psoriatic lesions, which has been adapted to the evaluation of GPP patients.&#xD;
The investigator (or qualified site personnel) scores the erythema, pustules and scaling of all psoriatic lesions from 0 - 4. Each component is graded separately, the average is calculated and the final GPPGA is determined from this composite score. A lower score then indicates a lesser severity, with 0 being clear and 1 being almost clear. To receive a score of 0 or 1, the patient should be afebrile, in addition to skin presentation requirements.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score at Week 2</measure>
    <time_frame>At baseline and at Week 2.</time_frame>
    <description>Change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scale scale score at Week 2 is presented.&#xD;
The FACIT-Fatigue scale is a brief and reliable instrument for monitoring fatigue and its effects on patients. It is a comprehensive compilation of questions that measure health-related quality of life in patients with chronic illnesses. It comprises 13 questions, the responses to which are each recorded on a 5-point Likert scale. Scores range from 0 to 52, with lower scores representing greater fatigue, i.e. higher changes from baseline indicate higher improvement (compared to baseline)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Pain Visual Analog Scale (VAS) Score at Week 2</measure>
    <time_frame>At baseline and at Week 2.</time_frame>
    <description>The pain VAS is a unidimensional measure of pain intensity. It is a continuous scale comprised of a horizontal or vertical line, usually 10 centimeters (100 millimeters (mm) in length, anchored by word descriptors at each end ('no pain', 'very severe pain'). The pain VAS is self-completed by the respondent. The respondent is asked to place a line perpendicular to the VAS line at the point that represents their pain intensity. Using a ruler, the score is determined by measuring the distance (mm) on the 10-cm line between the 'no pain' anchor and the patient's mark, providing a range of scores from 0-100. A higher score indicates greater pain intensity, i.e., a negative change from baseline indicates an improvement (compared to baseline).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Area Under the Concentration-time Curve of Spesolimab in Plasma Over the Time Interval From 0 Extrapolated to Infinity (AUC0-∞)</measure>
    <time_frame>Within 1.5 hours (h) before and 0.5 h, 24 h, 48 h, 72 h, 96 h, 120 h, 144 h, 312 h, 480 h, 648 h, 1992 h, 3336 h after spesolimab administration.</time_frame>
    <description>Area under the concentration-time curve of spesolimab in plasma over the time interval from 0 extrapolated to infinity (AUC0-∞) is reported.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Maximum Measured Concentration of Spesolimab in Plasma (Cmax)</measure>
    <time_frame>Within 1.5 hours (h) before and 0.5 h, 24 h, 48 h, 72 h, 96 h, 120 h, 144 h, 312 h, 480 h, 648 h, 1992 h, 3336 h after spesolimab administration.</time_frame>
    <description>Maximum measured concentration of spesolimab in plasma (Cmax) is reported.</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">7</enrollment>
  <condition>Psoriasis</condition>
  <arm_group>
    <arm_group_label>Spesolimab</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Spesolimab</intervention_name>
    <description>Solution for infusion</description>
    <arm_group_label>Spesolimab</arm_group_label>
    <other_name>BI 655130</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion criteria:&#xD;
&#xD;
          -  Male or female patients, aged 18 to 75 years at screening,&#xD;
&#xD;
          -  A known and documented history of Generalized Pustular Psoriasis&#xD;
&#xD;
          -  Presenting with a flare of Generalized Pustular Psoriasis&#xD;
&#xD;
          -  A Generalized Pustular Psoriasis Physician Global Assessment score of at least&#xD;
             moderate severity,&#xD;
&#xD;
          -  Generalized Pustular Psoriasis patients receiving maintenance treatment with retinoids&#xD;
             and/or methotrexate for at least 4 weeks or Generalized Pustular Psoriasis patients&#xD;
             not receiving any maintenance therapy, at screening,&#xD;
&#xD;
          -  Signed and dated written informed consent prior to admission to the study,&#xD;
&#xD;
          -  Women of childbearing potential must be ready and able to use highly effective methods&#xD;
             of birth control per ICH M3 (R2) that result in a low failure rate of less than 1% per&#xD;
             year when used consistently and correctly. A list of contraception methods meeting&#xD;
             these criteria is provided in the patient information.&#xD;
&#xD;
        Male patients must be ready and able to use condoms.&#xD;
&#xD;
        - Further inclusion criteria apply&#xD;
&#xD;
        Exclusion criteria:&#xD;
&#xD;
          -  Women who are pregnant, nursing, or who plan to become pregnant while in the trial.&#xD;
&#xD;
          -  Immediate life-threatening flare of Generalized Pustular Psoriasis or requiring&#xD;
             intensive care treatment, according, to the judgement of the investigator.&#xD;
             Life-threatening complications mainly include, but are not limited to,&#xD;
             cardiovascular/cytokine driven shock, pulmonary distress,&#xD;
&#xD;
          -  Identified, ongoing serious/severe infection,&#xD;
&#xD;
          -  Acute generalized exanthematous pustulosis (AEGP)&#xD;
&#xD;
          -  Patient's clinical presentation being considered due to the differential diagnosis of&#xD;
             toxic epidermal necrosis or Stevens-Johnson syndrome,&#xD;
&#xD;
          -  Currently involved in or intending to participate in another investigational study&#xD;
             during the course of this trial,&#xD;
&#xD;
          -  Previous enrolment in this trial&#xD;
&#xD;
          -  Use of any restricted medication, or any drug considered likely to interfere with the&#xD;
             safe conduct of the study&#xD;
&#xD;
          -  Background therapy with ciclosporin within the last 30 days preceding the second&#xD;
             screening visit,&#xD;
&#xD;
          -  Severe, progressive, or uncontrolled renal, hepatic, haematological, endocrine,&#xD;
             pulmonary, cardiac, neurologic, cerebral, or psychiatric disease, or signs and&#xD;
             symptoms thereof, as judged by the investigator.&#xD;
&#xD;
          -  Known chronic or relevant acute infections including active tuberculosis, HIV or viral&#xD;
             hepatitis; QuantiFERON® tuberculosis test will be performed at screening. If the&#xD;
             result is positive, patients may participate in the study if further work up&#xD;
             (according to local practice/guidelines) establishes conclusively that the patient has&#xD;
             no evidence of active tuberculosis. If presence of latent tuberculosis is established,&#xD;
             then treatment should have been initiated and maintained according to local country&#xD;
             guidelines.&#xD;
&#xD;
          -  Patient with a transplanted organ (with exception of a corneal transplant &gt; 12 weeks&#xD;
             prior to screening) or who have ever received stem cell therapy (e.g., Prochymal).&#xD;
&#xD;
        Known history of lymphoproliferative disease, including lymphoma, or signs and symptoms&#xD;
        suggestive of possible lymphoproliferative disease, such as lymphadenopathy and/or&#xD;
        splenomegaly.&#xD;
&#xD;
          -  Any documented active or suspected malignancy or history of malignancy within 5 years&#xD;
             prior to second screening visit, except appropriately treated basal or squamous cell&#xD;
             carcinoma of the skin or in situ carcinoma of uterine cervix.&#xD;
&#xD;
          -  Evidence of a current or previous disease, medical condition (including chronic&#xD;
             alcohol or drug abuse) other than Generalized Pustular Psoriasis, surgical procedure&#xD;
             (i.e., organ transplant), medical examination finding (including vital signs and&#xD;
             electrocardiogram), or laboratory value at the second screening visit outside the&#xD;
             reference range, that is in the opinion of the investigator, is clinically significant&#xD;
             and would make the study participant unreliable to adhere to the protocol or to&#xD;
             complete the trial, compromise the safety of the patient, or compromise the quality of&#xD;
             the data,&#xD;
&#xD;
          -  Further exclusion criteria apply&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>75 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Boehringer Ingelheim</last_name>
    <role>Study Chair</role>
    <affiliation>Boehringer Ingelheim</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>HOP Saint-Louis</name>
      <address>
        <city>Paris</city>
        <zip>75010</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Nagoya City University Hospital</name>
      <address>
        <city>Aichi, Nagoya</city>
        <zip>467-8602</zip>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Pusan National Univ. Hosp</name>
      <address>
        <city>Busan</city>
        <zip>49241</zip>
        <country>Korea, Republic of</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Sultanah Aminah</name>
      <address>
        <city>Johor Bahru</city>
        <zip>80100</zip>
        <country>Malaysia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>National Taiwan University Hospital</name>
      <address>
        <city>Taipei</city>
        <zip>10002</zip>
        <country>Taiwan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hedi Chaker Hospital, Department of Dermatology</name>
      <address>
        <city>Tunisia</city>
        <zip>1053</zip>
        <country>Tunisia</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>France</country>
    <country>Japan</country>
    <country>Korea, Republic of</country>
    <country>Malaysia</country>
    <country>Taiwan</country>
    <country>Tunisia</country>
  </location_countries>
  <removed_countries>
    <country>Singapore</country>
  </removed_countries>
  <link>
    <url>http://www.mystudywindow.com</url>
    <description>Related Info</description>
  </link>
  <verification_date>September 2022</verification_date>
  <study_first_submitted>November 29, 2016</study_first_submitted>
  <study_first_submitted_qc>November 29, 2016</study_first_submitted_qc>
  <study_first_posted type="Estimate">December 1, 2016</study_first_posted>
  <results_first_submitted>September 26, 2022</results_first_submitted>
  <results_first_submitted_qc>September 26, 2022</results_first_submitted_qc>
  <results_first_posted type="Actual">August 21, 2023</results_first_posted>
  <last_update_submitted>September 26, 2022</last_update_submitted>
  <last_update_submitted_qc>September 26, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">August 21, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Psoriasis</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
    <ipd_description>Clinical studies sponsored by Boehringer Ingelheim, phases I to IV, interventional and non-interventional, are in scope for sharing of the raw clinical study data and clinical study documents. Exceptions might apply, e.g. studies in products where Boehringer Ingelheim is not the license holder; studies regarding pharmaceutical formulations and associated analytical methods, and studies pertinent to pharmacokinetics using human biomaterials; studies conducted in a single center or targeting rare diseases (in case of low number of patients and therefore limitations with anonymization).&#xD;
For more details refer to:&#xD;
https://www.mystudywindow.com/msw/datatransparency</ipd_description>
  </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>November 9, 2016</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/90/NCT02978690/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>November 6, 2017</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/90/NCT02978690/SAP_001.pdf</document_url>
    </provided_document>
  </provided_document_section>

  <clinical_results>

    <participant_flow>
      <recruitment_details>At the screening visit, patients had to be on stable maintenance treatment (for at least 4 weeks) with retinoids and/or methotrexate or not to receive any maintenance therapy at all. All patients were to use an effective birth control method throughout the trial.</recruitment_details>
      <pre_assignment_details>This exploratory, Phase I, multi-centre, multi-national trial in male and female patients with a flare of Generalised Pustular Psoriasis (GPP) applied an open-label design.</pre_assignment_details>
      <group_list>
        <group group_id="P1">
          <title>Spesolimab</title>
          <description>Patients received a single intravenous dose of 10 milligram/kilogram (mg/kg) body weight spesolimab (BI 655130) in an open label manner.&#xD;
The intravenous infusion over 60 minutes (min), could be prolonged by the investigator up to 240 min.&#xD;
Patients were to be followed up for 140 days (20 weeks) after dosing.</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="7"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="7"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
              </participants_list>
            </milestone>
          </milestone_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <population>Full Analysis Set (FAS): All patients in the treated set (TS) who had a baseline and at least one post baseline GPP Area and Severity Index (GPPASI) or GPP Physician Global Assessment (GPPGA) measurement and no important protocol violation flagged for exclusion from the FAS.</population>
      <group_list>
        <group group_id="B1">
          <title>Spesolimab</title>
          <description>Patients received a single intravenous dose of 10 milligram/kilogram (mg/kg) body weight spesolimab (BI 655130) in an open label manner.&#xD;
The intravenous infusion over 60 minutes (min), could be prolonged by the investigator up to 240 min.&#xD;
Patients were to be followed up for 140 days (20 weeks) after dosing.</description>
        </group>
      </group_list>
      <analyzed_list>
        <analyzed>
          <units>Participants</units>
          <scope>Overall</scope>
          <count_list>
            <count group_id="B1" value="7"/>
          </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="38.6" spread="13.8"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Sex: Female, Male</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Female</title>
                  <measurement_list>
                    <measurement group_id="B1" value="4"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="3"/>
                  </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_list>
                </category>
                <category>
                  <title>Not Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="7"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" 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_list>
                </category>
                <category>
                  <title>Asian</title>
                  <measurement_list>
                    <measurement group_id="B1" value="4"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Native Hawaiian or Other Pacific Islander</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Black or African American</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>White</title>
                  <measurement_list>
                    <measurement group_id="B1" value="2"/>
                  </measurement_list>
                </category>
                <category>
                  <title>More than one race</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Percentage of Patients With Adverse Reactions, Defined as Drug-related Adverse Events (AE)</title>
        <description>Percentage of patients with adverse reactions, defined as drug-related Adverse Events is presented.</description>
        <time_frame>Up to 140 days from the administration of spesolimab.</time_frame>
        <population>Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).</population>
        <group_list>
          <group group_id="O1">
            <title>Spesolimab</title>
            <description>Patients received a single intravenous dose of 10 milligram/kilogram (mg/kg) body weight spesolimab (BI 655130) in an open label manner.&#xD;
The intravenous infusion over 60 minutes (min), could be prolonged by the investigator up to 240 min.&#xD;
Patients were to be followed up for 140 days (20 weeks) after dosing.</description>
          </group>
        </group_list>
        <measure>
          <title>Percentage of Patients With Adverse Reactions, Defined as Drug-related Adverse Events (AE)</title>
          <description>Percentage of patients with adverse reactions, defined as drug-related Adverse Events is presented.</description>
          <population>Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).</population>
          <units>percentage of participants</units>
          <param>Number</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="7"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="57.1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Percent Change From Baseline in Generalized Pustular Psoriasis Area and Severity Index (GPPASI) Total Score at Week 2</title>
        <description>The GPPASI is an adaptation for Generalized Pustular Psoriasis (GPP) patients of the Psoriasis Area and Severity Index (PASI), an established measure of severity and area of psoriatic lesions in patients with psoriasis. It is a tool which provides a numeric scoring for patients overall GPP disease state, ranging from 0 (no disease) to 72 (worse disease state). It is a linear combination of percent of surface area of skin that is affected and the severity of erythema, pustules, and scaling (desquamation) over four body regions. % GPPASI change from baseline=100* (GPPASI at baseline - GPPASI at post-baseline visit)/(GPPASI at baseline). For %GPPASI change, positive numbers show reduction in GPPASI with higher values representing a larger improvement or recovery of disease, while negative numbers show an increase in GPPASI, i.e. worsening of disease.</description>
        <time_frame>At baseline and at Week 2.</time_frame>
        <population>Full Analysis Set Last Observation Carry forward (FAS LOCF). Full Analysis Set (FAS): All patients in the treated set (TS) who had a baseline and at least one post baseline GPP Area and Severity Index (GPPASI) or GPP Physician Global Assessment (GPPGA) measurement and no important protocol violation flagged for exclusion from the FAS. LOCF: The last available value, including baseline, was carried forward to all subsequent visits within the on-treatment period at which a measurement was missing.</population>
        <group_list>
          <group group_id="O1">
            <title>Spesolimab</title>
            <description>Patients received a single intravenous dose of 10 milligram/kilogram (mg/kg) body weight spesolimab (BI 655130) in an open label manner.&#xD;
The intravenous infusion over 60 minutes (min), could be prolonged by the investigator up to 240 min.&#xD;
Patients were to be followed up for 140 days (20 weeks) after dosing.</description>
          </group>
        </group_list>
        <measure>
          <title>Percent Change From Baseline in Generalized Pustular Psoriasis Area and Severity Index (GPPASI) Total Score at Week 2</title>
          <description>The GPPASI is an adaptation for Generalized Pustular Psoriasis (GPP) patients of the Psoriasis Area and Severity Index (PASI), an established measure of severity and area of psoriatic lesions in patients with psoriasis. It is a tool which provides a numeric scoring for patients overall GPP disease state, ranging from 0 (no disease) to 72 (worse disease state). It is a linear combination of percent of surface area of skin that is affected and the severity of erythema, pustules, and scaling (desquamation) over four body regions. % GPPASI change from baseline=100* (GPPASI at baseline - GPPASI at post-baseline visit)/(GPPASI at baseline). For %GPPASI change, positive numbers show reduction in GPPASI with higher values representing a larger improvement or recovery of disease, while negative numbers show an increase in GPPASI, i.e. worsening of disease.</description>
          <population>Full Analysis Set Last Observation Carry forward (FAS LOCF). Full Analysis Set (FAS): All patients in the treated set (TS) who had a baseline and at least one post baseline GPP Area and Severity Index (GPPASI) or GPP Physician Global Assessment (GPPGA) measurement and no important protocol violation flagged for exclusion from the FAS. LOCF: The last available value, including baseline, was carried forward to all subsequent visits within the on-treatment period at which a measurement was missing.</population>
          <units>percent GPPASI change</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="7"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="73.2" spread="16.2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Proportion of Patients With Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) Total Score of 0 (Clear) or 1 (Almost Clear) at Week 2</title>
        <description>GPPGA relies on clinical assessment of the GPP patient's skin presentation. It is a modified Physician Global Assessment (PGA), a physician's assessment of psoriatic lesions, which has been adapted to the evaluation of GPP patients.&#xD;
The investigator (or qualified site personnel) scores the erythema, pustules and scaling of all psoriatic lesions from 0 - 4. Each component is graded separately, the average is calculated and the final GPPGA is determined from this composite score. A lower score then indicates a lesser severity, with 0 being clear and 1 being almost clear. To receive a score of 0 or 1, the patient should be afebrile, in addition to skin presentation requirements.</description>
        <time_frame>At Week 2.</time_frame>
        <population>Full Analysis Set No Response Imputed (FAS NRI). Full Analysis Set (FAS): All patients in the treated set (TS) who had a baseline and at least one post baseline GPP Area and Severity Index (GPPASI) or GPP Physician Global Assessment (GPPGA) measurement and no important protocol violation flagged for exclusion from the FAS. NRI: If there were data at the visits both immediately before and immediately after the visit with a missing outcome, a failure to achieve response imputed.</population>
        <group_list>
          <group group_id="O1">
            <title>Spesolimab</title>
            <description>Patients received a single intravenous dose of 10 milligram/kilogram (mg/kg) body weight spesolimab (BI 655130) in an open label manner.&#xD;
The intravenous infusion over 60 minutes (min), could be prolonged by the investigator up to 240 min.&#xD;
Patients were to be followed up for 140 days (20 weeks) after dosing.</description>
          </group>
        </group_list>
        <measure>
          <title>Proportion of Patients With Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) Total Score of 0 (Clear) or 1 (Almost Clear) at Week 2</title>
          <description>GPPGA relies on clinical assessment of the GPP patient's skin presentation. It is a modified Physician Global Assessment (PGA), a physician's assessment of psoriatic lesions, which has been adapted to the evaluation of GPP patients.&#xD;
The investigator (or qualified site personnel) scores the erythema, pustules and scaling of all psoriatic lesions from 0 - 4. Each component is graded separately, the average is calculated and the final GPPGA is determined from this composite score. A lower score then indicates a lesser severity, with 0 being clear and 1 being almost clear. To receive a score of 0 or 1, the patient should be afebrile, in addition to skin presentation requirements.</description>
          <population>Full Analysis Set No Response Imputed (FAS NRI). Full Analysis Set (FAS): All patients in the treated set (TS) who had a baseline and at least one post baseline GPP Area and Severity Index (GPPASI) or GPP Physician Global Assessment (GPPGA) measurement and no important protocol violation flagged for exclusion from the FAS. NRI: If there were data at the visits both immediately before and immediately after the visit with a missing outcome, a failure to achieve response imputed.</population>
          <units>proportion of patients</units>
          <param>Number</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="7"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.714"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score at Week 2</title>
        <description>Change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scale scale score at Week 2 is presented.&#xD;
The FACIT-Fatigue scale is a brief and reliable instrument for monitoring fatigue and its effects on patients. It is a comprehensive compilation of questions that measure health-related quality of life in patients with chronic illnesses. It comprises 13 questions, the responses to which are each recorded on a 5-point Likert scale. Scores range from 0 to 52, with lower scores representing greater fatigue, i.e. higher changes from baseline indicate higher improvement (compared to baseline)</description>
        <time_frame>At baseline and at Week 2.</time_frame>
        <population>Full Analysis Set Last Observation Carry forward (FAS LOCF). Full Analysis Set (FAS): All patients in the treated set (TS) who had a baseline and at least one post baseline GPP Area and Severity Index (GPPASI) or GPP Physician Global Assessment (GPPGA) measurement and no important protocol violation flagged for exclusion from the FAS. LOCF: The last available value, including baseline, was carried forward to all subsequent visits within the on-treatment period at which a measurement was missing.</population>
        <group_list>
          <group group_id="O1">
            <title>Spesolimab</title>
            <description>Patients received a single intravenous dose of 10 milligram/kilogram (mg/kg) body weight spesolimab (BI 655130) in an open label manner.&#xD;
The intravenous infusion over 60 minutes (min), could be prolonged by the investigator up to 240 min.&#xD;
Patients were to be followed up for 140 days (20 weeks) after dosing.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score at Week 2</title>
          <description>Change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scale scale score at Week 2 is presented.&#xD;
The FACIT-Fatigue scale is a brief and reliable instrument for monitoring fatigue and its effects on patients. It is a comprehensive compilation of questions that measure health-related quality of life in patients with chronic illnesses. It comprises 13 questions, the responses to which are each recorded on a 5-point Likert scale. Scores range from 0 to 52, with lower scores representing greater fatigue, i.e. higher changes from baseline indicate higher improvement (compared to baseline)</description>
          <population>Full Analysis Set Last Observation Carry forward (FAS LOCF). Full Analysis Set (FAS): All patients in the treated set (TS) who had a baseline and at least one post baseline GPP Area and Severity Index (GPPASI) or GPP Physician Global Assessment (GPPGA) measurement and no important protocol violation flagged for exclusion from the FAS. LOCF: The last available value, including baseline, was carried forward to all subsequent visits within the on-treatment period at which a measurement was missing.</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="7"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="12.3" spread="10.1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline in Pain Visual Analog Scale (VAS) Score at Week 2</title>
        <description>The pain VAS is a unidimensional measure of pain intensity. It is a continuous scale comprised of a horizontal or vertical line, usually 10 centimeters (100 millimeters (mm) in length, anchored by word descriptors at each end ('no pain', 'very severe pain'). The pain VAS is self-completed by the respondent. The respondent is asked to place a line perpendicular to the VAS line at the point that represents their pain intensity. Using a ruler, the score is determined by measuring the distance (mm) on the 10-cm line between the 'no pain' anchor and the patient's mark, providing a range of scores from 0-100. A higher score indicates greater pain intensity, i.e., a negative change from baseline indicates an improvement (compared to baseline).</description>
        <time_frame>At baseline and at Week 2.</time_frame>
        <population>Full Analysis Set Last Observation Carry forward (FAS LOCF). Full Analysis Set (FAS): All patients in the treated set (TS) who had a baseline and at least one post baseline GPP Area and Severity Index (GPPASI) or GPP Physician Global Assessment (GPPGA) measurement and no important protocol violation flagged for exclusion from the FAS. LOCF: The last available value, including baseline, was carried forward to all subsequent visits within the on-treatment period at which a measurement was missing.</population>
        <group_list>
          <group group_id="O1">
            <title>Spesolimab</title>
            <description>Patients received a single intravenous dose of 10 milligram/kilogram (mg/kg) body weight spesolimab (BI 655130) in an open label manner.&#xD;
The intravenous infusion over 60 minutes (min), could be prolonged by the investigator up to 240 min.&#xD;
Patients were to be followed up for 140 days (20 weeks) after dosing.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Pain Visual Analog Scale (VAS) Score at Week 2</title>
          <description>The pain VAS is a unidimensional measure of pain intensity. It is a continuous scale comprised of a horizontal or vertical line, usually 10 centimeters (100 millimeters (mm) in length, anchored by word descriptors at each end ('no pain', 'very severe pain'). The pain VAS is self-completed by the respondent. The respondent is asked to place a line perpendicular to the VAS line at the point that represents their pain intensity. Using a ruler, the score is determined by measuring the distance (mm) on the 10-cm line between the 'no pain' anchor and the patient's mark, providing a range of scores from 0-100. A higher score indicates greater pain intensity, i.e., a negative change from baseline indicates an improvement (compared to baseline).</description>
          <population>Full Analysis Set Last Observation Carry forward (FAS LOCF). Full Analysis Set (FAS): All patients in the treated set (TS) who had a baseline and at least one post baseline GPP Area and Severity Index (GPPASI) or GPP Physician Global Assessment (GPPGA) measurement and no important protocol violation flagged for exclusion from the FAS. LOCF: The last available value, including baseline, was carried forward to all subsequent visits within the on-treatment period at which a measurement was missing.</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="7"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-45.9" spread="32.3"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Area Under the Concentration-time Curve of Spesolimab in Plasma Over the Time Interval From 0 Extrapolated to Infinity (AUC0-∞)</title>
        <description>Area under the concentration-time curve of spesolimab in plasma over the time interval from 0 extrapolated to infinity (AUC0-∞) is reported.</description>
        <time_frame>Within 1.5 hours (h) before and 0.5 h, 24 h, 48 h, 72 h, 96 h, 120 h, 144 h, 312 h, 480 h, 648 h, 1992 h, 3336 h after spesolimab administration.</time_frame>
        <population>Pharmacokinetic (PK) parameter set (PKS): All patients in the treated set (TS) who provided at least one secondary PK endpoint not flagged for exclusion due to a protocol violation relevant to the evaluation of PK (to be decided no later than at the Report Planning Meeting) or due to PK non-evaluability (as revealed during data analysis).</population>
        <group_list>
          <group group_id="O1">
            <title>Spesolimab</title>
            <description>Patients received a single intravenous dose of 10 milligram/kilogram (mg/kg) body weight spesolimab (BI 655130) in an open label manner.&#xD;
The intravenous infusion over 60 minutes (min), could be prolonged by the investigator up to 240 min.&#xD;
Patients were to be followed up for 140 days (20 weeks) after dosing.</description>
          </group>
        </group_list>
        <measure>
          <title>Area Under the Concentration-time Curve of Spesolimab in Plasma Over the Time Interval From 0 Extrapolated to Infinity (AUC0-∞)</title>
          <description>Area under the concentration-time curve of spesolimab in plasma over the time interval from 0 extrapolated to infinity (AUC0-∞) is reported.</description>
          <population>Pharmacokinetic (PK) parameter set (PKS): All patients in the treated set (TS) who provided at least one secondary PK endpoint not flagged for exclusion due to a protocol violation relevant to the evaluation of PK (to be decided no later than at the Report Planning Meeting) or due to PK non-evaluability (as revealed during data analysis).</population>
          <units>microgram (μg)·day/ milliliter (mL)</units>
          <param>Geometric Mean</param>
          <dispersion>Geometric Coefficient of Variation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="6"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2350" spread="41.1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Maximum Measured Concentration of Spesolimab in Plasma (Cmax)</title>
        <description>Maximum measured concentration of spesolimab in plasma (Cmax) is reported.</description>
        <time_frame>Within 1.5 hours (h) before and 0.5 h, 24 h, 48 h, 72 h, 96 h, 120 h, 144 h, 312 h, 480 h, 648 h, 1992 h, 3336 h after spesolimab administration.</time_frame>
        <population>Pharmacokinetic (PK) parameter set (PKS): All patients in the treated set (TS) who provided at least one secondary PK endpoint not flagged for exclusion due to a protocol violation relevant to the evaluation of PK (to be decided no later than at the Report Planning Meeting) or due to PK non-evaluability (as revealed during data analysis).</population>
        <group_list>
          <group group_id="O1">
            <title>Spesolimab</title>
            <description>Patients received a single intravenous dose of 10 milligram/kilogram (mg/kg) body weight spesolimab (BI 655130) in an open label manner.&#xD;
The intravenous infusion over 60 minutes (min), could be prolonged by the investigator up to 240 min.&#xD;
Patients were to be followed up for 140 days (20 weeks) after dosing.</description>
          </group>
        </group_list>
        <measure>
          <title>Maximum Measured Concentration of Spesolimab in Plasma (Cmax)</title>
          <description>Maximum measured concentration of spesolimab in plasma (Cmax) is reported.</description>
          <population>Pharmacokinetic (PK) parameter set (PKS): All patients in the treated set (TS) who provided at least one secondary PK endpoint not flagged for exclusion due to a protocol violation relevant to the evaluation of PK (to be decided no later than at the Report Planning Meeting) or due to PK non-evaluability (as revealed during data analysis).</population>
          <units>microgram (μg)/milliliter (mL)</units>
          <param>Geometric Mean</param>
          <dispersion>Geometric Coefficient of Variation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="6"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="203" spread="11.1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>Up to 140 days from the administration of spesolimab.</time_frame>
      <desc>Treated Set (TS): All patients who entered the trial and received the trial medication on Day 1 (Visit 3).</desc>
      <group_list>
        <group group_id="E1">
          <title>Spesolimab</title>
          <description>Patients received a single intravenous dose of 10 milligram/kilogram (mg/kg) body weight spesolimab (BI 655130) in an open label manner.&#xD;
The intravenous infusion over 60 minutes (min), could be prolonged by the investigator up to 240 min.&#xD;
Patients were to be followed up for 140 days (20 weeks) after dosing.</description>
        </group>
      </group_list>
      <serious_events>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, all-cause mortality</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>5</frequency_threshold>
        <default_vocab>MedDRA 20.1</default_vocab>
        <default_assessment>Systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, other adverse events</sub_title>
                <counts group_id="E1" subjects_affected="7" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Blood and lymphatic system disorders</title>
            <event_list>
              <event>
                <sub_title>Eosinophilia</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Abdominal pain</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Diarrhoea</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Vomiting</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Asthenia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Chills</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Oedema peripheral</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Pain</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Pyrexia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Pharyngitis</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Rash pustular</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Upper respiratory tract infection</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Urinary tract infection</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Viral tonsillitis</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Injury, poisoning and procedural complications</title>
            <event_list>
              <event>
                <sub_title>Infusion related reaction</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Investigations</title>
            <event_list>
              <event>
                <sub_title>Blood creatine phosphokinase increased</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Musculoskeletal and connective tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Arthralgia</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Pain in extremity</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Renal and urinary disorders</title>
            <event_list>
              <event>
                <sub_title>Haematuria</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Nephrolithiasis</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Renal cyst</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Reproductive system and breast disorders</title>
            <event_list>
              <event>
                <sub_title>Pelvic pain</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Respiratory, thoracic and mediastinal disorders</title>
            <event_list>
              <event>
                <sub_title>Oropharyngeal pain</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Skin and subcutaneous tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Eczema</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Pustular psoriasis</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Skin fragility</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Skin striae</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </other_events>
    </reported_events>

    <certain_agreements>
      <pi_employee>Principal Investigators are NOT employed by the organization sponsoring the study.</pi_employee>
      <restrictive_agreement>Boehringer Ingelheim (BI) acknowledges that investigators have the right to publish the study results. Investigators shall provide BI with a copy of any publication or presentation for review prior to any submission. Such review will be done with regard to proprietary information, information related to patentable inventions, medical, scientific, and statistical accuracy within 60 days. BI may request a delay of the publication in order to protect BI's intellectual property rights.</restrictive_agreement>
    </certain_agreements>
    <limitations_and_caveats>The results should be interpreted with caution given the limited sample size.</limitations_and_caveats>
    <point_of_contact>
      <name_or_title>Boehringer Ingelheim, Call Center</name_or_title>
      <organization>Boehringer Ingelheim</organization>
      <phone>1-800-243-0127</phone>
      <email>clintriage.rdg@boehringer-ingelheim.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/NCT02978690
org study id: 1368.11
secondary id: 2016-001236-35
nct id: NCT02978690
lead sponsor: 
has dmc: No
This is a phase I, open label, single group study that is being performed to assess the
      safety, tolerability, Pharmacokinetics (PK) , Pharmacogenomics (PGx) and efficacy of a single
      dose of spesolimab in adult patients with active Generalized Pustular Psoriasis (GPP).
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Percentage of Patients With Adverse Reactions, Defined as Drug-related Adverse Events (AE)
time frame: Up to 140 days from the administration of spesolimab.
description: Percentage of patients with adverse reactions, defined as drug-related Adverse Events is presented.
measure: Percent Change From Baseline in Generalized Pustular Psoriasis Area and Severity Index (GPPASI) Total Score at Week 2
time frame: At baseline and at Week 2.
description: The GPPASI is an adaptation for Generalized Pustular Psoriasis (GPP) patients of the Psoriasis Area and Severity Index (PASI), an established measure of severity and area of psoriatic lesions in patients with psoriasis. It is a tool which provides a numeric scoring for patients overall GPP disease state, ranging from 0 (no disease) to 72 (worse disease state). It is a linear combination of percent of surface area of skin that is affected and the severity of erythema, pustules, and scaling (desquamation) over four body regions. % GPPASI change from baseline=100* (GPPASI at baseline - GPPASI at post-baseline visit)/(GPPASI at baseline). For %GPPASI change, positive numbers show reduction in GPPASI with higher values representing a larger improvement or recovery of disease, while negative numbers show an increase in GPPASI, i.e. worsening of disease.
measure: Proportion of Patients With Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) Total Score of 0 (Clear) or 1 (Almost Clear) at Week 2
time frame: At Week 2.
description: GPPGA relies on clinical assessment of the GPP patient's skin presentation. It is a modified Physician Global Assessment (PGA), a physician's assessment of psoriatic lesions, which has been adapted to the evaluation of GPP patients.
The investigator (or qualified site personnel) scores the erythema, pustules and scaling of all psoriatic lesions from 0 - 4. Each component is graded separately, the average is calculated and the final GPPGA is determined from this composite score. A lower score then indicates a lesser severity, with 0 being clear and 1 being almost clear. To receive a score of 0 or 1, the patient should be afebrile, in addition to skin presentation requirements.
measure: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score at Week 2
time frame: At baseline and at Week 2.
description: Change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scale scale score at Week 2 is presented.
The FACIT-Fatigue scale is a brief and reliable instrument for monitoring fatigue and its effects on patients. It is a comprehensive compilation of questions that measure health-related quality of life in patients with chronic illnesses. It comprises 13 questions, the responses to which are each recorded on a 5-point Likert scale. Scores range from 0 to 52, with lower scores representing greater fatigue, i.e. higher changes from baseline indicate higher improvement (compared to baseline)
measure: Change From Baseline in Pain Visual Analog Scale (VAS) Score at Week 2
time frame: At baseline and at Week 2.
description: The pain VAS is a unidimensional measure of pain intensity. It is a continuous scale comprised of a horizontal or vertical line, usually 10 centimeters (100 millimeters (mm) in length, anchored by word descriptors at each end ('no pain', 'very severe pain'). The pain VAS is self-completed by the respondent. The respondent is asked to place a line perpendicular to the VAS line at the point that represents their pain intensity. Using a ruler, the score is determined by measuring the distance (mm) on the 10-cm line between the 'no pain' anchor and the patient's mark, providing a range of scores from 0-100. A higher score indicates greater pain intensity, i.e., a negative change from baseline indicates an improvement (compared to baseline).
measure: Area Under the Concentration-time Curve of Spesolimab in Plasma Over the Time Interval From 0 Extrapolated to Infinity (AUC0-∞)
time frame: Within 1.5 hours (h) before and 0.5 h, 24 h, 48 h, 72 h, 96 h, 120 h, 144 h, 312 h, 480 h, 648 h, 1992 h, 3336 h after spesolimab administration.
description: Area under the concentration-time curve of spesolimab in plasma over the time interval from 0 extrapolated to infinity (AUC0-∞) is reported.
measure: Maximum Measured Concentration of Spesolimab in Plasma (Cmax)
time frame: Within 1.5 hours (h) before and 0.5 h, 24 h, 48 h, 72 h, 96 h, 120 h, 144 h, 312 h, 480 h, 648 h, 1992 h, 3336 h after spesolimab administration.
description: Maximum measured concentration of spesolimab in plasma (Cmax) is reported.
arm group label: Spesolimab
arm group type: Experimental
intervention type: Drug
intervention name: Spesolimab
description: Solution for infusion
arm group label: Spesolimab
other name: BI 655130
criteria: 
gender: All
minimum age: 18 Years
maximum age: 75 Years
healthy volunteers: No
last name: Boehringer Ingelheim
role: Study Chair
affiliation: Boehringer Ingelheim
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: France
country: Japan
country: Korea, Republic of
country: Malaysia
country: Taiwan
country: Tunisia
country: Singapore
url: http://www.mystudywindow.com
description: Related Info
responsible party type: Sponsor
mesh term: Psoriasis
sharing ipd: No
ipd description: Clinical studies sponsored by Boehringer Ingelheim, phases I to IV, interventional and non-interventional, are in scope for sharing of the raw clinical study data and clinical study documents. Exceptions might apply, e.g. studies in products where Boehringer Ingelheim is not the license holder; studies regarding pharmaceutical formulations and associated analytical methods, and studies pertinent to pharmacokinetics using human biomaterials; studies conducted in a single center or targeting rare diseases (in case of low number of patients and therefore limitations with anonymization).
For more details refer to:
https://www.mystudywindow.com/msw/datatransparency
provided document: 
provided document: 
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
limitations and caveats: The results should be interpreted with caution given the limited sample size.
point of contact: 