<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/NCT01670110</url>
  </required_header>
  <id_info>
    <org_study_id>11-007405</org_study_id>
    <nct_id>NCT01670110</nct_id>
  </id_info>
  <brief_title>Pasireotide LAR in Severe Polycystic Liver Disease</brief_title>
  <acronym>SOM230</acronym>
  <official_title>A Randomized, Placebo Controlled Clinical Trial of SOM230 (Pasireotide LAR) In Severe Polycystic Liver Disease</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Mayo Clinic</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Mayo Clinic</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this study is to compare SOM230 treatment to placebo. The investigators will&#xD;
      also assess the efficacy and safety of SOM230 in reducing total liver volume and improving&#xD;
      quality of life.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Pasireotide (SOM230) is a novel multi-receptor-targeted analog that has high affinity for&#xD;
      four of the five SST receptor subtypes (SSTr1, SSTr2, SSTr3 and SSTr5); it has a 40-fold&#xD;
      higher affinity and 158-fold higher functional activity for the SST5 receptor than&#xD;
      octreotide. Because of its broad receptor binding profile, pasireotide may be more potent in&#xD;
      Polycystic Liver Disease (PLD) than octreotide. In this randomized double blind placebo&#xD;
      controlled trial the investigators will compare SOM230 treatment to placebo for 12 months in&#xD;
      patients with PLD. The primary endpoints will be assessed at 12 months and patients receiving&#xD;
      placebo then crossed over to SOM230, permitting all participants to receive SOM230 for the&#xD;
      subsequent two years. Magnetic resonance imaging (MRI) will be used to assess liver volume -&#xD;
      the primary endpoint, which will be assessed at baseline, end of years 1 and 3. This study&#xD;
      will assess the efficacy and safety of SOM230 in reducing total liver volume and improving&#xD;
      quality of life over 12 months. (The investigators will not be assessing efficacy at 24&#xD;
      months.) The therapy way be effective in PLD but also may prove to be effective for many more&#xD;
      patients with Polycystic Kidney Disease (PKD) which will be evaluated using eGFR and kidney&#xD;
      volume using MRI.&#xD;
&#xD;
      The investigators plan to add other sub-sites in other locations.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">August 2012</start_date>
  <completion_date type="Actual">September 2018</completion_date>
  <primary_completion_date type="Actual">September 2018</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>Crossover Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>Double (Participant, Investigator)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Change in Liver Volume</measure>
    <time_frame>baseline , 12 month</time_frame>
    <description>Percent change was calculated for liver volumes using the equation=[(12 month value-baseline value)/baseline value]*100*12/12 month</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Change in Kidney Volume</measure>
    <time_frame>baseline to 12 months</time_frame>
    <description>Percent change was calculated for kidney volumes using the equation=[(12 month value-baseline value)/baseline value]*100*12/12 month</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Percentage Change in Estimated Glomerular Filtration Rate (eGFR)</measure>
    <time_frame>Baseline, 12 months</time_frame>
    <description>eGFR was measured using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation. This value at baseline and 12 months was used to calculate the percentage change by [12 month value-baseline value)/baseline value]*100</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Percentage Change in Serum Creatinine</measure>
    <time_frame>Baseline, 12 months</time_frame>
    <description>Serum creatinine level at baseline and 12 months was used to calculate the percentage change by [12 month value-baseline value)/baseline value]*100</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Percent Change in Blood Glucose</measure>
    <time_frame>Baseline, 12 months</time_frame>
    <description>Blood glucose (mg/dLb) level at baseline and 12 months was used to calculate the percentage change by [12 month value-baseline value)/baseline value]*100</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Percentage Change in Hemoglobin A1C</measure>
    <time_frame>Baseline, 12 months</time_frame>
    <description>Hemoglobin A1C level at baseline and 12 months was used to calculate the percentage change by [12 month value-baseline value)/baseline value]*100</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Percentage Change in Heart Rate</measure>
    <time_frame>Baseline, 12 months</time_frame>
    <description>Heart rate, measured in beats per minute (BPM), at baseline and 12 months was used to calculate the percentage change by [12 month value-baseline value)/baseline value]*100</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change in Quality of Life</measure>
    <time_frame>Baseline, 12 months</time_frame>
    <description>Measured using the SF-36 health survey, which consist of eight subscales each scored on a range of 0 to 100 (0=worst imaginable, 100=best imaginable). Change calculated from baseline = 12 month value-baseline value</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">48</enrollment>
  <condition>Somatostatin Analogs</condition>
  <condition>Polycystic Liver Disease</condition>
  <condition>Autosomal Dominant Polycystic Kidney Disease</condition>
  <condition>Autosomal Dominant Polycystic Liver Disease</condition>
  <arm_group>
    <arm_group_label>Pasireotide LAR (SOM230)</arm_group_label>
    <arm_group_type>Active Comparator</arm_group_type>
    <description>Active Pasireotide LAR</description>
  </arm_group>
  <arm_group>
    <arm_group_label>placebo injection</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Pasireotide LAR</intervention_name>
    <description>Injectible, 60mg per month</description>
    <arm_group_label>Pasireotide LAR (SOM230)</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebo</intervention_name>
    <description>To be injected once per month</description>
    <arm_group_label>placebo injection</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Male or female Age ≥ 18 years.&#xD;
&#xD;
          -  Diagnosis of PLD associated with ADPKD (meeting the Modified Ravine's criteria) or&#xD;
             isolated ADPLD (defined by the criteria described by Reynolds et al)&#xD;
&#xD;
          -  Severe PLD defined as a liver volume &gt;4000mL or symptomatic disease due to mass&#xD;
             effects from hepatic cysts (must be able to undergo MRI or CT scan to determine this).&#xD;
&#xD;
          -  Not a candidate for or declining surgical intervention.&#xD;
&#xD;
          -  Capable of providing informed consent.&#xD;
&#xD;
          -  Life expectancy ≥ 12 weeks&#xD;
&#xD;
          -  Patients with a known history of impaired fasting blood glucose (glucose &gt;100 and&#xD;
             &lt;126) may be included at the discretion of the PI. These patients should be monitored&#xD;
             closely throughout the trial and antihyperglycemic treatment adjusted as necessary.&#xD;
             Patients that are deemed non eligible due to elevated glucose can be re-screened after&#xD;
             adequate medical treatment.&#xD;
&#xD;
          -  Adequate end organ function as defined by:&#xD;
&#xD;
          -  Adequate bone marrow function:&#xD;
&#xD;
               -  WBC ≥ 2.5 x 109/L&#xD;
&#xD;
               -  Absolute Neutrophil Count (ANC) ≥ 1.5 x 109/L&#xD;
&#xD;
               -  Platelets ≥ 100 x 109/L&#xD;
&#xD;
               -  Hb ≥ 9 g/dL&#xD;
&#xD;
          -  No evidence of significant liver disease:&#xD;
&#xD;
               -  Serum bilirubin ≤1.5 x ULN&#xD;
&#xD;
               -  INR &lt; 1.3&#xD;
&#xD;
               -  ALT and AST ≤ 2 x ULN&#xD;
&#xD;
          -  Estimated glomerular filtration rate (eGFR) &gt;30 ml/min/m2&#xD;
&#xD;
          -  Serum amylase and lipase ≤ 1.5 x ULN&#xD;
&#xD;
          -  Alkaline phosphatase ≤ 2.5 x ULN&#xD;
&#xD;
          -  Written informed consent obtained prior to any screening procedures&#xD;
&#xD;
          -  Willingness and ability to comply with scheduled visits, treatment plans, laboratory&#xD;
             tests and other study procedures&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Patients will be considered ineligible for this study if they meet any of the&#xD;
             following criteria:&#xD;
&#xD;
          -  Patients with a known hypersensitivity to SST analogs or any component of the&#xD;
             pasireotide LAR or SQ formulations.&#xD;
&#xD;
          -  Patients with known malabsorption syndrome, short bowel or chologenic diarrhea not&#xD;
             controlled by specific therapeutic means.&#xD;
&#xD;
          -  Patients with abnormal coagulation (PT or a PTT elevated by 30% above normal limits).&#xD;
&#xD;
          -  Patients on continuous anticoagulation therapy. Patients who were on anticoagulant&#xD;
             therapy must complete a washout period of at least 10 days and have confirmed normal&#xD;
             coagulation parameters before study inclusion.&#xD;
&#xD;
          -  Patients with symptomatic cholelithiasis.&#xD;
&#xD;
          -  Patients who are not biochemically euthyroid.&#xD;
&#xD;
          -  Patients with known history of hypothyroidism are eligible if they are on adequate and&#xD;
             stable re-placement thyroid hormone therapy for at least 3 months.&#xD;
&#xD;
          -  Serum magnesium ≥ ULN&#xD;
&#xD;
          -  QT-related exclusion criteria:&#xD;
&#xD;
          -  QTcF at screening &gt; 470 msec&#xD;
&#xD;
          -  Patients with a history of syncope or family history of idiopathic sudden death&#xD;
&#xD;
          -  Patients who have sustained or clinically significant cardiac arrhythmias&#xD;
&#xD;
          -  Risk factors for Torsades de Pointes such as hypokalemia, hypomagnesemia, cardiac&#xD;
             failure, clinically significant/symptomatic bradycardia, or high-grade AV block&#xD;
&#xD;
          -  Patients with concomitant disease(s) that could prolong QT such as autonomic&#xD;
             neuropathy (caused by diabetes, or Parkinson's disease), HIV, cirrhosis, uncontrolled&#xD;
             hypothyroidism or cardiac failure&#xD;
&#xD;
          -  Family history of long QT syndrome&#xD;
&#xD;
          -  Concomitant medications known to prolong the QT interval.&#xD;
&#xD;
          -  Potassium &lt; or = to 3.5&#xD;
&#xD;
          -  Patients who have any severe and/or uncontrolled medical conditions or other&#xD;
             conditions that could affect their participation in the study such as:&#xD;
&#xD;
          -  Patients who have Uncontrolled diabetes as defined by HbA1c&gt;8%* despite adequate&#xD;
             therapy&#xD;
&#xD;
          -  Patients with the presence of active or suspected acute or chronic uncontrolled&#xD;
             infection or with a history of immunodeficiency, including a positive HIV test result&#xD;
             (ELISA and Western blot). An HIV test will not be required; however, previous medical&#xD;
             history will be reviewed.&#xD;
&#xD;
          -  Non-malignant medical illnesses that are uncontrolled or whose control may be&#xD;
             jeopardized by the treatment with this study treatment.&#xD;
&#xD;
          -  Liver disease such as cirrhosis, decompensated liver disease, chronic active hepatitis&#xD;
             or chronic persistent hepatitis.&#xD;
&#xD;
          -  Baseline ALT or AST &gt;3x ULN&#xD;
&#xD;
          -  Patients with life-threatening autoimmune and ischemic disorders.&#xD;
&#xD;
          -  Uncontrolled hypertension&#xD;
&#xD;
          -  Patients who have a history of a primary malignancy, with the exception of locally&#xD;
             excised non-melanoma skin cancer and carcinoma in situ of uterine cervix. (Patients&#xD;
             who have had no evidence of disease from primary cancer for 3 or more years are&#xD;
             allowed to participate in the study.)&#xD;
&#xD;
          -  History of pancreatitis&#xD;
&#xD;
          -  Patients with a known history of hepatitis B or C&#xD;
&#xD;
          -  Presence of Hepatitis B surface antigen (HbsAg)&#xD;
&#xD;
          -  Presence of Hepatitis C antibody (anti-HCV)&#xD;
&#xD;
          -  Patients with a history of, or current, alcohol misuse/abuse within the past 12 months&#xD;
&#xD;
          -  Known gallbladder or bile duct disease, acute or chronic pancreatitis&#xD;
&#xD;
          -  Patients who have any current or prior medical condition that may interfere with the&#xD;
             conduct of the study or the evaluation of its results in the opinion of the&#xD;
             Investigator or the Sponsor's Medical Monitor&#xD;
&#xD;
          -  Use of an investigational drug within 1 month prior to dosing&#xD;
&#xD;
          -  Patients with a history of non-compliance to medical regimens or who are considered&#xD;
             potentially unreliable or will not be able to complete the entire study&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>100 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Marie C Hogan, MD PhD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Mayo Clinic</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Mayo Clinic</name>
      <address>
        <city>Rochester</city>
        <state>Minnesota</state>
        <zip>55905</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <link>
    <url>https://www.mayo.edu/research/clinical-trials</url>
    <description>Mayo Clinic Clinical Trials</description>
  </link>
  <verification_date>April 2020</verification_date>
  <study_first_submitted>August 17, 2012</study_first_submitted>
  <study_first_submitted_qc>August 17, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">August 21, 2012</study_first_posted>
  <results_first_submitted>September 30, 2019</results_first_submitted>
  <results_first_submitted_qc>February 18, 2020</results_first_submitted_qc>
  <results_first_posted type="Actual">March 2, 2020</results_first_posted>
  <last_update_submitted>April 30, 2020</last_update_submitted>
  <last_update_submitted_qc>April 30, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">May 1, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Mayo Clinic</investigator_affiliation>
    <investigator_full_name>Marie Hogan</investigator_full_name>
    <investigator_title>Assistant Professor of Medicine</investigator_title>
  </responsible_party>
  <keyword>Polycystic Liver Disease</keyword>
  <keyword>Autosomal dominant polycystic kidney disease</keyword>
  <keyword>Autosomal dominant polycystic liver disease</keyword>
  <keyword>Somatostatin analogs</keyword>
  <keyword>Pasireotide LAR</keyword>
  <keyword>SOM230</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Cysts</mesh_term>
    <mesh_term>Arthrogryposis</mesh_term>
    <mesh_term>Liver Diseases</mesh_term>
    <mesh_term>Kidney Diseases</mesh_term>
    <mesh_term>Polycystic Kidney Diseases</mesh_term>
    <mesh_term>Polycystic Kidney, Autosomal Dominant</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Pasireotide</mesh_term>
  </intervention_browse>
  <provided_document_section>
    <provided_document>
      <document_type>Study Protocol and Statistical Analysis Plan</document_type>
      <document_has_protocol>Yes</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>Yes</document_has_sap>
      <document_date>October 14, 2014</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/10/NCT01670110/Prot_SAP_000.pdf</document_url>
    </provided_document>
  </provided_document_section>

  <clinical_results>

    <participant_flow>
      <group_list>
        <group group_id="P1">
          <title>Pasireotide LAR (SOM230)</title>
          <description>Active Pasireotide LAR&#xD;
Pasireotide LAR: Injectible, 60mg per month</description>
        </group>
        <group group_id="P2">
          <title>Placebo Injection</title>
          <description>Placebo: To be injected once per month</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="33"/>
                <participants group_id="P2" count="15"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="29"/>
                <participants group_id="P2" count="12"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="4"/>
                <participants group_id="P2" count="3"/>
              </participants_list>
            </milestone>
          </milestone_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <group_list>
        <group group_id="B1">
          <title>Pasireotide LAR (SOM230)</title>
          <description>Active Pasireotide LAR&#xD;
Pasireotide LAR: Injectible, 60mg per month</description>
        </group>
        <group group_id="B2">
          <title>Placebo Injection</title>
          <description>Placebo: To be injected once per month</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="33"/>
            <count group_id="B2" value="15"/>
            <count group_id="B3" value="48"/>
          </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="50.16" spread="8.63"/>
                    <measurement group_id="B2" value="51.40" spread="7.97"/>
                    <measurement group_id="B3" value="50.55" spread="8.37"/>
                  </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="31"/>
                    <measurement group_id="B2" value="12"/>
                    <measurement group_id="B3" value="43"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="2"/>
                    <measurement group_id="B2" value="3"/>
                    <measurement group_id="B3" value="5"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Race/Ethnicity, Customized</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <title>White</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="29"/>
                    <measurement group_id="B2" value="15"/>
                    <measurement group_id="B3" value="44"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>African American</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Other</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="3"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="3"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Region of Enrollment</title>
          <units>participants</units>
          <param>Number</param>
          <class_list>
            <class>
              <title>United States</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="33"/>
                    <measurement group_id="B2" value="15"/>
                    <measurement group_id="B3" value="48"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Change in Liver Volume</title>
        <description>Percent change was calculated for liver volumes using the equation=[(12 month value-baseline value)/baseline value]*100*12/12 month</description>
        <time_frame>baseline , 12 month</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Pasireotide LAR (SOM230)</title>
            <description>Active Pasireotide LAR&#xD;
Pasireotide LAR: Injectible, 60mg per month</description>
          </group>
          <group group_id="O2">
            <title>Placebo Injection</title>
            <description>Placebo: To be injected once per month</description>
          </group>
        </group_list>
        <measure>
          <title>Change in Liver Volume</title>
          <description>Percent change was calculated for liver volumes using the equation=[(12 month value-baseline value)/baseline value]*100*12/12 month</description>
          <units>percentage of 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="29"/>
                <count group_id="O2" value="12"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-3.36" spread="7.33"/>
                    <measurement group_id="O2" value="6.29" spread="6.97"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <p_value>0.001</p_value>
            <method>ANCOVA</method>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Change in Kidney Volume</title>
        <description>Percent change was calculated for kidney volumes using the equation=[(12 month value-baseline value)/baseline value]*100*12/12 month</description>
        <time_frame>baseline to 12 months</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Pasireotide LAR (SOM230)</title>
            <description>Active Pasireotide LAR&#xD;
Pasireotide LAR: Injectible, 60mg per month</description>
          </group>
          <group group_id="O2">
            <title>Placebo Injection</title>
            <description>Placebo: To be injected once per month</description>
          </group>
        </group_list>
        <measure>
          <title>Change in Kidney Volume</title>
          <description>Percent change was calculated for kidney volumes using the equation=[(12 month value-baseline value)/baseline value]*100*12/12 month</description>
          <units>percentage of 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="29"/>
                <count group_id="O2" value="12"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-1.37" spread="3.52"/>
                    <measurement group_id="O2" value="3.85" spread="4.49"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <p_value>0.003</p_value>
            <method>ANCOVA</method>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Percentage Change in Estimated Glomerular Filtration Rate (eGFR)</title>
        <description>eGFR was measured using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation. This value at baseline and 12 months was used to calculate the percentage change by [12 month value-baseline value)/baseline value]*100</description>
        <time_frame>Baseline, 12 months</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Pasireotide LAR (SOM230)</title>
            <description>Active Pasireotide LAR&#xD;
Pasireotide LAR: Injectible, 60mg per month</description>
          </group>
          <group group_id="O2">
            <title>Placebo Injection</title>
            <description>Placebo: To be injected once per month</description>
          </group>
        </group_list>
        <measure>
          <title>Percentage Change in Estimated Glomerular Filtration Rate (eGFR)</title>
          <description>eGFR was measured using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation. This value at baseline and 12 months was used to calculate the percentage change by [12 month value-baseline value)/baseline value]*100</description>
          <units>percentage of 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="29"/>
                <count group_id="O2" value="12"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.3" spread="14"/>
                    <measurement group_id="O2" value="-2.2" spread="17.7"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <p_value>0.79</p_value>
            <method>t-test, 2 sided</method>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Percentage Change in Serum Creatinine</title>
        <description>Serum creatinine level at baseline and 12 months was used to calculate the percentage change by [12 month value-baseline value)/baseline value]*100</description>
        <time_frame>Baseline, 12 months</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Pasireotide LAR (SOM230)</title>
            <description>Active Pasireotide LAR&#xD;
Pasireotide LAR: Injectible, 60mg per month</description>
          </group>
          <group group_id="O2">
            <title>Placebo Injection</title>
            <description>Placebo: To be injected once per month</description>
          </group>
        </group_list>
        <measure>
          <title>Percentage Change in Serum Creatinine</title>
          <description>Serum creatinine level at baseline and 12 months was used to calculate the percentage change by [12 month value-baseline value)/baseline value]*100</description>
          <units>percentage of 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="29"/>
                <count group_id="O2" value="12"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1.8" spread="12"/>
                    <measurement group_id="O2" value="3.4" spread="15.6"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <p_value>0.79</p_value>
            <method>t-test, 2 sided</method>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Percent Change in Blood Glucose</title>
        <description>Blood glucose (mg/dLb) level at baseline and 12 months was used to calculate the percentage change by [12 month value-baseline value)/baseline value]*100</description>
        <time_frame>Baseline, 12 months</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Pasireotide LAR (SOM230)</title>
            <description>Active Pasireotide LAR&#xD;
Pasireotide LAR: Injectible, 60mg per month</description>
          </group>
          <group group_id="O2">
            <title>Placebo Injection</title>
            <description>Placebo: To be injected once per month</description>
          </group>
        </group_list>
        <measure>
          <title>Percent Change in Blood Glucose</title>
          <description>Blood glucose (mg/dLb) level at baseline and 12 months was used to calculate the percentage change by [12 month value-baseline value)/baseline value]*100</description>
          <units>percentage of 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="29"/>
                <count group_id="O2" value="12"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="39" spread="30"/>
                    <measurement group_id="O2" value="2.2" spread="15.2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <p_value>0.001</p_value>
            <method>t-test, 2 sided</method>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Percentage Change in Hemoglobin A1C</title>
        <description>Hemoglobin A1C level at baseline and 12 months was used to calculate the percentage change by [12 month value-baseline value)/baseline value]*100</description>
        <time_frame>Baseline, 12 months</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Pasireotide LAR (SOM230)</title>
            <description>Active Pasireotide LAR&#xD;
Pasireotide LAR: Injectible, 60mg per month</description>
          </group>
          <group group_id="O2">
            <title>Placebo Injection</title>
            <description>Placebo: To be injected once per month</description>
          </group>
        </group_list>
        <measure>
          <title>Percentage Change in Hemoglobin A1C</title>
          <description>Hemoglobin A1C level at baseline and 12 months was used to calculate the percentage change by [12 month value-baseline value)/baseline value]*100</description>
          <units>percentage of 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="29"/>
                <count group_id="O2" value="12"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="18" spread="11"/>
                    <measurement group_id="O2" value="1.6" spread="3.3"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <p_value>0.001</p_value>
            <method>t-test, 2 sided</method>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Percentage Change in Heart Rate</title>
        <description>Heart rate, measured in beats per minute (BPM), at baseline and 12 months was used to calculate the percentage change by [12 month value-baseline value)/baseline value]*100</description>
        <time_frame>Baseline, 12 months</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Pasireotide LAR (SOM230)</title>
            <description>Active Pasireotide LAR&#xD;
Pasireotide LAR: Injectible, 60mg per month</description>
          </group>
          <group group_id="O2">
            <title>Placebo Injection</title>
            <description>Placebo: To be injected once per month</description>
          </group>
        </group_list>
        <measure>
          <title>Percentage Change in Heart Rate</title>
          <description>Heart rate, measured in beats per minute (BPM), at baseline and 12 months was used to calculate the percentage change by [12 month value-baseline value)/baseline value]*100</description>
          <units>percentage of 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="29"/>
                <count group_id="O2" value="12"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.15" spread="0.2"/>
                    <measurement group_id="O2" value="-0.1" spread="0.1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <p_value>0.73</p_value>
            <method>t-test, 2 sided</method>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change in Quality of Life</title>
        <description>Measured using the SF-36 health survey, which consist of eight subscales each scored on a range of 0 to 100 (0=worst imaginable, 100=best imaginable). Change calculated from baseline = 12 month value-baseline value</description>
        <time_frame>Baseline, 12 months</time_frame>
        <population>Data for 19 participants in the pasireotide LAR (SOM230) group and 9 participants from the placebo injection group was available for analysis.</population>
        <group_list>
          <group group_id="O1">
            <title>Pasireotide LAR (SOM230)</title>
            <description>Active Pasireotide LAR&#xD;
Pasireotide LAR: Injectible, 60mg per month</description>
          </group>
          <group group_id="O2">
            <title>Placebo Injection</title>
            <description>Placebo: To be injected once per month</description>
          </group>
        </group_list>
        <measure>
          <title>Change in Quality of Life</title>
          <description>Measured using the SF-36 health survey, which consist of eight subscales each scored on a range of 0 to 100 (0=worst imaginable, 100=best imaginable). Change calculated from baseline = 12 month value-baseline value</description>
          <population>Data for 19 participants in the pasireotide LAR (SOM230) group and 9 participants from the placebo injection group was available for analysis.</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="19"/>
                <count group_id="O2" value="9"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Physical functioning</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="4.7" spread="16"/>
                    <measurement group_id="O2" value="-1" spread="8"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Physical role</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="7.9" spread="36"/>
                    <measurement group_id="O2" value="-3" spread="38"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Bodily pain</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="5.5" spread="22"/>
                    <measurement group_id="O2" value="7" spread="12"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>General health</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-6.2" spread="14"/>
                    <measurement group_id="O2" value="2" spread="15"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Vitality</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="4.5" spread="14"/>
                    <measurement group_id="O2" value="-2" spread="17"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Social functioning</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.0" spread="18"/>
                    <measurement group_id="O2" value="-3" spread="8"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Role emotional</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.0" spread="22"/>
                    <measurement group_id="O2" value="11" spread="5"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Mental health</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1.5" spread="12"/>
                    <measurement group_id="O2" value="-1.8" spread="11"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <groups_desc>Physical functioning</groups_desc>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <p_value>0.31</p_value>
            <method>t-test, 2 sided</method>
          </analysis>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <groups_desc>Physical role</groups_desc>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <p_value>0.48</p_value>
            <method>t-test, 2 sided</method>
          </analysis>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <groups_desc>Bodily pain</groups_desc>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <p_value>0.89</p_value>
            <method>t-test, 2 sided</method>
          </analysis>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <groups_desc>General health</groups_desc>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <p_value>0.18</p_value>
            <method>t-test, 2 sided</method>
          </analysis>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <groups_desc>Vitality</groups_desc>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <p_value>0.28</p_value>
            <method>t-test, 2 sided</method>
          </analysis>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <groups_desc>Social functioning</groups_desc>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <p_value>0.66</p_value>
            <method>t-test, 2 sided</method>
          </analysis>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <groups_desc>Role emotional</groups_desc>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <p_value>0.43</p_value>
            <method>t-test, 2 sided</method>
          </analysis>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <groups_desc>Mental health</groups_desc>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <p_value>0.51</p_value>
            <method>t-test, 2 sided</method>
          </analysis>
        </analysis_list>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>Adverse Events were collected from baseline to 12 months</time_frame>
      <group_list>
        <group group_id="E1">
          <title>Pasireotide LAR (SOM230)</title>
          <description>Active Pasireotide LAR&#xD;
Pasireotide LAR: Injectible, 60mg per month</description>
        </group>
        <group group_id="E2">
          <title>Placebo Injection</title>
          <description>Placebo: To be injected once per month</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="33"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="15"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="4" subjects_at_risk="33"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="15"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Cardiac disorders</title>
            <event_list>
              <event>
                <sub_title>Prolonged QT&gt;480msec</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="33"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="15"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Abdominal pain</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="33"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="15"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Right upper quadrant pain</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="33"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="15"/>
              </event>
              <event>
                <sub_title>Ascites</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="33"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="15"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Hepatobiliary disorders</title>
            <event_list>
              <event>
                <sub_title>Ruptured liver cyst</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="33"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="15"/>
              </event>
              <event>
                <sub_title>Elevated alkaline phosphatase</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="33"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="15"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Surgical and medical procedures</title>
            <event_list>
              <event>
                <sub_title>Abdominal hernia repair</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="33"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="15"/>
              </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="33" subjects_at_risk="33"/>
                <counts group_id="E2" subjects_affected="15" subjects_at_risk="15"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Cardiac disorders</title>
            <event_list>
              <event>
                <sub_title>Bradycardia</sub_title>
                <counts group_id="E1" events="10" subjects_affected="10" subjects_at_risk="33"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="15"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Endocrine disorders</title>
            <event_list>
              <event>
                <sub_title>Diabetes</sub_title>
                <counts group_id="E1" events="19" subjects_affected="19" subjects_at_risk="32"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="15"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Diarrhea</sub_title>
                <counts group_id="E1" events="17" subjects_affected="17" subjects_at_risk="33"/>
                <counts group_id="E2" events="8" subjects_affected="8" subjects_at_risk="15"/>
              </event>
              <event>
                <sub_title>Pain abdomen</sub_title>
                <counts group_id="E1" events="11" subjects_affected="11" subjects_at_risk="33"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="15"/>
              </event>
              <event>
                <sub_title>Nausea</sub_title>
                <counts group_id="E1" events="9" subjects_affected="9" subjects_at_risk="33"/>
                <counts group_id="E2" events="3" subjects_affected="3" subjects_at_risk="15"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Fatigue</sub_title>
                <counts group_id="E1" events="13" subjects_affected="13" subjects_at_risk="33"/>
                <counts group_id="E2" events="3" subjects_affected="3" subjects_at_risk="15"/>
              </event>
              <event>
                <sub_title>Alopecia</sub_title>
                <counts group_id="E1" events="9" subjects_affected="9" subjects_at_risk="33"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="15"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Metabolism and nutrition disorders</title>
            <event_list>
              <event>
                <sub_title>Hyperglycemia</sub_title>
                <counts group_id="E1" events="26" subjects_affected="26" subjects_at_risk="33"/>
                <counts group_id="E2" events="4" subjects_affected="4" subjects_at_risk="15"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Nervous system disorders</title>
            <event_list>
              <event>
                <sub_title>Dizziness</sub_title>
                <counts group_id="E1" events="10" subjects_affected="10" subjects_at_risk="33"/>
                <counts group_id="E2" events="3" subjects_affected="3" subjects_at_risk="15"/>
              </event>
              <event>
                <sub_title>Headache</sub_title>
                <counts group_id="E1" events="5" subjects_affected="5" subjects_at_risk="33"/>
                <counts group_id="E2" events="3" subjects_affected="3" subjects_at_risk="15"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </other_events>
    </reported_events>

    <certain_agreements>
      <pi_employee>All Principal Investigators ARE employed by the organization sponsoring the study.</pi_employee>
      <restrictive_agreement>There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed. </restrictive_agreement>
    </certain_agreements>
    <point_of_contact>
      <name_or_title>Marie C. Hogan, M.D., Ph.D.</name_or_title>
      <organization>Mayo Clinic</organization>
      <phone>507-284-3479</phone>
      <email>Hogan.Marie@mayo.edu</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/NCT01670110
org study id: 11-007405
nct id: NCT01670110
lead sponsor: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
The purpose of this study is to compare SOM230 treatment to placebo. The investigators will
      also assess the efficacy and safety of SOM230 in reducing total liver volume and improving
      quality of life.
Pasireotide (SOM230) is a novel multi-receptor-targeted analog that has high affinity for
      four of the five SST receptor subtypes (SSTr1, SSTr2, SSTr3 and SSTr5); it has a 40-fold
      higher affinity and 158-fold higher functional activity for the SST5 receptor than
      octreotide. Because of its broad receptor binding profile, pasireotide may be more potent in
      Polycystic Liver Disease (PLD) than octreotide. In this randomized double blind placebo
      controlled trial the investigators will compare SOM230 treatment to placebo for 12 months in
      patients with PLD. The primary endpoints will be assessed at 12 months and patients receiving
      placebo then crossed over to SOM230, permitting all participants to receive SOM230 for the
      subsequent two years. Magnetic resonance imaging (MRI) will be used to assess liver volume -
      the primary endpoint, which will be assessed at baseline, end of years 1 and 3. This study
      will assess the efficacy and safety of SOM230 in reducing total liver volume and improving
      quality of life over 12 months. (The investigators will not be assessing efficacy at 24
      months.) The therapy way be effective in PLD but also may prove to be effective for many more
      patients with Polycystic Kidney Disease (PKD) which will be evaluated using eGFR and kidney
      volume using MRI.

      The investigators plan to add other sub-sites in other locations.
allocation: Randomized
intervention model: Crossover Assignment
primary purpose: Treatment
masking: Double (Participant, Investigator)
measure: Change in Liver Volume
time frame: baseline , 12 month
description: Percent change was calculated for liver volumes using the equation=[(12 month value-baseline value)/baseline value]*100*12/12 month
measure: Change in Kidney Volume
time frame: baseline to 12 months
description: Percent change was calculated for kidney volumes using the equation=[(12 month value-baseline value)/baseline value]*100*12/12 month
measure: Percentage Change in Estimated Glomerular Filtration Rate (eGFR)
time frame: Baseline, 12 months
description: eGFR was measured using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation. This value at baseline and 12 months was used to calculate the percentage change by [12 month value-baseline value)/baseline value]*100
measure: Percentage Change in Serum Creatinine
time frame: Baseline, 12 months
description: Serum creatinine level at baseline and 12 months was used to calculate the percentage change by [12 month value-baseline value)/baseline value]*100
measure: Percent Change in Blood Glucose
time frame: Baseline, 12 months
description: Blood glucose (mg/dLb) level at baseline and 12 months was used to calculate the percentage change by [12 month value-baseline value)/baseline value]*100
measure: Percentage Change in Hemoglobin A1C
time frame: Baseline, 12 months
description: Hemoglobin A1C level at baseline and 12 months was used to calculate the percentage change by [12 month value-baseline value)/baseline value]*100
measure: Percentage Change in Heart Rate
time frame: Baseline, 12 months
description: Heart rate, measured in beats per minute (BPM), at baseline and 12 months was used to calculate the percentage change by [12 month value-baseline value)/baseline value]*100
measure: Change in Quality of Life
time frame: Baseline, 12 months
description: Measured using the SF-36 health survey, which consist of eight subscales each scored on a range of 0 to 100 (0=worst imaginable, 100=best imaginable). Change calculated from baseline = 12 month value-baseline value
arm group label: Pasireotide LAR (SOM230)
arm group type: Active Comparator
description: Active Pasireotide LAR
arm group label: placebo injection
arm group type: Placebo Comparator
intervention type: Drug
intervention name: Pasireotide LAR
description: Injectible, 60mg per month
arm group label: Pasireotide LAR (SOM230)
intervention type: Drug
intervention name: Placebo
description: To be injected once per month
arm group label: placebo injection
criteria: 
gender: All
minimum age: 18 Years
maximum age: 100 Years
healthy volunteers: No
last name: Marie C Hogan, MD PhD
role: Principal Investigator
affiliation: Mayo Clinic
facility: 
country: United States
url: https://www.mayo.edu/research/clinical-trials
description: Mayo Clinic Clinical Trials
responsible party type: Principal Investigator
investigator affiliation: Mayo Clinic
investigator full name: Marie Hogan
investigator title: Assistant Professor of Medicine
mesh term: Cysts
mesh term: Arthrogryposis
mesh term: Liver Diseases
mesh term: Kidney Diseases
mesh term: Polycystic Kidney Diseases
mesh term: Polycystic Kidney, Autosomal Dominant
mesh term: Pasireotide
provided document: 
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
point of contact: 