<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/NCT01665391</url>
  </required_header>
  <id_info>
    <org_study_id>GC1008FSGS03110</org_study_id>
    <secondary_id>2010-019545-25</secondary_id>
    <secondary_id>U1111-1139-9082</secondary_id>
    <secondary_id>DRI12792</secondary_id>
    <nct_id>NCT01665391</nct_id>
  </id_info>
  <brief_title>A Study of Fresolimumab in Patients With Steroid-Resistant Primary Focal Segmental Glomerulosclerosis (FSGS)</brief_title>
  <acronym>FSGS</acronym>
  <official_title>A Phase 2, Multicenter, Double-Blind, Parallel Dosing, Randomized Study of Fresolimumab or Placebo in Patients With Steroid-Resistant Primary Focal Segmental Glomerulosclerosis</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Genzyme, a Sanofi Company</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Sanofi</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The primary objectives of this trial are as follows:&#xD;
&#xD;
        -  to compare the achievement of a partial remission (PR) or complete remission (CR) in&#xD;
           urinary protein: creatinine ratio (Up/c ratio) in patients treated with fresolimumab&#xD;
           versus placebo&#xD;
&#xD;
        -  to compare the safety profile of patients treated with fresolimumab versus placebo&#xD;
&#xD;
      The secondary objectives are as follows:&#xD;
&#xD;
        -  To compare the reduction in proteinuria in patients treated with fresolimumab versus&#xD;
           placebo&#xD;
&#xD;
        -  To evaluate fresolimumab dose-dependent reduction in proteinuria&#xD;
&#xD;
        -  To compare the change in renal function (estimated glomerular filtration rate [eGFR]) in&#xD;
           patients treated with fresolimumab versus placebo&#xD;
&#xD;
        -  To evaluate the multiple-dose pharmacokinetics of fresolimumab&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date>August 2012</start_date>
  <completion_date type="Actual">November 2014</completion_date>
  <primary_completion_date type="Actual">June 2014</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>Double (Participant, Investigator)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Percentage of patients achieving partial remission (PR) or complete remission (CR) in urinary protein: creatinine ratio (Up/c ratio)</measure>
    <time_frame>Up to Day 112</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Number of patients reporting adverse events (AEs), serious adverse events (SAEs), and medical events of interest (MEOIs)</measure>
    <time_frame>Up to Day 112</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>Percentage of patients achieving CR in Up/c ratio</measure>
    <time_frame>Up to Day 112</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Percentage of patients achieving PR in Up/c ratio</measure>
    <time_frame>Up to Day 112</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from baseline in Up/c ratio and urinary protein excretion rate</measure>
    <time_frame>Up to Day 112</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Time to first PR or CR</measure>
    <time_frame>Up to Day 112</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from baseline in eGFR (estimated glomerular filtration rate)</measure>
    <time_frame>Up to Day 112</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Percentage of patients achieving PR or CR with stable eGFR (estimated glomerular filtration rate)</measure>
    <time_frame>Up to Day 112</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Mean Fresolimumab serum concentration at each sample collection time point</measure>
    <time_frame>Up to Day 252</time_frame>
  </secondary_outcome>
  <number_of_arms>3</number_of_arms>
  <enrollment type="Actual">36</enrollment>
  <condition>Primary Focal Segmental Glomerulosclerosis</condition>
  <arm_group>
    <arm_group_label>fresolimumab 1 mg/kg total body weight</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <arm_group>
    <arm_group_label>fresolimumab 4 mg/kg total body weight</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <arm_group>
    <arm_group_label>Placebo</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>fresolimumab</intervention_name>
    <description>1 mg/kg total body weight administered intravenous (IV)</description>
    <arm_group_label>fresolimumab 1 mg/kg total body weight</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>fresolimumab</intervention_name>
    <description>4 mg/kg total body weight administered intravenous (IV)</description>
    <arm_group_label>fresolimumab 4 mg/kg total body weight</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebo</intervention_name>
    <description>Placebo administered to match active treatment group</description>
    <arm_group_label>Placebo</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  The patient's renal biopsy is consistent with the diagnosis of primary Focal Segmental&#xD;
             Glomerulosclerosis (FSGS) including all histological subtypes.&#xD;
&#xD;
          -  The patient has an eGFR ≥ 30 mL/min/1.73 m2&#xD;
&#xD;
          -  The patient has a urinary total protein:creatinine ratio ≥ 3 mg protein/mg creatinine&#xD;
&#xD;
          -  In the opinion of the Investigator, the patient has steroid-resistant FSGS. The&#xD;
             patient must have been treated for FSGS with a course of high-dose steroid therapy for&#xD;
             a minimum of 4 weeks&#xD;
&#xD;
          -  The patient has been treated with an ACEi (angiotensin converting enzyme inhibitor)&#xD;
             and/or ARB (angiotensin receptor blocker) at a stable dose for a minimum of 4 weeks&#xD;
             prior to Visit 2 (treatment start)&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  The patient has FSGS which in the Investigator's opinion is secondary to another&#xD;
             condition&#xD;
&#xD;
          -  The patient has been taking prednisone at a dose &gt; 10 mg/day (or equivalent dose of an&#xD;
             alternative glucocorticoid) within 4 weeks prior to Visit 1 (Screening Visit).&#xD;
&#xD;
          -  The patient has received any other systemically administered immunosuppressive drugs&#xD;
             (other than glucocorticoids) within 8 weeks prior to Visit 1.&#xD;
&#xD;
          -  The patient has received rituximab within 6 months prior to Visit 1.&#xD;
&#xD;
          -  The patient has a history of organ transplantation.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Medical Monitor</last_name>
    <role>Study Director</role>
    <affiliation>Genzyme, a Sanofi Company</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Investigational Site Number 4008</name>
      <address>
        <city>Birmingham</city>
        <state>Alabama</state>
        <zip>35205</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4029</name>
      <address>
        <city>Phoenix</city>
        <state>Arizona</state>
        <zip>85032</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4009</name>
      <address>
        <city>San Francisco</city>
        <state>California</state>
        <zip>94143</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4023</name>
      <address>
        <city>Stanford</city>
        <state>California</state>
        <zip>94305</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4005</name>
      <address>
        <city>Atlanta</city>
        <state>Georgia</state>
        <zip>30322</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4019</name>
      <address>
        <city>Springfield</city>
        <state>Illinois</state>
        <zip>01107</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4004</name>
      <address>
        <city>Baltimore</city>
        <state>Maryland</state>
        <zip>21287</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4002</name>
      <address>
        <city>Bethesda</city>
        <state>Maryland</state>
        <zip>20892</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4022</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02115</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4012</name>
      <address>
        <city>Ann Arbor</city>
        <state>Michigan</state>
        <zip>48109</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4025</name>
      <address>
        <city>Detroit</city>
        <state>Michigan</state>
        <zip>48202</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4006</name>
      <address>
        <city>Rochester</city>
        <state>Minnesota</state>
        <zip>55905</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4024</name>
      <address>
        <city>St Louis</city>
        <state>Missouri</state>
        <zip>63110</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4016</name>
      <address>
        <city>New York</city>
        <state>New York</state>
        <zip>10029</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4003</name>
      <address>
        <city>New York</city>
        <state>New York</state>
        <zip>10032</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4014</name>
      <address>
        <city>Rosedale</city>
        <state>New York</state>
        <zip>11422</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4018</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <zip>19104</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4001</name>
      <address>
        <city>Chattanooga</city>
        <state>Tennessee</state>
        <zip>37404</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4020</name>
      <address>
        <city>Dallas</city>
        <state>Texas</state>
        <zip>75390</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4027</name>
      <address>
        <city>Houston</city>
        <state>Texas</state>
        <zip>77030</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4013</name>
      <address>
        <city>Seattle</city>
        <state>Washington</state>
        <zip>98195</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4104</name>
      <address>
        <city>Porto Alegre</city>
        <zip>90035 003</zip>
        <country>Brazil</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4302</name>
      <address>
        <city>Aachen</city>
        <zip>52074</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4301</name>
      <address>
        <city>Düsseldorf</city>
        <zip>40225</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4304</name>
      <address>
        <city>Hamburg</city>
        <zip>20246</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4401</name>
      <address>
        <city>Bari</city>
        <zip>70124</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4404</name>
      <address>
        <city>Bergamo</city>
        <zip>24127</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4403</name>
      <address>
        <city>Montichiari</city>
        <zip>25018</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4405</name>
      <address>
        <city>Reggio Calabria</city>
        <zip>89124</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4503</name>
      <address>
        <city>Barcelona</city>
        <zip>08025</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4504</name>
      <address>
        <city>Madrid</city>
        <zip>28040</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number 4502</name>
      <address>
        <city>Madrid</city>
        <zip>28041</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Brazil</country>
    <country>Germany</country>
    <country>Italy</country>
    <country>Spain</country>
    <country>United States</country>
  </location_countries>
  <verification_date>August 2015</verification_date>
  <study_first_submitted>August 13, 2012</study_first_submitted>
  <study_first_submitted_qc>August 13, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">August 15, 2012</study_first_posted>
  <disposition_first_submitted>August 12, 2015</disposition_first_submitted>
  <disposition_first_submitted_qc>August 12, 2015</disposition_first_submitted_qc>
  <disposition_first_posted type="Estimate">August 26, 2015</disposition_first_posted>
  <last_update_submitted>August 12, 2015</last_update_submitted>
  <last_update_submitted_qc>August 12, 2015</last_update_submitted_qc>
  <last_update_posted type="Estimate">August 26, 2015</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>Glomerulosclerosis, Focal Segmental</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Antibodies, Monoclonal</mesh_term>
  </intervention_browse>
  <!-- Results have not yet been posted, but a results disposition has been submitted           -->
</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/NCT01665391
org study id: GC1008FSGS03110
secondary id: 2010-019545-25
secondary id: U1111-1139-9082
secondary id: DRI12792
nct id: NCT01665391
lead sponsor: 
has dmc: Yes
The primary objectives of this trial are as follows:

        -  to compare the achievement of a partial remission (PR) or complete remission (CR) in
           urinary protein: creatinine ratio (Up/c ratio) in patients treated with fresolimumab
           versus placebo

        -  to compare the safety profile of patients treated with fresolimumab versus placebo

      The secondary objectives are as follows:

        -  To compare the reduction in proteinuria in patients treated with fresolimumab versus
           placebo

        -  To evaluate fresolimumab dose-dependent reduction in proteinuria

        -  To compare the change in renal function (estimated glomerular filtration rate [eGFR]) in
           patients treated with fresolimumab versus placebo

        -  To evaluate the multiple-dose pharmacokinetics of fresolimumab
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Double (Participant, Investigator)
measure: Percentage of patients achieving partial remission (PR) or complete remission (CR) in urinary protein: creatinine ratio (Up/c ratio)
time frame: Up to Day 112
measure: Number of patients reporting adverse events (AEs), serious adverse events (SAEs), and medical events of interest (MEOIs)
time frame: Up to Day 112
measure: Percentage of patients achieving CR in Up/c ratio
time frame: Up to Day 112
measure: Percentage of patients achieving PR in Up/c ratio
time frame: Up to Day 112
measure: Change from baseline in Up/c ratio and urinary protein excretion rate
time frame: Up to Day 112
measure: Time to first PR or CR
time frame: Up to Day 112
measure: Change from baseline in eGFR (estimated glomerular filtration rate)
time frame: Up to Day 112
measure: Percentage of patients achieving PR or CR with stable eGFR (estimated glomerular filtration rate)
time frame: Up to Day 112
measure: Mean Fresolimumab serum concentration at each sample collection time point
time frame: Up to Day 252
arm group label: fresolimumab 1 mg/kg total body weight
arm group type: Experimental
arm group label: fresolimumab 4 mg/kg total body weight
arm group type: Experimental
arm group label: Placebo
arm group type: Placebo Comparator
intervention type: Drug
intervention name: fresolimumab
description: 1 mg/kg total body weight administered intravenous (IV)
arm group label: fresolimumab 1 mg/kg total body weight
intervention type: Drug
intervention name: fresolimumab
description: 4 mg/kg total body weight administered intravenous (IV)
arm group label: fresolimumab 4 mg/kg total body weight
intervention type: Drug
intervention name: Placebo
description: Placebo administered to match active treatment group
arm group label: Placebo
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Medical Monitor
role: Study Director
affiliation: Genzyme, a Sanofi Company
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country: Brazil
country: Germany
country: Italy
country: Spain
country: United States
responsible party type: Sponsor
mesh term: Glomerulosclerosis, Focal Segmental
mesh term: Antibodies, Monoclonal