<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/NCT03010046</url>
  </required_header>
  <id_info>
    <org_study_id>ANX005-CP01</org_study_id>
    <nct_id>NCT03010046</nct_id>
  </id_info>
  <brief_title>Single Dose Study of ANX005 in Healthy Volunteers</brief_title>
  <official_title>A Phase 1, Randomized, Placebo-controlled, Double Blind, Single, Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ANX005 Monotherapy and ANX005 in Combination With IVIg in Healthy Volunteers</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Annexon, Inc.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Annexon, Inc.</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      This is a single-center, randomized, double-blind, placebo-controlled, ascending,&#xD;
      single-infusion, sequential group study. Single, ascending doses will be administered to&#xD;
      approximately 64 subjects, with an option for 1 additional multi-dose cohort in approximately&#xD;
      8 subjects. The primary objective is to evaluate the safety of ANX005 administered as an&#xD;
      intravenous infusion as a single agent and in combination with intravenous immunoglobulin&#xD;
      (IVIg). The optional multi-dose cohort will evaluate either additional subjects at the&#xD;
      maximum tolerated dose or ANX005 administered as 2 infusions.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Terminated</overall_status>
  <why_stopped>
    ANX005 well tolerated at doses studied; study discontinued and initiated in patient population&#xD;
  </why_stopped>
  <start_date>December 2016</start_date>
  <completion_date type="Actual">June 2018</completion_date>
  <primary_completion_date type="Actual">June 2018</primary_completion_date>
  <phase>Phase 1</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>Triple (Participant, Care Provider, Investigator)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Number of subjects with treatment-related adverse events as assessed by CTCAE v4.03</measure>
    <time_frame>Day 43</time_frame>
    <description>Safety is assessed throughout the study. Day 43 is the last visit.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Peak plasma concentration</measure>
    <time_frame>Day 43</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Determine effective dose of ANX005</measure>
    <time_frame>Day 43</time_frame>
    <description>Percent of subjects with a biologic response, defined as a reduction of serum CH50 percent change from baseline at 21 and 28 days after the first ANX005 infusion</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Area under the plasma concentration versus time curve (AUC)</measure>
    <time_frame>Day 43</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Terminal half-life</measure>
    <time_frame>Day 43</time_frame>
  </secondary_outcome>
  <other_outcome>
    <measure>Explore relationship of AUC with PD responses in serum</measure>
    <time_frame>Day 43</time_frame>
  </other_outcome>
  <other_outcome>
    <measure>Explore relationship of AUC with PD responses in CSF</measure>
    <time_frame>Day 43</time_frame>
  </other_outcome>
  <other_outcome>
    <measure>Explore relationship of half-life with PD responses in serum</measure>
    <time_frame>Day 43</time_frame>
  </other_outcome>
  <other_outcome>
    <measure>Explore relationship of half-life with PD responses in CSF</measure>
    <time_frame>Day 43</time_frame>
  </other_outcome>
  <number_of_arms>3</number_of_arms>
  <enrollment type="Actual">27</enrollment>
  <condition>Safety and Tolerability in Healthy Volunteers</condition>
  <arm_group>
    <arm_group_label>ANX005 Monotherapy</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>ANX005 intravenous infusion</description>
  </arm_group>
  <arm_group>
    <arm_group_label>ANX005 and IVIg Combination Therapy</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>ANX005 intravenous infusion in combination with intravenous immunoglobulin (IVIg)</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Placebo</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Placebo intravenous infusion</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>ANX005</intervention_name>
    <description>Single ascending dose intravenous infusion</description>
    <arm_group_label>ANX005 Monotherapy</arm_group_label>
    <arm_group_label>ANX005 and IVIg Combination Therapy</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>IVIg</intervention_name>
    <description>IVIg infusion in Cohorts 4b and 5b only. Randomized to ANX005 followed by IVIg or placebo followed by IVIg.</description>
    <arm_group_label>ANX005 and IVIg Combination Therapy</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebos</intervention_name>
    <description>0.9% saline intravenous infusion</description>
    <arm_group_label>Placebo</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Male and females 18 years and older&#xD;
&#xD;
          -  Females must be postmenopausal, surgically sterilized, or willing and able to use 2&#xD;
             methods of contraception throughout the study and for 1 month after the final study&#xD;
             visit&#xD;
&#xD;
          -  Willing and able to undergo vaccination if not vaccinated recently&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  History of any autoimmune disease, meningitis, septicemia or pneumonia&#xD;
&#xD;
          -  History of hypercoagulable diseases, hyperviscosity, thrombosis, renal dysfunction or&#xD;
             acute renal failure&#xD;
&#xD;
          -  Known genetic deficiencies of the complement cascade system&#xD;
&#xD;
          -  History of conditions whose symptoms and effects could alter protein catabolism or IgG&#xD;
             utilization, e.g. protein-losing enteropathies or nephrotic syndrome&#xD;
&#xD;
          -  Body weight less than 50 kg or greater than 100 kg&#xD;
&#xD;
          -  Hypersensitivity or allergic reactions to any excipients in the ANX005 drug product&#xD;
&#xD;
          -  (Cohorts 4b and 5b) Known selective IgA deficiency or presence of antibodies to IgA at&#xD;
             screening&#xD;
&#xD;
          -  (Cohorts 4b and 5b) Prior reaction or hypersensitivity to blood products, including&#xD;
             IVIg or any of the excipients in IVIg.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>Accepts Healthy Volunteers</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Sandy Calman, MD</last_name>
    <role>Study Director</role>
    <affiliation>Annexon Medical Monitor</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Jason Lickliter, MBBS PhD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Nucleus Network Ltd</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Nucleus Network</name>
      <address>
        <city>Melbourne</city>
        <state>Victoria</state>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Australia</country>
  </location_countries>
  <verification_date>August 2020</verification_date>
  <study_first_submitted>December 21, 2016</study_first_submitted>
  <study_first_submitted_qc>January 3, 2017</study_first_submitted_qc>
  <study_first_posted type="Estimate">January 4, 2017</study_first_posted>
  <last_update_submitted>August 19, 2020</last_update_submitted>
  <last_update_submitted_qc>August 19, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">August 20, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</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/NCT03010046
org study id: ANX005-CP01
nct id: NCT03010046
lead sponsor: 
has dmc: No
This is a single-center, randomized, double-blind, placebo-controlled, ascending,
      single-infusion, sequential group study. Single, ascending doses will be administered to
      approximately 64 subjects, with an option for 1 additional multi-dose cohort in approximately
      8 subjects. The primary objective is to evaluate the safety of ANX005 administered as an
      intravenous infusion as a single agent and in combination with intravenous immunoglobulin
      (IVIg). The optional multi-dose cohort will evaluate either additional subjects at the
      maximum tolerated dose or ANX005 administered as 2 infusions.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Triple (Participant, Care Provider, Investigator)
measure: Number of subjects with treatment-related adverse events as assessed by CTCAE v4.03
time frame: Day 43
description: Safety is assessed throughout the study. Day 43 is the last visit.
measure: Peak plasma concentration
time frame: Day 43
measure: Determine effective dose of ANX005
time frame: Day 43
description: Percent of subjects with a biologic response, defined as a reduction of serum CH50 percent change from baseline at 21 and 28 days after the first ANX005 infusion
measure: Area under the plasma concentration versus time curve (AUC)
time frame: Day 43
measure: Terminal half-life
time frame: Day 43
measure: Explore relationship of AUC with PD responses in serum
time frame: Day 43
measure: Explore relationship of AUC with PD responses in CSF
time frame: Day 43
measure: Explore relationship of half-life with PD responses in serum
time frame: Day 43
measure: Explore relationship of half-life with PD responses in CSF
time frame: Day 43
arm group label: ANX005 Monotherapy
arm group type: Experimental
description: ANX005 intravenous infusion
arm group label: ANX005 and IVIg Combination Therapy
arm group type: Experimental
description: ANX005 intravenous infusion in combination with intravenous immunoglobulin (IVIg)
arm group label: Placebo
arm group type: Placebo Comparator
description: Placebo intravenous infusion
intervention type: Drug
intervention name: ANX005
description: Single ascending dose intravenous infusion
arm group label: ANX005 Monotherapy
arm group label: ANX005 and IVIg Combination Therapy
intervention type: Drug
intervention name: IVIg
description: IVIg infusion in Cohorts 4b and 5b only. Randomized to ANX005 followed by IVIg or placebo followed by IVIg.
arm group label: ANX005 and IVIg Combination Therapy
intervention type: Drug
intervention name: Placebos
description: 0.9% saline intravenous infusion
arm group label: Placebo
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: Accepts Healthy Volunteers
last name: Sandy Calman, MD
role: Study Director
affiliation: Annexon Medical Monitor
last name: Jason Lickliter, MBBS PhD
role: Principal Investigator
affiliation: Nucleus Network Ltd
facility: 
country: Australia
responsible party type: Sponsor
sharing ipd: No