<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/NCT03815825</url>
  </required_header>
  <id_info>
    <org_study_id>ISIS 696844-CS5</org_study_id>
    <secondary_id>2020-005174-94</secondary_id>
    <nct_id>NCT03815825</nct_id>
  </id_info>
  <brief_title>GOLDEN STUDY: A Study to Assess Safety and Efficacy of Multiple Doses of IONIS-FB-LRx in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration (AMD)</brief_title>
  <official_title>A Phase 2, Randomized Placebo-Controlled, Double-Masked Study to Assess Safety and Efficacy of Multiple Doses of IONIS-FB-LRx, an Antisense Inhibitor of Complement Factor B, in Patients With Geographic Atrophy Secondary to Age-Related Macular Degeneration (AMD)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Ionis Pharmaceuticals, Inc.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Ionis Pharmaceuticals, Inc.</source>
  <oversight_info>
    <has_dmc>No</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>
      Evaluate the effect of IONIS-FB-LRx on the rate of change of the area of geographic atrophy&#xD;
      (GA) secondary to age-related macular degeneration (AMD) measured by fundus autofluorescence&#xD;
      (FAF).&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      The study will assess the rate of change of the area of GA secondary to AMD by measuring FAF&#xD;
      in up to 330 participants with GA due to AMD being treated with IONIS-FB-LRx. The study is a&#xD;
      Phase 2, double-masked, randomized, stratified, placebo-controlled study conducted at&#xD;
      multiple centers. It is an adaptive design in which three dose levels will be evaluated in a&#xD;
      subset of participants (Stage 1) and, following an interim analysis, the number of&#xD;
      participants in two of the dose cohorts will be expanded (Stage 2).&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Active, not recruiting</overall_status>
  <start_date type="Actual">March 4, 2019</start_date>
  <completion_date type="Anticipated">July 2024</completion_date>
  <primary_completion_date type="Anticipated">April 2024</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>Absolute Change from Baseline in the Geographic Atrophy (GA) Area at Week 49, as Assessed by Retinal Imaging</measure>
    <time_frame>Week 49</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>Percentage Change from Baseline in Levels of Factor B (FB) in Plasma</measure>
    <time_frame>Baseline and up to Week 49</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Percentage Change from Baseline in Levels of Serum AH50 Activity</measure>
    <time_frame>Baseline and up to Week 49</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Absolute Change from Baseline in Low Luminance Visual Acuity (LLVA)</measure>
    <time_frame>Baseline and up to Week 49</time_frame>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">332</enrollment>
  <condition>Macular Degeneration</condition>
  <condition>Geographic Atrophy</condition>
  <arm_group>
    <arm_group_label>IONIS-FB-LRx</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Stage 1 participants will receive IONIS-FB-LRx randomized to 1 of 3 dose levels, administered subcutaneously every 4 weeks, completing the treatment period at Week 45. Stage 2 will expand 2 of the dosing cohorts in a new randomized group of participants based on the Stage 1 interim analysis. Participants will be randomized to 1 of 2 of the expanded cohorts, administered subcutaneously every 4 weeks, completing the treatment period at Week 45.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Placebo</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Stage 1 participants will receive a placebo matching solution, administered subcutaneously every 4 weeks, completing the treatment period at Week 45. Stage 2 participants will receive a placebo matching solution, administered subcutaneously every 4 weeks, completing the treatment period at Week 45.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>IONIS-FB-LRx</intervention_name>
    <description>IONIS-FB-LRx at multiple ascending doses, administered subcutaneously every 4 weeks</description>
    <arm_group_label>IONIS-FB-LRx</arm_group_label>
    <other_name>ISIS 696844</other_name>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebo</intervention_name>
    <description>Placebo matching solution, administered subcutaneously every 4 weeks</description>
    <arm_group_label>Placebo</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Females must be non-pregnant and non-lactating, and either surgically sterile or&#xD;
             post-menopausal.&#xD;
&#xD;
          -  Vaccination against Neisseria meningitidis and Streptococcus pneumoniae received at&#xD;
             least 2 weeks prior to first dose of investigational product&#xD;
&#xD;
          -  Well-demarcated geographic atrophy (GA) due to AMD&#xD;
&#xD;
          -  Best-corrected visual acuity (BCVA) letter score of 35 letters (approx. 20/200 Snellen&#xD;
             equivalent) or better on the ETDRS chart&#xD;
&#xD;
          -  Must have clear ocular media and adequate pupillary dilation in the study eye to&#xD;
             permit high-quality fundus imaging&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Clinically-significant abnormalities in medical history&#xD;
&#xD;
          -  A lack of full recovery from any infection for at least 14 days prior to the Study&#xD;
             Drug administration&#xD;
&#xD;
          -  Chronic treatment with steroids, including topically or intravitreally administered&#xD;
&#xD;
          -  History or presence of diabetic retinopathy or diabetic macular edema (DME)&#xD;
&#xD;
          -  History or presence of a disease other than AMD that could affect vision or safety&#xD;
             assessments&#xD;
&#xD;
          -  Prior treatment with another investigational drug, biological agent, or device&#xD;
&#xD;
          -  Other protocol-specified inclusion/exclusion criteria may apply&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>50 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Phoenix</city>
        <state>Arizona</state>
        <zip>85020</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Phoenix</city>
        <state>Arizona</state>
        <zip>85021</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Phoenix</city>
        <state>Arizona</state>
        <zip>85053</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Sun City</city>
        <state>Arizona</state>
        <zip>85351</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Beverly Hills</city>
        <state>California</state>
        <zip>90211</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Huntington Beach</city>
        <state>California</state>
        <zip>92697</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>La Jolla</city>
        <state>California</state>
        <zip>92093</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Long Beach</city>
        <state>California</state>
        <zip>90807</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Poway</city>
        <state>California</state>
        <zip>92064</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>San Francisco</city>
        <state>California</state>
        <zip>94109</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Colorado Springs</city>
        <state>Colorado</state>
        <zip>80909</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Clearwater</city>
        <state>Florida</state>
        <zip>33761</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Fort Myers</city>
        <state>Florida</state>
        <zip>33907</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Lake Worth</city>
        <state>Florida</state>
        <zip>33461</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Lakeland</city>
        <state>Florida</state>
        <zip>33801</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Melbourne</city>
        <state>Florida</state>
        <zip>32901</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Stuart</city>
        <state>Florida</state>
        <zip>34994</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Tampa</city>
        <state>Florida</state>
        <zip>33612</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Winter Haven</city>
        <state>Florida</state>
        <zip>33880</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Augusta</city>
        <state>Georgia</state>
        <zip>30909-6440</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Lemont</city>
        <state>Illinois</state>
        <zip>60439</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Springfield</city>
        <state>Illinois</state>
        <zip>62702</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Leawood</city>
        <state>Kansas</state>
        <zip>66211-1669</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Baltimore</city>
        <state>Maryland</state>
        <zip>21204</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Baltimore</city>
        <state>Maryland</state>
        <zip>21209</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Hagerstown</city>
        <state>Maryland</state>
        <zip>21740</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Southaven</city>
        <state>Mississippi</state>
        <zip>38671</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Albuquerque</city>
        <state>New Mexico</state>
        <zip>87109</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Asheville</city>
        <state>North Carolina</state>
        <zip>28803</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Hickory</city>
        <state>North Carolina</state>
        <zip>28602</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Winston-Salem</city>
        <state>North Carolina</state>
        <zip>27157</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Cleveland</city>
        <state>Ohio</state>
        <zip>44106</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Cleveland</city>
        <state>Ohio</state>
        <zip>44130</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Cleveland</city>
        <state>Ohio</state>
        <zip>44195</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Tulsa</city>
        <state>Oklahoma</state>
        <zip>74114</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <zip>19104</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Pittsburgh</city>
        <state>Pennsylvania</state>
        <zip>15213</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>West Mifflin</city>
        <state>Pennsylvania</state>
        <zip>15122</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Greenville</city>
        <state>South Carolina</state>
        <zip>29605</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Ladson</city>
        <state>South Carolina</state>
        <zip>29456</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Nashville</city>
        <state>Tennessee</state>
        <zip>37203</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Abilene</city>
        <state>Texas</state>
        <zip>79606</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Arlington</city>
        <state>Texas</state>
        <zip>76012</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Austin</city>
        <state>Texas</state>
        <zip>78705</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Bellaire</city>
        <state>Texas</state>
        <zip>77401</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Dallas</city>
        <state>Texas</state>
        <zip>75231</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>McAllen</city>
        <state>Texas</state>
        <zip>78503</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>San Antonio</city>
        <state>Texas</state>
        <zip>78240-1502</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>San Antonio</city>
        <state>Texas</state>
        <zip>78240</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Southlake</city>
        <state>Texas</state>
        <zip>76051</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>The Woodlands</city>
        <state>Texas</state>
        <zip>77384</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Willow Park</city>
        <state>Texas</state>
        <zip>76087</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Salt Lake City</city>
        <state>Utah</state>
        <zip>84107</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Lynchburg</city>
        <state>Virginia</state>
        <zip>24502</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Richmond</city>
        <state>Virginia</state>
        <zip>23235</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Silverdale</city>
        <state>Washington</state>
        <zip>98383</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Albury</city>
        <state>New South Wales</state>
        <zip>2640</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Hurstville</city>
        <state>New South Wales</state>
        <zip>2228</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Parramatta</city>
        <state>New South Wales</state>
        <zip>2150</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Sydney</city>
        <state>New South Wales</state>
        <zip>2000</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Adelaide</city>
        <state>South Australia</state>
        <zip>5000</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>East Melbourne</city>
        <state>Victoria</state>
        <zip>3002</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Essendon</city>
        <state>Victoria</state>
        <zip>3040</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Glen Iris</city>
        <state>Victoria</state>
        <zip>3146</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Malvern</city>
        <state>Victoria</state>
        <zip>3144</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Parkville</city>
        <state>Victoria</state>
        <zip>3050</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Perth</city>
        <state>Western Australia</state>
        <zip>6009</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Linz</city>
        <state>Oberösterreich</state>
        <zip>4020</zip>
        <country>Austria</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Klagenfurt</city>
        <zip>9020</zip>
        <country>Austria</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Vienna</city>
        <zip>1090</zip>
        <country>Austria</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Vienna</city>
        <zip>1140</zip>
        <country>Austria</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>London</city>
        <state>Ontario</state>
        <zip>N6A 4V2</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Ottawa</city>
        <state>Ontario</state>
        <zip>K1H 8L6</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Ottawa</city>
        <state>Ontario</state>
        <zip>K2B 7E9</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Québec</city>
        <state>Quebec</state>
        <zip>G1S 4L8</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Prague</city>
        <zip>15000</zip>
        <country>Czechia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Nijmegen</city>
        <state>Gelderland</state>
        <zip>6525EX</zip>
        <country>Netherlands</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Rotterdam</city>
        <zip>3011 BH</zip>
        <country>Netherlands</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Christchurch</city>
        <zip>8013</zip>
        <country>New Zealand</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Katowice</city>
        <state>Silesian</state>
        <zip>40-514</zip>
        <country>Poland</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Bydgoszcz</city>
        <zip>85-631</zip>
        <country>Poland</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Terrassa</city>
        <state>Barcelona</state>
        <zip>8227</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Majadahonda</city>
        <state>Madrid</state>
        <zip>28222</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Pamplona</city>
        <state>Navarra</state>
        <zip>31008</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Burjassot</city>
        <state>Valencia</state>
        <zip>46100</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Barcelona</city>
        <zip>8022</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Barcelona</city>
        <zip>8034</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Valladolid</city>
        <zip>47012</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>IONIS Investigative Site</name>
      <address>
        <city>Zaragoza</city>
        <zip>50009</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Australia</country>
    <country>Austria</country>
    <country>Canada</country>
    <country>Czechia</country>
    <country>Netherlands</country>
    <country>New Zealand</country>
    <country>Poland</country>
    <country>Spain</country>
    <country>United States</country>
  </location_countries>
  <verification_date>August 2023</verification_date>
  <study_first_submitted>January 22, 2019</study_first_submitted>
  <study_first_submitted_qc>January 22, 2019</study_first_submitted_qc>
  <study_first_posted type="Actual">January 24, 2019</study_first_posted>
  <last_update_submitted>August 3, 2023</last_update_submitted>
  <last_update_submitted_qc>August 3, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">August 7, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Macular Degeneration</mesh_term>
    <mesh_term>Geographic Atrophy</mesh_term>
    <mesh_term>Atrophy</mesh_term>
  </condition_browse>
  <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/NCT03815825
org study id: ISIS 696844-CS5
secondary id: 2020-005174-94
nct id: NCT03815825
lead sponsor: 
has dmc: No
is fda regulated drug: Yes
is fda regulated device: No
Evaluate the effect of IONIS-FB-LRx on the rate of change of the area of geographic atrophy
      (GA) secondary to age-related macular degeneration (AMD) measured by fundus autofluorescence
      (FAF).
The study will assess the rate of change of the area of GA secondary to AMD by measuring FAF
      in up to 330 participants with GA due to AMD being treated with IONIS-FB-LRx. The study is a
      Phase 2, double-masked, randomized, stratified, placebo-controlled study conducted at
      multiple centers. It is an adaptive design in which three dose levels will be evaluated in a
      subset of participants (Stage 1) and, following an interim analysis, the number of
      participants in two of the dose cohorts will be expanded (Stage 2).
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Double (Participant, Investigator)
measure: Absolute Change from Baseline in the Geographic Atrophy (GA) Area at Week 49, as Assessed by Retinal Imaging
time frame: Week 49
measure: Percentage Change from Baseline in Levels of Factor B (FB) in Plasma
time frame: Baseline and up to Week 49
measure: Percentage Change from Baseline in Levels of Serum AH50 Activity
time frame: Baseline and up to Week 49
measure: Absolute Change from Baseline in Low Luminance Visual Acuity (LLVA)
time frame: Baseline and up to Week 49
arm group label: IONIS-FB-LRx
arm group type: Experimental
description: Stage 1 participants will receive IONIS-FB-LRx randomized to 1 of 3 dose levels, administered subcutaneously every 4 weeks, completing the treatment period at Week 45. Stage 2 will expand 2 of the dosing cohorts in a new randomized group of participants based on the Stage 1 interim analysis. Participants will be randomized to 1 of 2 of the expanded cohorts, administered subcutaneously every 4 weeks, completing the treatment period at Week 45.
arm group label: Placebo
arm group type: Experimental
description: Stage 1 participants will receive a placebo matching solution, administered subcutaneously every 4 weeks, completing the treatment period at Week 45. Stage 2 participants will receive a placebo matching solution, administered subcutaneously every 4 weeks, completing the treatment period at Week 45.
intervention type: Drug
intervention name: IONIS-FB-LRx
description: IONIS-FB-LRx at multiple ascending doses, administered subcutaneously every 4 weeks
arm group label: IONIS-FB-LRx
other name: ISIS 696844
intervention type: Drug
intervention name: Placebo
description: Placebo matching solution, administered subcutaneously every 4 weeks
arm group label: Placebo
criteria: 
gender: All
minimum age: 50 Years
maximum age: N/A
healthy volunteers: No
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country: Australia
country: Austria
country: Canada
country: Czechia
country: Netherlands
country: New Zealand
country: Poland
country: Spain
country: United States
responsible party type: Sponsor
mesh term: Macular Degeneration
mesh term: Geographic Atrophy
mesh term: Atrophy
sharing ipd: No