<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/NCT01727934</url>
  </required_header>
  <id_info>
    <org_study_id>SPC3649-207</org_study_id>
    <nct_id>NCT01727934</nct_id>
  </id_info>
  <brief_title>Miravirsen Study in Null Responder to Pegylated Interferon Alpha Plus Ribavirin Subjects With Chronic Hepatitis C</brief_title>
  <official_title>A Phase 2, Open-Label, Clinical Trial of Miravirsen Sodium in Null Responder to Pegylated-Interferon Alpha Plus Ribavirin Subjects With Chronic Hepatitis C (CHC) Virus Genotype 1 Infection</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Santaris Pharma A/S</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Santaris Pharma A/S</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this open-label study is to assess the safety, antiviral activity, and&#xD;
      pharmacokinetics of 9 subcutaneous injections of miravirsen monotherapy (5 weekly doses over&#xD;
      5 weeks, followed by a further 4 doses once every other week over 7 weeks) over a total of 12&#xD;
      weeks of treatment. The subjects enrolled in this study are chronically infected with HCV&#xD;
      genotype 1 and are null responders to treatment with peg IFNα/RBV therapy.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Unknown status</overall_status>
  <last_known_status>Active, not recruiting</last_known_status>
  <start_date>November 2012</start_date>
  <completion_date type="Anticipated">April 2014</completion_date>
  <primary_completion_date type="Anticipated">January 2014</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>The proportion of subjects with sustained virological response 24 weeks after the end of therapy.</measure>
    <time_frame>36 weeks</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>The proportion of subjects with a sustained virological response 12 and 48 weeks after the end of therapy.</measure>
    <time_frame>60 weeks</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The proportion of subjects with undetectable HCV RNA levels at the end of treatment.</measure>
    <time_frame>12 weeks</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change in HCV RNA levels from baseline throughout the study.</measure>
    <time_frame>60 weeks</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>The proportion of subjects who experience virological failure throughout the study.</measure>
    <time_frame>60 weeks</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Safety will be assessed by evaluation of adverse events, physical examinations, vital signs, 12-lead ECGs, and laboratory assessments (clinical chemistry, hematology, urinalysis).</measure>
    <time_frame>60 weeks</time_frame>
  </secondary_outcome>
  <other_outcome>
    <measure>Viral resistance analysis at baseline and throughout the study.</measure>
    <time_frame>60 weeks</time_frame>
    <description>The miR-122 seed sites in HCV RNA from subjects at baseline and following viral breakthrough or relapse will be subjected to genotypic sequence analysis.</description>
  </other_outcome>
  <other_outcome>
    <measure>Plasma pharmacokinetics</measure>
    <time_frame>28 weeks</time_frame>
    <description>Plasma PK for miravirsen levels will be determined for up to 2 hours post-dose on Day 1, up to 24 hours post-dose on Days 29 and 84, and pre-dose for all other treatment period visits. Additionally, plasma PK will be evaluated at all follow-up visits through Week 28.</description>
  </other_outcome>
  <other_outcome>
    <measure>Urine pharmacokinetics</measure>
    <time_frame>Up to 24 hours post-dose on Day 29 and Day 84</time_frame>
  </other_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Anticipated">10</enrollment>
  <condition>Hepatitis C</condition>
  <arm_group>
    <arm_group_label>Miravirsen sodium</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Miravirsen will be dosed as single subcutaneous injections. Subjects will receive 5 weekly doses at 7 mg/kg then 4 every other week doses at 5 mg/kg.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Miravirsen sodium</intervention_name>
    <description>Subcutaneous injection</description>
    <arm_group_label>Miravirsen sodium</arm_group_label>
    <other_name>SPC3649</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Diagnosis of chronic hepatitis C&#xD;
&#xD;
          -  HCV genotype 1&#xD;
&#xD;
          -  BMI 18-38 kg/m2&#xD;
&#xD;
          -  Null responder to pegylated interferon alpha and ribavirin&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Co-infection with hepatitis B virus (HBV) or human immunodeficiency virus (HIV)&#xD;
&#xD;
          -  Significant liver disease in addition to hepatitis C&#xD;
&#xD;
          -  Decompensated liver disease medical history or current clinical features&#xD;
&#xD;
          -  Histologic evidence of hepatic cirrhosis&#xD;
&#xD;
          -  Concurrent clinically significant medical diagnosis (other than CHC)&#xD;
&#xD;
          -  Concurrent social conditions (e.g. drugs of abuse, alcohol excess, poor living&#xD;
             accommodation)&#xD;
&#xD;
          -  Clinically significant illness within 30 days preceding entry into the study&#xD;
&#xD;
          -  Participated in an investigational drug study within 30 days or 5 half-lives,&#xD;
             whichever is longer, prior to the start of study medication&#xD;
&#xD;
          -  History of clinically significant allergic drug reactions&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>21 Years</minimum_age>
    <maximum_age>70 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Maribel Rodriguez-Torres, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Fundacion de Investgacion de Diego</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Fundacion de Investigation de Diego</name>
      <address>
        <city>San Juan</city>
        <zip>00927</zip>
        <country>Puerto Rico</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Puerto Rico</country>
  </location_countries>
  <verification_date>January 2014</verification_date>
  <study_first_submitted>November 12, 2012</study_first_submitted>
  <study_first_submitted_qc>November 12, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">November 16, 2012</study_first_posted>
  <last_update_submitted>January 3, 2014</last_update_submitted>
  <last_update_submitted_qc>January 3, 2014</last_update_submitted_qc>
  <last_update_posted type="Estimate">January 6, 2014</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Antisense</keyword>
  <keyword>miR-122 antagonist</keyword>
  <keyword>host factor</keyword>
  <keyword>Chronic hepatitis C</keyword>
  <keyword>Hepatitis C</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Hepatitis A</mesh_term>
    <mesh_term>Hepatitis C</mesh_term>
    <mesh_term>Hepatitis C, Chronic</mesh_term>
    <mesh_term>Hepatitis</mesh_term>
    <mesh_term>Hepatitis, Chronic</mesh_term>
  </condition_browse>
  <!-- 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/NCT01727934
org study id: SPC3649-207
nct id: NCT01727934
lead sponsor: 
has dmc: No
The purpose of this open-label study is to assess the safety, antiviral activity, and
      pharmacokinetics of 9 subcutaneous injections of miravirsen monotherapy (5 weekly doses over
      5 weeks, followed by a further 4 doses once every other week over 7 weeks) over a total of 12
      weeks of treatment. The subjects enrolled in this study are chronically infected with HCV
      genotype 1 and are null responders to treatment with peg IFNα/RBV therapy.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: The proportion of subjects with sustained virological response 24 weeks after the end of therapy.
time frame: 36 weeks
measure: The proportion of subjects with a sustained virological response 12 and 48 weeks after the end of therapy.
time frame: 60 weeks
measure: The proportion of subjects with undetectable HCV RNA levels at the end of treatment.
time frame: 12 weeks
measure: Change in HCV RNA levels from baseline throughout the study.
time frame: 60 weeks
measure: The proportion of subjects who experience virological failure throughout the study.
time frame: 60 weeks
measure: Safety will be assessed by evaluation of adverse events, physical examinations, vital signs, 12-lead ECGs, and laboratory assessments (clinical chemistry, hematology, urinalysis).
time frame: 60 weeks
measure: Viral resistance analysis at baseline and throughout the study.
time frame: 60 weeks
description: The miR-122 seed sites in HCV RNA from subjects at baseline and following viral breakthrough or relapse will be subjected to genotypic sequence analysis.
measure: Plasma pharmacokinetics
time frame: 28 weeks
description: Plasma PK for miravirsen levels will be determined for up to 2 hours post-dose on Day 1, up to 24 hours post-dose on Days 29 and 84, and pre-dose for all other treatment period visits. Additionally, plasma PK will be evaluated at all follow-up visits through Week 28.
measure: Urine pharmacokinetics
time frame: Up to 24 hours post-dose on Day 29 and Day 84
arm group label: Miravirsen sodium
arm group type: Experimental
description: Miravirsen will be dosed as single subcutaneous injections. Subjects will receive 5 weekly doses at 7 mg/kg then 4 every other week doses at 5 mg/kg.
intervention type: Drug
intervention name: Miravirsen sodium
description: Subcutaneous injection
arm group label: Miravirsen sodium
other name: SPC3649
criteria: 
gender: All
minimum age: 21 Years
maximum age: 70 Years
healthy volunteers: No
last name: Maribel Rodriguez-Torres, MD
role: Principal Investigator
affiliation: Fundacion de Investgacion de Diego
facility: 
country: Puerto Rico
responsible party type: Sponsor
mesh term: Hepatitis A
mesh term: Hepatitis C
mesh term: Hepatitis C, Chronic
mesh term: Hepatitis
mesh term: Hepatitis, Chronic