<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/NCT04433234</url>
  </required_header>
  <id_info>
    <org_study_id>DS5141-A-J201</org_study_id>
    <secondary_id>205321</secondary_id>
    <nct_id>NCT04433234</nct_id>
  </id_info>
  <brief_title>Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy</brief_title>
  <official_title>A Phase II, Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Daiichi Sankyo Co., Ltd.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Daiichi Sankyo, Inc.</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      This is a multicenter, open-label, long-term, extension, phase 2 study to evaluate the safety&#xD;
      and efficacy of long-term treatment with DS-5141b in patients with DMD who have completed&#xD;
      DS5141-A-J101.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Active, not recruiting</overall_status>
  <start_date type="Actual">June 30, 2020</start_date>
  <completion_date type="Anticipated">January 31, 2027</completion_date>
  <primary_completion_date type="Anticipated">January 31, 2027</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Non-Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Incidence of Adverse Events (AEs)</measure>
    <time_frame>From first injection to after the last injection of DS-5141b in this study (within approximately 2 years)</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Change in distance walked during 6-minute walk test (6MWT)</measure>
    <time_frame>Every 3 months (within approximately 2 years)</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Change in time to stand (TTSTAND)</measure>
    <time_frame>Every 3 months (within approximately 2 years)</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Change in time in Timed up and go test</measure>
    <time_frame>Every 3 months (within approximately 2 years)</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Change in time in 10-meter Run/Walk test</measure>
    <time_frame>Every 3 months (within approximately 2 years)</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Change in score in the North Star Ambulatory Assessment (NSAA)</measure>
    <time_frame>Every 3 months (within approximately 2 years)</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Change in score in the Performance of Upper Limb (PUL)</measure>
    <time_frame>Every 3 months (within approximately 2 years)</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Change in Left Ventricular Ejection Fraction percentage (LVEF %)</measure>
    <time_frame>Every 6 months (within approximately 2 years)</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Change in in Forced Vital Capacity (FVC) (percent predicted)</measure>
    <time_frame>Every 6 months (within approximately 2 years)</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Change in Muscle Strength Measured by Quantitative Muscle Strength Assessment</measure>
    <time_frame>Every 3 months (within approximately 2 years)</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>Concentration of DS-5141a in plasma</measure>
    <time_frame>Every 3 months (within approximately 2 years)</time_frame>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Anticipated">8</enrollment>
  <condition>Duchenne Muscular Dystrophy</condition>
  <arm_group>
    <arm_group_label>DS-5141b 2.0 mg/kg</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Participants who will receive DS-5141b 2.0 mg/kg once weekly.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>DS-5141b 6.0 mg/kg</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Participants who will receive DS-5141b 6.0 mg/kg once weekly.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>DS-5141b</intervention_name>
    <description>Administered via subcutaneous injection once weekly</description>
    <arm_group_label>DS-5141b 2.0 mg/kg</arm_group_label>
    <arm_group_label>DS-5141b 6.0 mg/kg</arm_group_label>
    <other_name>Renadirsen</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Has competed a study of DS5141-A-J101&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Significant safety issues in a study of DS5141-A-J101&#xD;
&#xD;
          -  Patient who does not consent to use appropriate contraception&#xD;
&#xD;
          -  Patient not appropriate to participant in the study as determined by the Investigator&#xD;
      </textblock>
    </criteria>
    <gender>Male</gender>
    <gender_based>Yes</gender_based>
    <minimum_age>5 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Clinical Study Leader</last_name>
    <role>Study Director</role>
    <affiliation>Daiichi Sankyo Co., Ltd.</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>National Center of Neurology and Psychiatry</name>
      <address>
        <city>Tokyo</city>
        <state>Kodaira-Shi</state>
        <zip>187-8551</zip>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Kobe University Hospital</name>
      <address>
        <city>Hyōgo, Kobe-shi</city>
        <zip>650-0017</zip>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Japan</country>
  </location_countries>
  <verification_date>July 2023</verification_date>
  <study_first_submitted>June 12, 2020</study_first_submitted>
  <study_first_submitted_qc>June 12, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 16, 2020</study_first_posted>
  <last_update_submitted>July 26, 2023</last_update_submitted>
  <last_update_submitted_qc>July 26, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">July 28, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Duchenne muscular dystrophy</keyword>
  <keyword>DMD</keyword>
  <keyword>Oligonucleotides, antisense</keyword>
  <keyword>Exon skipping</keyword>
  <keyword>DS-5141b</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Muscular Dystrophies</mesh_term>
    <mesh_term>Muscular Dystrophy, Duchenne</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/NCT04433234
org study id: DS5141-A-J201
secondary id: 205321
nct id: NCT04433234
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
This is a multicenter, open-label, long-term, extension, phase 2 study to evaluate the safety
      and efficacy of long-term treatment with DS-5141b in patients with DMD who have completed
      DS5141-A-J101.
allocation: Non-Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Incidence of Adverse Events (AEs)
time frame: From first injection to after the last injection of DS-5141b in this study (within approximately 2 years)
measure: Change in distance walked during 6-minute walk test (6MWT)
time frame: Every 3 months (within approximately 2 years)
measure: Change in time to stand (TTSTAND)
time frame: Every 3 months (within approximately 2 years)
measure: Change in time in Timed up and go test
time frame: Every 3 months (within approximately 2 years)
measure: Change in time in 10-meter Run/Walk test
time frame: Every 3 months (within approximately 2 years)
measure: Change in score in the North Star Ambulatory Assessment (NSAA)
time frame: Every 3 months (within approximately 2 years)
measure: Change in score in the Performance of Upper Limb (PUL)
time frame: Every 3 months (within approximately 2 years)
measure: Change in Left Ventricular Ejection Fraction percentage (LVEF %)
time frame: Every 6 months (within approximately 2 years)
measure: Change in in Forced Vital Capacity (FVC) (percent predicted)
time frame: Every 6 months (within approximately 2 years)
measure: Change in Muscle Strength Measured by Quantitative Muscle Strength Assessment
time frame: Every 3 months (within approximately 2 years)
measure: Concentration of DS-5141a in plasma
time frame: Every 3 months (within approximately 2 years)
arm group label: DS-5141b 2.0 mg/kg
arm group type: Experimental
description: Participants who will receive DS-5141b 2.0 mg/kg once weekly.
arm group label: DS-5141b 6.0 mg/kg
arm group type: Experimental
description: Participants who will receive DS-5141b 6.0 mg/kg once weekly.
intervention type: Drug
intervention name: DS-5141b
description: Administered via subcutaneous injection once weekly
arm group label: DS-5141b 2.0 mg/kg
arm group label: DS-5141b 6.0 mg/kg
other name: Renadirsen
criteria: 
gender: Male
gender based: Yes
minimum age: 5 Years
maximum age: N/A
healthy volunteers: No
last name: Clinical Study Leader
role: Study Director
affiliation: Daiichi Sankyo Co., Ltd.
facility: 
facility: 
country: Japan
responsible party type: Sponsor
mesh term: Muscular Dystrophies
mesh term: Muscular Dystrophy, Duchenne
sharing ipd: No