<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/NCT03038399</url>
  </required_header>
  <id_info>
    <org_study_id>VBP15-LTE</org_study_id>
    <nct_id>NCT03038399</nct_id>
  </id_info>
  <brief_title>Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)</brief_title>
  <official_title>A 24-month Phase II Open-label, Multicenter Long-term Extension Study to Assess the Long-Term Safety and Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>ReveraGen BioPharma, Inc.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>University of Pittsburgh</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Cooperative International Neuromuscular Research Group</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>ReveraGen BioPharma, Inc.</source>
  <oversight_info>
    <has_dmc>Yes</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>
      This long-term extension study is an open-label, multiple-dose study to evaluate the&#xD;
      long-term safety, tolerability, efficacy and PD of vamorolone administered once daily by&#xD;
      liquid oral suspension over a Treatment Period of 24 months to young boys with DMD who&#xD;
      participated in the VBP15-002 Phase IIa and VBP15-003 Phase IIa extension core studies.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This study will evaluate if it is safe to use a new medication called vamorolone for more&#xD;
      than two weeks in children with DMD, if boys with DMD who take the study medication have&#xD;
      improved muscle function compared to boys with DMD in other studies who did not take any type&#xD;
      of steroid, and to see if boys with DMD who take the study medication gain less weight&#xD;
      compared to boys with DMD in a prior study who took another type of steroid called&#xD;
      prednisone. Enrolled participants will take the study medication for 24 months.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">February 2, 2017</start_date>
  <completion_date type="Actual">April 30, 2020</completion_date>
  <primary_completion_date type="Actual">April 30, 2020</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>Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE Version 4.03</measure>
    <time_frame>24 months</time_frame>
    <description>To evaluate the long-term safety and tolerability of vamorolone, administered orally at daily doses up to 6.0 mg/kg/day over a 24- month Treatment Period, in boys ages 4-7 years with DMD; Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination);</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Total Number of Adverse Events as Assessed by CTCAE Version 4.03</measure>
    <time_frame>24 Months</time_frame>
    <description>To evaluate the long-term safety and tolerability of vamorolone, administered orally at daily doses up to 6.0 mg/kg/day over a 24-month Treatment Period, in boys ages 4-7 years with DMD. Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination).</description>
  </primary_outcome>
  <number_of_arms>4</number_of_arms>
  <enrollment type="Actual">46</enrollment>
  <condition>Duchenne Muscular Dystrophy</condition>
  <arm_group>
    <arm_group_label>Dose Level Group 1</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Dose Level Group 2</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Dose Level Group 3</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Dose Level Group 4</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Vamorolone 0.25 mg/day/day</intervention_name>
    <description>Oral administration of 0.25 mg/kg/day daily for 24 months.</description>
    <arm_group_label>Dose Level Group 1</arm_group_label>
    <other_name>VBP15</other_name>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Vamorolone 0.75 mg/day/day</intervention_name>
    <description>Oral administration of 0.75 mg/kg/day daily for 24 months.</description>
    <arm_group_label>Dose Level Group 2</arm_group_label>
    <other_name>VBP15</other_name>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Vamorolone 2.0 mg/day/day</intervention_name>
    <description>Oral administration of 2.0 mg/kg/day daily for 24 months.</description>
    <arm_group_label>Dose Level Group 3</arm_group_label>
    <other_name>VBP15</other_name>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Vamorolone 6.0 mg/day/day</intervention_name>
    <description>Oral administration of 6.0 mg/kg/day daily for 24 months.</description>
    <arm_group_label>Dose Level Group 4</arm_group_label>
    <other_name>VBP15</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Subject's parent or legal guardian has provided written informed consent and HIPAA&#xD;
             authorization (if applicable) prior to any VBP15-LTE long-term extension&#xD;
             study-specific procedures;&#xD;
&#xD;
          2. Subject has previously completed study VBP15-003 up to and including the Week 24 Final&#xD;
             assessments, prior to enrolling in the VBP15-LTE study at the conclusion of the&#xD;
             VBP15-003 Week 24 Visit [Note: if entering the dose-tapering period, subject is&#xD;
             enrolling within 8 weeks after the VBP15-003 final visit following dose-tapering]; and&#xD;
&#xD;
          3. Subject and parent/guardian are willing and able to comply with scheduled visits,&#xD;
             study drug administration plan, and study procedures.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Subject had a serious or severe adverse event in study VBP15-003 that, in the opinion&#xD;
             of the Investigator, was probably or definitely related to vamorolone use and&#xD;
             precludes safe use of vamorolone for the subject in this long-term extension study;&#xD;
&#xD;
          2. Subject has current or history of major renal or hepatic impairment, diabetes mellitus&#xD;
             or immunosuppression;&#xD;
&#xD;
          3. Subject has current or history of chronic systemic fungal or viral infections;&#xD;
&#xD;
          4. Subject has used mineralocorticoid receptor agents, such as spironolactone,&#xD;
             eplerenone, canrenone (canrenoate potassium), prorenone (prorenoate potassium),&#xD;
             mexrenone (mexrenoate potassium) within 4 weeks prior to the first dose of study&#xD;
             medication;&#xD;
&#xD;
          5. Subject has evidence of symptomatic cardiomyopathy. [Note: Asymptomatic cardiac&#xD;
             abnormality on investigation would not be exclusionary];&#xD;
&#xD;
          6. Subject is currently being treated or has received previous treatment with oral&#xD;
             glucocorticoids or other immunosuppressive agents [Notes: Past transient use of oral&#xD;
             glucocorticoids or other oral immunosuppressive agents for no longer than 3 months&#xD;
             cumulative, with last use at least 3 months prior to first dose of study medication,&#xD;
             will be considered for eligibility on a case-by-case basis. Inhaled and/or topical&#xD;
             glucocorticoids prescribed for an indication other than DMD are permitted but must be&#xD;
             administered at stable dose for at least 3 months prior to study drug administration];&#xD;
&#xD;
          7. Subject has used idebenone within 4 weeks prior to the first dose of study medication;&#xD;
&#xD;
          8. Subject has an allergy or hypersensitivity to the study medication or to any of its&#xD;
             constituents;&#xD;
&#xD;
          9. Subject has severe behavioral or cognitive problems that preclude participation in the&#xD;
             study, in the opinion of the Investigator;&#xD;
&#xD;
         10. Subject has previous or ongoing medical condition, medical history, physical findings&#xD;
             or laboratory abnormalities that could affect safety, make it unlikely that treatment&#xD;
             and follow-up will be correctly completed or impair the assessment of study results,&#xD;
             in the opinion of the Investigator&#xD;
&#xD;
         11. Subject is currently taking any investigational drug, or has taken any investigational&#xD;
             drug other than vamorolone within 3 months prior to the start of study treatment.&#xD;
&#xD;
        Note: Subjects may be re-evaluated if ineligible due to a transient condition which would&#xD;
        prevent the subject from participating.&#xD;
      </textblock>
    </criteria>
    <gender>Male</gender>
    <minimum_age>4 Years</minimum_age>
    <maximum_age>7 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Paula R Clemens, MD</last_name>
    <role>Study Chair</role>
    <affiliation>University of Pittsburgh</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>University of California Davis</name>
      <address>
        <city>Davis</city>
        <state>California</state>
        <zip>95616</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Florida</name>
      <address>
        <city>Gainesville</city>
        <state>Florida</state>
        <zip>32611</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Nemours Children's Hospital</name>
      <address>
        <city>Orlando</city>
        <state>Florida</state>
        <zip>32827</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Ann &amp; Robert H. Lurie Children's Hospital</name>
      <address>
        <city>Chicago</city>
        <state>Illinois</state>
        <zip>60611</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Duke University</name>
      <address>
        <city>Durham</city>
        <state>North Carolina</state>
        <zip>27710</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Texas Southwestern Medical Center</name>
      <address>
        <city>Dallas</city>
        <state>Texas</state>
        <zip>75207</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Royal Children's Hospital</name>
      <address>
        <city>Melbourne</city>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Sydney Children's Hospital</name>
      <address>
        <city>Westmead</city>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Alberta Children's Hospital</name>
      <address>
        <city>Calgary</city>
        <state>Alberta</state>
        <zip>T3B 6A8</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Schneider Children's Medical Center</name>
      <address>
        <city>Petah Tikwah</city>
        <zip>49202</zip>
        <country>Israel</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Queen Silvia Children's Hospital</name>
      <address>
        <city>Gothenburg</city>
        <zip>41685</zip>
        <country>Sweden</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Newcastle upon Tyne Hospitals NHS Foundation Trust</name>
      <address>
        <city>Newcastle upon Tyne</city>
        <zip>NE7 7DN</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Australia</country>
    <country>Canada</country>
    <country>Israel</country>
    <country>Sweden</country>
    <country>United Kingdom</country>
    <country>United States</country>
  </location_countries>
  <link>
    <url>https://pubmed.ncbi.nlm.nih.gov/32956407/</url>
    <description>Link to publication</description>
  </link>
  <results_reference>
    <citation>Smith EC, Conklin LS, Hoffman EP, Clemens PR, Mah JK, Finkel RS, Guglieri M, Tulinius M, Nevo Y, Ryan MM, Webster R, Castro D, Kuntz NL, Kerchner L, Morgenroth LP, Arrieta A, Shimony M, Jaros M, Shale P, Gordish-Dressman H, Hagerty L, Dang UJ, Damsker JM, Schwartz BD, Mengle-Gaw LJ, McDonald CM; CINRG VBP15 and DNHS Investigators. Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study. PLoS Med. 2020 Sep 21;17(9):e1003222. doi: 10.1371/journal.pmed.1003222. eCollection 2020 Sep.</citation>
    <PMID>32956407</PMID>
  </results_reference>
  <verification_date>April 2021</verification_date>
  <study_first_submitted>January 30, 2017</study_first_submitted>
  <study_first_submitted_qc>January 30, 2017</study_first_submitted_qc>
  <study_first_posted type="Estimate">January 31, 2017</study_first_posted>
  <results_first_submitted>April 28, 2021</results_first_submitted>
  <results_first_submitted_qc>April 28, 2021</results_first_submitted_qc>
  <results_first_posted type="Actual">May 20, 2021</results_first_posted>
  <last_update_submitted>April 28, 2021</last_update_submitted>
  <last_update_submitted_qc>April 28, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">May 20, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Duchenne muscular dystrophy</keyword>
  <keyword>vamorolone</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>
  <provided_document_section>
    <provided_document>
      <document_type>Study Protocol</document_type>
      <document_has_protocol>Yes</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>No</document_has_sap>
      <document_date>October 3, 2018</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/99/NCT03038399/Prot_000.pdf</document_url>
    </provided_document>
    <provided_document>
      <document_type>Statistical Analysis Plan</document_type>
      <document_has_protocol>No</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>Yes</document_has_sap>
      <document_date>June 21, 2019</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/99/NCT03038399/SAP_002.pdf</document_url>
    </provided_document>
    <provided_document>
      <document_type>Informed Consent Form</document_type>
      <document_has_protocol>No</document_has_protocol>
      <document_has_icf>Yes</document_has_icf>
      <document_has_sap>No</document_has_sap>
      <document_date>October 17, 2018</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/99/NCT03038399/ICF_001.pdf</document_url>
    </provided_document>
  </provided_document_section>

  <clinical_results>

    <participant_flow>
      <group_list>
        <group group_id="P1">
          <title>Dose Level Group 1</title>
          <description>Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.&#xD;
Vamorolone 0.25 mg/day/day: Oral administration of 0.25 mg/kg/day daily</description>
        </group>
        <group group_id="P2">
          <title>Dose Level Group 2</title>
          <description>Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.&#xD;
Vamorolone 0.75 mg/day/day: Oral administration of 0.75 mg/kg/day daily</description>
        </group>
        <group group_id="P3">
          <title>Dose Level Group 3</title>
          <description>Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.&#xD;
Vamorolone 2.0 mg/day/day: Oral administration of 2.0 mg/kg/day daily</description>
        </group>
        <group group_id="P4">
          <title>Dose Level Group 4</title>
          <description>Participants enrolled in Dose Level Group 5 will receive vamorolone 6.0 mg/kg/day.&#xD;
Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="11"/>
                <participants group_id="P2" count="12"/>
                <participants group_id="P3" count="12"/>
                <participants group_id="P4" count="11"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="10"/>
                <participants group_id="P2" count="11"/>
                <participants group_id="P3" count="11"/>
                <participants group_id="P4" count="9">Subjects escalated from lower dose groups</participants>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
                <participants group_id="P2" count="1"/>
                <participants group_id="P3" count="1"/>
                <participants group_id="P4" count="2"/>
              </participants_list>
            </milestone>
          </milestone_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <population>Subjects entered this study in one of 4 dose level groups, but were allowed to escalate/de-escalate during the course of the study.</population>
      <group_list>
        <group group_id="B1">
          <title>Dose Level Group 1</title>
          <description>Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.&#xD;
Vamorolone 0.25 mg/day/day: Oral administration of 0.25 mg/kg/day daily</description>
        </group>
        <group group_id="B2">
          <title>Dose Level Group 2</title>
          <description>Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.&#xD;
Vamorolone 0.75 mg/day/day: Oral administration of 0.75 mg/kg/day daily</description>
        </group>
        <group group_id="B3">
          <title>Dose Level Group 3</title>
          <description>Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.&#xD;
Vamorolone 2.0 mg/day/day: Oral administration of 2.0 mg/kg/day daily</description>
        </group>
        <group group_id="B4">
          <title>Dose Level Group 4</title>
          <description>Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.&#xD;
Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily</description>
        </group>
        <group group_id="B5">
          <title>Total</title>
          <description>Total of all reporting groups</description>
        </group>
      </group_list>
      <analyzed_list>
        <analyzed>
          <units>Participants</units>
          <scope>Overall</scope>
          <count_list>
            <count group_id="B1" value="11"/>
            <count group_id="B2" value="12"/>
            <count group_id="B3" value="12"/>
            <count group_id="B4" value="11"/>
            <count group_id="B5" value="46"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>years</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="5.87" spread="1.135"/>
                    <measurement group_id="B2" value="5.11" spread="0.805"/>
                    <measurement group_id="B3" value="5.13" spread=".964"/>
                    <measurement group_id="B4" value="5.27" spread="0.856"/>
                    <measurement group_id="B5" value="5.33" spread="0.965"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Sex: Female, Male</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Female</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                    <measurement group_id="B5" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="11"/>
                    <measurement group_id="B2" value="12"/>
                    <measurement group_id="B3" value="12"/>
                    <measurement group_id="B4" value="11"/>
                    <measurement group_id="B5" value="46"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Ethnicity (NIH/OMB)</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="3"/>
                    <measurement group_id="B5" value="4"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Not Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="11"/>
                    <measurement group_id="B2" value="11"/>
                    <measurement group_id="B3" value="12"/>
                    <measurement group_id="B4" value="8"/>
                    <measurement group_id="B5" value="42"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                    <measurement group_id="B5" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Race (NIH/OMB)</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>American Indian or Alaska Native</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                    <measurement group_id="B5" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Asian</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                    <measurement group_id="B5" value="1"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Native Hawaiian or Other Pacific Islander</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                    <measurement group_id="B5" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Black or African American</title>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                    <measurement group_id="B5" value="1"/>
                  </measurement_list>
                </category>
                <category>
                  <title>White</title>
                  <measurement_list>
                    <measurement group_id="B1" value="10"/>
                    <measurement group_id="B2" value="10"/>
                    <measurement group_id="B3" value="12"/>
                    <measurement group_id="B4" value="11"/>
                    <measurement group_id="B5" value="43"/>
                  </measurement_list>
                </category>
                <category>
                  <title>More than one race</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                    <measurement group_id="B5" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                    <measurement group_id="B5" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Region of Enrollment</title>
          <units>participants</units>
          <param>Number</param>
          <class_list>
            <class>
              <title>Canada</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="3"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="2"/>
                    <measurement group_id="B4" value="2"/>
                    <measurement group_id="B5" value="7"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Sweden</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="4"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                    <measurement group_id="B5" value="4"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>United States</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="8"/>
                    <measurement group_id="B2" value="3"/>
                    <measurement group_id="B3" value="2"/>
                    <measurement group_id="B4" value="5"/>
                    <measurement group_id="B5" value="18"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>United Kingdom</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="4"/>
                    <measurement group_id="B4" value="2"/>
                    <measurement group_id="B5" value="6"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Israel</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="3"/>
                    <measurement group_id="B3" value="2"/>
                    <measurement group_id="B4" value="0"/>
                    <measurement group_id="B5" value="5"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Australia</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="2"/>
                    <measurement group_id="B3" value="2"/>
                    <measurement group_id="B4" value="2"/>
                    <measurement group_id="B5" value="6"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE Version 4.03</title>
        <description>To evaluate the long-term safety and tolerability of vamorolone, administered orally at daily doses up to 6.0 mg/kg/day over a 24- month Treatment Period, in boys ages 4-7 years with DMD; Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination);</description>
        <time_frame>24 months</time_frame>
        <population>All subjects who receive at least one dose of vamorolone study medication in the study will be included in the Safety Population. The Safety Population is the primary analysis population for safety assessments.</population>
        <group_list>
          <group group_id="O1">
            <title>Dose Level Group 1</title>
            <description>Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.&#xD;
Vamorolone 0.25 mg/day/day: Oral administration of 0.25 mg/kg/day daily</description>
          </group>
          <group group_id="O2">
            <title>Dose Level Group 2</title>
            <description>Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.&#xD;
Vamorolone 0.75 mg/day/day: Oral administration of 0.75 mg/kg/day daily</description>
          </group>
          <group group_id="O3">
            <title>Dose Level Group 3</title>
            <description>Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.&#xD;
Vamorolone 2.0 mg/day/day: Oral administration of 2.0 mg/kg/day daily</description>
          </group>
          <group group_id="O4">
            <title>Dose Level Group 4</title>
            <description>Participants enrolled in Dose Level Group 4 will receive vamorolone 4.0 mg/kg/day.&#xD;
Vamorolone 4.0 mg/day/day: Oral administration of 4.0 mg/kg/day daily</description>
          </group>
          <group group_id="O5">
            <title>Dose Level Group 5</title>
            <description>Participants enrolled in Dose Level Group 5 will receive vamorolone 6.0 mg/kg/day.&#xD;
Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE Version 4.03</title>
          <description>To evaluate the long-term safety and tolerability of vamorolone, administered orally at daily doses up to 6.0 mg/kg/day over a 24- month Treatment Period, in boys ages 4-7 years with DMD; Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination);</description>
          <population>All subjects who receive at least one dose of vamorolone study medication in the study will be included in the Safety Population. The Safety Population is the primary analysis population for safety assessments.</population>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="11"/>
                <count group_id="O2" value="23"/>
                <count group_id="O3" value="38"/>
                <count group_id="O4" value="3"/>
                <count group_id="O5" value="41"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="4"/>
                    <measurement group_id="O2" value="14"/>
                    <measurement group_id="O3" value="29"/>
                    <measurement group_id="O4" value="1"/>
                    <measurement group_id="O5" value="39"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Total Number of Adverse Events as Assessed by CTCAE Version 4.03</title>
        <description>To evaluate the long-term safety and tolerability of vamorolone, administered orally at daily doses up to 6.0 mg/kg/day over a 24-month Treatment Period, in boys ages 4-7 years with DMD. Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination).</description>
        <time_frame>24 Months</time_frame>
        <population>All subjects who receive at least one dose of vamorolone study medication in the study will be included in the Safety Population. The Safety Population is the primary analysis population for safety assessments.</population>
        <group_list>
          <group group_id="O1">
            <title>Dose Level Group 1</title>
            <description>Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.&#xD;
Vamorolone 0.25 mg/day/day: Oral administration of 0.25 mg/kg/day daily</description>
          </group>
          <group group_id="O2">
            <title>Dose Level Group 2</title>
            <description>Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.&#xD;
Vamorolone 0.75 mg/day/day: Oral administration of 0.75 mg/kg/day daily</description>
          </group>
          <group group_id="O3">
            <title>Dose Level Group 3</title>
            <description>Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.&#xD;
Vamorolone 2.0 mg/day/day: Oral administration of 2.0 mg/kg/day daily</description>
          </group>
          <group group_id="O4">
            <title>Dose Level Group 4</title>
            <description>Participants enrolled in Dose Level Group 4 will receive vamorolone 4.0 mg/kg/day.&#xD;
Vamorolone 4.0 mg/day/day: Oral administration of 4.0 mg/kg/day daily</description>
          </group>
          <group group_id="O5">
            <title>Dose Level Group 5</title>
            <description>Participants enrolled in Dose Level Group 5 will receive vamorolone 6.0 mg/kg/day.&#xD;
Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily</description>
          </group>
        </group_list>
        <measure>
          <title>Total Number of Adverse Events as Assessed by CTCAE Version 4.03</title>
          <description>To evaluate the long-term safety and tolerability of vamorolone, administered orally at daily doses up to 6.0 mg/kg/day over a 24-month Treatment Period, in boys ages 4-7 years with DMD. Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination).</description>
          <population>All subjects who receive at least one dose of vamorolone study medication in the study will be included in the Safety Population. The Safety Population is the primary analysis population for safety assessments.</population>
          <units>Events</units>
          <param>Number</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="11"/>
                <count group_id="O2" value="23"/>
                <count group_id="O3" value="38"/>
                <count group_id="O4" value="3"/>
                <count group_id="O5" value="41"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Total Number of AEs</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="15"/>
                    <measurement group_id="O2" value="34"/>
                    <measurement group_id="O3" value="203"/>
                    <measurement group_id="O4" value="5"/>
                    <measurement group_id="O5" value="302"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Total Number of TEAEs</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="14"/>
                    <measurement group_id="O2" value="34"/>
                    <measurement group_id="O3" value="202"/>
                    <measurement group_id="O4" value="5"/>
                    <measurement group_id="O5" value="300"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>Adverse events, including Serious Adverse Events (SAEs), and concomitant medications will be assessed at each study visit and recorded throughout the 24 months treatment period.</time_frame>
      <group_list>
        <group group_id="E1">
          <title>Dose Level Group 1</title>
          <description>Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.&#xD;
Vamorolone 0.25 mg/day/day: Oral administration of 0.25 mg/kg/day daily</description>
        </group>
        <group group_id="E2">
          <title>Dose Level Group 2</title>
          <description>Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.&#xD;
Vamorolone 0.75 mg/day/day: Oral administration of 0.75 mg/kg/day daily</description>
        </group>
        <group group_id="E3">
          <title>Dose Level Group 3</title>
          <description>Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.&#xD;
Vamorolone 2.0 mg/day/day: Oral administration of 2.0 mg/kg/day daily</description>
        </group>
        <group group_id="E4">
          <title>Dose Level Group 4</title>
          <description>Participants enrolled in Dose Level Group 4 will receive vamorolone 4.0 mg/kg/day.&#xD;
Vamorolone 4.0 mg/day/day: Oral administration of 4.0 mg/kg/day daily</description>
        </group>
        <group group_id="E5">
          <title>Dose Level Group 5</title>
          <description>Participants enrolled in Dose Level Group 5 will receive vamorolone 6.0 mg/kg/day.&#xD;
Vamorolone 6.0 mg/day/day: Oral administration of 6.0 mg/kg/day daily</description>
        </group>
      </group_list>
      <serious_events>
        <default_vocab>MedDRA</default_vocab>
        <default_assessment>Systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, all-cause mortality</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="1" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Pneumonia</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Renal and urinary disorders</title>
            <event_list>
              <event>
                <sub_title>Myoglobinuria</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" events="1" subjects_affected="1" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>5</frequency_threshold>
        <default_vocab>MedDRA</default_vocab>
        <default_assessment>Systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, other adverse events</sub_title>
                <counts group_id="E1" subjects_affected="4" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="14" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="29" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="39" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Ear and labyrinth disorders</title>
            <event_list>
              <event>
                <sub_title>Ear Pain</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="5" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="4" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Abdominal Pain</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="6" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Abdominal Pain Upper</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="5" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Constipation</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="3" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="8" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Diarrhoea</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="3" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Vomiting</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="5" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="7" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Chest Pain</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="3" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Influenza like illness</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="3" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Pyrexia</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="9" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="8" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Upper respiratory tract infection</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="2" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="5" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="6" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Medical device site pain</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Medical device site rash</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Immune system disorders</title>
            <event_list>
              <event>
                <sub_title>Hypersensitivity</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="2" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Ear Infection</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="2" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="4" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Gastroenteritis</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="3" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="3" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Influenza</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="3" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="6" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Nasopharyngitis</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="7" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="12" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Otitis media</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="3" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="2" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Pharyngitis streptococcal</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="2" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="2" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="3" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Sinusitis</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Body Tinea</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Gastroenteritis viral</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="2" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Hand-foot-and-mouth disease</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Injury, poisoning and procedural complications</title>
            <event_list>
              <event>
                <sub_title>Fall</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="4" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="6" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Joint Injury</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="2" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Limb Injury</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="3" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Arthropod bite</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="1" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Investigations</title>
            <event_list>
              <event>
                <sub_title>Weight increased</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="2" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="10" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Blood triglycerides increased</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="3" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Lipids Abnormal</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Musculoskeletal and connective tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Muscle spasms</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="3" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Osteopenia</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="4" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Pain in extremity</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="3" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="5" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="6" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Back Pain</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="2" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="1" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Nervous system disorders</title>
            <event_list>
              <event>
                <sub_title>Headache</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="4" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="7" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Psychiatric disorders</title>
            <event_list>
              <event>
                <sub_title>Aggression</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Anxiety</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Respiratory, thoracic and mediastinal disorders</title>
            <event_list>
              <event>
                <sub_title>Cough</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="7" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="8" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Oropharyngeal pain</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="3" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Skin and subcutaneous tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Rash</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="2" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="1" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Urticaria</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="23"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="38"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="3"/>
                <counts group_id="E5" subjects_affected="1" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </other_events>
    </reported_events>

    <certain_agreements>
      <pi_employee>Principal Investigators are NOT employed by the organization sponsoring the study.</pi_employee>
      <restrictive_agreement>There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed. </restrictive_agreement>
    </certain_agreements>
    <point_of_contact>
      <name_or_title>Dr. Eric Hoffman</name_or_title>
      <organization>ReveraGen BioPharma Inc.</organization>
      <phone>301-762-7980</phone>
      <email>ericphoffman@gmail.com</email>
    </point_of_contact>
  </clinical_results>
</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/NCT03038399
org study id: VBP15-LTE
nct id: NCT03038399
lead sponsor: 
collaborator: 
collaborator: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
This long-term extension study is an open-label, multiple-dose study to evaluate the
      long-term safety, tolerability, efficacy and PD of vamorolone administered once daily by
      liquid oral suspension over a Treatment Period of 24 months to young boys with DMD who
      participated in the VBP15-002 Phase IIa and VBP15-003 Phase IIa extension core studies.
This study will evaluate if it is safe to use a new medication called vamorolone for more
      than two weeks in children with DMD, if boys with DMD who take the study medication have
      improved muscle function compared to boys with DMD in other studies who did not take any type
      of steroid, and to see if boys with DMD who take the study medication gain less weight
      compared to boys with DMD in a prior study who took another type of steroid called
      prednisone. Enrolled participants will take the study medication for 24 months.
allocation: Non-Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE Version 4.03
time frame: 24 months
description: To evaluate the long-term safety and tolerability of vamorolone, administered orally at daily doses up to 6.0 mg/kg/day over a 24- month Treatment Period, in boys ages 4-7 years with DMD; Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination);
measure: Total Number of Adverse Events as Assessed by CTCAE Version 4.03
time frame: 24 Months
description: To evaluate the long-term safety and tolerability of vamorolone, administered orally at daily doses up to 6.0 mg/kg/day over a 24-month Treatment Period, in boys ages 4-7 years with DMD. Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination).
arm group label: Dose Level Group 1
arm group type: Experimental
description: Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.
arm group label: Dose Level Group 2
arm group type: Experimental
description: Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.
arm group label: Dose Level Group 3
arm group type: Experimental
description: Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.
arm group label: Dose Level Group 4
arm group type: Experimental
description: Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.
intervention type: Drug
intervention name: Vamorolone 0.25 mg/day/day
description: Oral administration of 0.25 mg/kg/day daily for 24 months.
arm group label: Dose Level Group 1
other name: VBP15
intervention type: Drug
intervention name: Vamorolone 0.75 mg/day/day
description: Oral administration of 0.75 mg/kg/day daily for 24 months.
arm group label: Dose Level Group 2
other name: VBP15
intervention type: Drug
intervention name: Vamorolone 2.0 mg/day/day
description: Oral administration of 2.0 mg/kg/day daily for 24 months.
arm group label: Dose Level Group 3
other name: VBP15
intervention type: Drug
intervention name: Vamorolone 6.0 mg/day/day
description: Oral administration of 6.0 mg/kg/day daily for 24 months.
arm group label: Dose Level Group 4
other name: VBP15
criteria: 
gender: Male
minimum age: 4 Years
maximum age: 7 Years
healthy volunteers: No
last name: Paula R Clemens, MD
role: Study Chair
affiliation: University of Pittsburgh
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: Australia
country: Canada
country: Israel
country: Sweden
country: United Kingdom
country: United States
url: https://pubmed.ncbi.nlm.nih.gov/32956407/
description: Link to publication
citation: Smith EC, Conklin LS, Hoffman EP, Clemens PR, Mah JK, Finkel RS, Guglieri M, Tulinius M, Nevo Y, Ryan MM, Webster R, Castro D, Kuntz NL, Kerchner L, Morgenroth LP, Arrieta A, Shimony M, Jaros M, Shale P, Gordish-Dressman H, Hagerty L, Dang UJ, Damsker JM, Schwartz BD, Mengle-Gaw LJ, McDonald CM; CINRG VBP15 and DNHS Investigators. Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study. PLoS Med. 2020 Sep 21;17(9):e1003222. doi: 10.1371/journal.pmed.1003222. eCollection 2020 Sep.
PMID: 32956407
responsible party type: Sponsor
mesh term: Muscular Dystrophies
mesh term: Muscular Dystrophy, Duchenne
provided document: 
provided document: 
provided document: 
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
point of contact: 