<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/NCT03265119</url>
  </required_header>
  <id_info>
    <org_study_id>AEVI-001-ADHD-202</org_study_id>
    <nct_id>NCT03265119</nct_id>
  </id_info>
  <brief_title>PART A: Efficacy and Safety of AEVI-001 in Children and Adolescents With ADHD and With mGluR Mutations</brief_title>
  <official_title>A Multicenter, 2-Part, 6-Week, Double-blind, Randomized, Placebo-controlled, Parallel-design Study to Assess the Efficacy and Safety of AEVI-001 in Children and Adolescents (Ages 6-17 Years) With Attention Deficit Hyperactivity Disorder and With or Without Copy Number Variants in Specific Genes Implicated in Glutamatergic Signaling and Neuronal Connectivity</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Aevi Genomic Medicine, LLC, a Cerecor company</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Avalo Therapeutics, Inc.</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      This is PART A of a 2-part, 6-week, double-blind, dose-optimization, parallel-group study in&#xD;
      children and adolescents (ages 6-17 years) with ADHD with and without CNVs in specific genes&#xD;
      implicated in glutamatergic signaling and neuronal activity. Parts A will include subjects&#xD;
      determined to have a specific gene mutations implicated in glutamatergic signaling and&#xD;
      neuronal connectivity.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">August 28, 2017</start_date>
  <completion_date type="Actual">October 1, 2018</completion_date>
  <primary_completion_date type="Actual">September 24, 2018</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>Double (Participant, Investigator)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Change From Baseline in Attention Deficit Hyperactivity Disorder Rating Scale, Version 5 (ADHD-RS-5) Total Score</measure>
    <time_frame>Baseline to Visit 8 (Week 6)</time_frame>
    <description>The ADHD-RS-5 is comprised of 18 frequency items and 12 impairment items. Each frequency item was scored on a scale from 0 = &quot;Never or rarely&quot; to 3 = &quot;Very often&quot;.&#xD;
The ADHD-RS-5 total score was calculated as the sum of the 18 frequency item scores. The total score ranges from 0 to 54. Higher scores indicate greater symptom severity. Change from baseline value were calculated as the assessment value minus the baseline value.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Clinical Global Impression - Global Improvement (CGI -I) Response</measure>
    <time_frame>Visit 8 (Week 6)</time_frame>
    <description>The CGI-I item is rated on a 7-point scale from 1 = &quot;Very much improved&quot;, 2 = &quot;Much improved&quot;, 3 = &quot;Minimally improved&quot;, 4 = &quot;No change&quot;, 5 = &quot;Minimally worse&quot;, 6 = &quot;Much worse&quot;, 7 = &quot;Very much worse&quot;.&#xD;
Response is defined as achieving a CGI-I score of 1 or 2, scores of 3 to 7 or missing are defined as Non Response</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">69</enrollment>
  <condition>Attention Deficit Hyperactivity Disorder</condition>
  <arm_group>
    <arm_group_label>AEVI-001</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <arm_group>
    <arm_group_label>Placebo</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>AEVI-001</intervention_name>
    <description>Oral doses of 100 mg, 200 mg or 400 mg of AEVI-001 will be administered twice daily during the treatment period.</description>
    <arm_group_label>AEVI-001</arm_group_label>
    <other_name>MDGN-001</other_name>
    <other_name>NFC-1</other_name>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebo</intervention_name>
    <description>Oral doses of 100 mg, 200 mg or 400 mg of Placebo will be administered twice daily during the treatment period.</description>
    <arm_group_label>Placebo</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Subject and parent/legally authorized representative (LAR) can speak English fluently&#xD;
             and have provided written informed consent, and assent (as applicable) for this study.&#xD;
&#xD;
          2. Subject is 6 to 17 years of age (inclusive) at the time of consent/assent. The date of&#xD;
             signature of the informed consent/assent is defined as the beginning of the Screening&#xD;
             Period. This inclusion criterion will only be assessed at the Screening Visit (Visit&#xD;
             1).&#xD;
&#xD;
          3. Subject is male or non-pregnant, non-lactating female, who if of childbearing&#xD;
             potential agrees to comply with any applicable contraceptive requirements prior to&#xD;
             administration of investigational product (IP).&#xD;
&#xD;
          4. Subject meets Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition&#xD;
             (DSM-5) criteria for a primary diagnosis of ADHD based upon DSM 5 criteria.&#xD;
&#xD;
          5. Subject has a minimum score of ≥28 on the ADHD-RS-5 at the Baseline Visit (Visit 2).&#xD;
&#xD;
          6. Subject has been genotyped previously and has their identity confirmed (if required).&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Subject or parent/LAR is, in the opinion of the investigator, mentally or legally&#xD;
             incapacitated, has significant emotional problems at the time of the Screening Visit&#xD;
             (Visit 1) which could interfere with the conduct of study evaluations.&#xD;
&#xD;
          2. Subject has a current, controlled or uncontrolled, co-morbid major psychiatric&#xD;
             diagnosis (aside from ADHD), including an anxiety disorder, major depression, bipolar&#xD;
             disease, schizophrenia (or any psychotic disorder), and moderate or severe&#xD;
             intellectual disability. Mild anxiety and/or depressive symptoms that do not meet&#xD;
             diagnostic criteria for an anxiety disorder or major depression and/or do not require&#xD;
             treatment are not exclusionary.&#xD;
&#xD;
          3. Subject has autism spectrum disorder to include a DSM-IV diagnosis of autistic&#xD;
             disorder, Asperger's disorder, or pervasive developmental disorder.&#xD;
&#xD;
          4. Subject is currently taking any medication that might confound the results of safety&#xD;
             assessments conducted in the study.&#xD;
&#xD;
          5. Subject has a known history of cardiovascular disease, advanced arteriosclerosis,&#xD;
             structural cardiac abnormality, cardiomyopathy, serious heart rhythm abnormalities,&#xD;
             coronary artery disease, cardiac conduction problems, exercise-related cardiac events&#xD;
             including syncope and pre-syncope, or other serious cardiac problems.&#xD;
&#xD;
          6. Subject has any clinically significant abnormality on 12-lead ECG performed at the&#xD;
             Screening Visit (Visit 1) and/or the Baseline Visit (Visit 2) such as serious&#xD;
             arrhythmia, cardiac conduction problems, or other abnormalities deemed to be a&#xD;
             potential safety issue.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>6 Years</minimum_age>
    <maximum_age>17 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>Aevi Genomic Medicine</name>
      <address>
        <city>Wayne</city>
        <state>Pennsylvania</state>
        <zip>19087</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>July 2021</verification_date>
  <study_first_submitted>August 24, 2017</study_first_submitted>
  <study_first_submitted_qc>August 25, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">August 29, 2017</study_first_posted>
  <results_first_submitted>June 25, 2019</results_first_submitted>
  <results_first_submitted_qc>June 25, 2019</results_first_submitted_qc>
  <results_first_posted type="Actual">July 16, 2019</results_first_posted>
  <last_update_submitted>July 2, 2021</last_update_submitted>
  <last_update_submitted_qc>July 2, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">July 7, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Hyperkinesis</mesh_term>
    <mesh_term>Attention Deficit Disorder with Hyperactivity</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <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>July 25, 2018</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/19/NCT03265119/Prot_001.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>September 24, 2018</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/19/NCT03265119/SAP_000.pdf</document_url>
    </provided_document>
  </provided_document_section>

  <clinical_results>

    <participant_flow>
      <group_list>
        <group group_id="P1">
          <title>AEVI-001</title>
          <description>100 mg, 200 mg or 400 mg of AEVI-001 b.i.d.</description>
        </group>
        <group group_id="P2">
          <title>Placebo</title>
          <description>Placebo b.i.d.</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="34"/>
                <participants group_id="P2" count="35"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="24"/>
                <participants group_id="P2" count="28"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="10"/>
                <participants group_id="P2" count="7"/>
              </participants_list>
            </milestone>
          </milestone_list>
          <drop_withdraw_reason_list>
            <drop_withdraw_reason>
              <title>Adverse Event</title>
              <participants_list>
                <participants group_id="P1" count="2"/>
                <participants group_id="P2" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Lack of Efficacy</title>
              <participants_list>
                <participants group_id="P1" count="3"/>
                <participants group_id="P2" count="0"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Protocol Violation</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
                <participants group_id="P2" count="2"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Withdrawal by Subject</title>
              <participants_list>
                <participants group_id="P1" count="2"/>
                <participants group_id="P2" count="4"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Lost to Follow-up</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
                <participants group_id="P2" count="0"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Lack of Compliance</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
                <participants group_id="P2" count="0"/>
              </participants_list>
            </drop_withdraw_reason>
          </drop_withdraw_reason_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <group_list>
        <group group_id="B1">
          <title>AEVI-001</title>
          <description>100 mg, 200 mg or 400 mg of AEVI-001 b.i.d.</description>
        </group>
        <group group_id="B2">
          <title>Placebo</title>
          <description>Placebo b.i.d.</description>
        </group>
        <group group_id="B3">
          <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="34"/>
            <count group_id="B2" value="35"/>
            <count group_id="B3" value="69"/>
          </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="9.9" spread="2.95"/>
                    <measurement group_id="B2" value="10.8" spread="2.86"/>
                    <measurement group_id="B3" value="10.4" spread="2.92"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Age, Customized</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <title>6 to 12 years</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="26"/>
                    <measurement group_id="B2" value="27"/>
                    <measurement group_id="B3" value="53"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>13 to 17 years</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="8"/>
                    <measurement group_id="B2" value="8"/>
                    <measurement group_id="B3" value="16"/>
                  </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="7"/>
                    <measurement group_id="B2" value="14"/>
                    <measurement group_id="B3" value="21"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="27"/>
                    <measurement group_id="B2" value="21"/>
                    <measurement group_id="B3" value="48"/>
                  </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="6"/>
                    <measurement group_id="B2" value="4"/>
                    <measurement group_id="B3" value="10"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Not Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="28"/>
                    <measurement group_id="B2" value="31"/>
                    <measurement group_id="B3" value="59"/>
                  </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_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_list>
                </category>
                <category>
                  <title>Asian</title>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" 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_list>
                </category>
                <category>
                  <title>Black or African American</title>
                  <measurement_list>
                    <measurement group_id="B1" value="24"/>
                    <measurement group_id="B2" value="20"/>
                    <measurement group_id="B3" value="44"/>
                  </measurement_list>
                </category>
                <category>
                  <title>White</title>
                  <measurement_list>
                    <measurement group_id="B1" value="7"/>
                    <measurement group_id="B2" value="12"/>
                    <measurement group_id="B3" value="19"/>
                  </measurement_list>
                </category>
                <category>
                  <title>More than one race</title>
                  <measurement_list>
                    <measurement group_id="B1" value="2"/>
                    <measurement group_id="B2" value="3"/>
                    <measurement group_id="B3" value="5"/>
                  </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_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Region of Enrollment</title>
          <units>participants</units>
          <param>Number</param>
          <class_list>
            <class>
              <title>United States</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="34"/>
                    <measurement group_id="B2" value="35"/>
                    <measurement group_id="B3" value="69"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Attention Deficit Hyperactivity Disorder Rating Scale, Version 5 (ADHD-RS-5) Total Score</title>
          <description>The ADHD-RS-5 is comprised of 18 frequency items and 12 impairment items. Each frequency item was scored on a scale from 0 = &quot;Never or rarely&quot; to 3 = &quot;Very often&quot;.&#xD;
The ADHD-RS-5 total score was calculated as the sum of the 18 frequency item scores. The total score ranges from 0 to 54. Higher scores indicate greater symptom severity.</description>
          <units>units on a scale</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="39.0" spread="6.76"/>
                    <measurement group_id="B2" value="38.1" spread="8.29"/>
                    <measurement group_id="B3" value="38.6" spread="7.53"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Change From Baseline in Attention Deficit Hyperactivity Disorder Rating Scale, Version 5 (ADHD-RS-5) Total Score</title>
        <description>The ADHD-RS-5 is comprised of 18 frequency items and 12 impairment items. Each frequency item was scored on a scale from 0 = &quot;Never or rarely&quot; to 3 = &quot;Very often&quot;.&#xD;
The ADHD-RS-5 total score was calculated as the sum of the 18 frequency item scores. The total score ranges from 0 to 54. Higher scores indicate greater symptom severity. Change from baseline value were calculated as the assessment value minus the baseline value.</description>
        <time_frame>Baseline to Visit 8 (Week 6)</time_frame>
        <population>Full Analysis Set</population>
        <group_list>
          <group group_id="O1">
            <title>AEVI-001</title>
            <description>100 mg, 200 mg or 400 mg of AEVI-001 b.i.d.</description>
          </group>
          <group group_id="O2">
            <title>Placebo</title>
            <description>Placebo b.i.d.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Attention Deficit Hyperactivity Disorder Rating Scale, Version 5 (ADHD-RS-5) Total Score</title>
          <description>The ADHD-RS-5 is comprised of 18 frequency items and 12 impairment items. Each frequency item was scored on a scale from 0 = &quot;Never or rarely&quot; to 3 = &quot;Very often&quot;.&#xD;
The ADHD-RS-5 total score was calculated as the sum of the 18 frequency item scores. The total score ranges from 0 to 54. Higher scores indicate greater symptom severity. Change from baseline value were calculated as the assessment value minus the baseline value.</description>
          <population>Full Analysis Set</population>
          <units>units on a scale</units>
          <param>Least Squares Mean</param>
          <dispersion>Standard Error</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="33"/>
                <count group_id="O2" value="34"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-9.17" spread="2.235"/>
                    <measurement group_id="O2" value="-11.34" spread="2.122"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Clinical Global Impression - Global Improvement (CGI -I) Response</title>
        <description>The CGI-I item is rated on a 7-point scale from 1 = &quot;Very much improved&quot;, 2 = &quot;Much improved&quot;, 3 = &quot;Minimally improved&quot;, 4 = &quot;No change&quot;, 5 = &quot;Minimally worse&quot;, 6 = &quot;Much worse&quot;, 7 = &quot;Very much worse&quot;.&#xD;
Response is defined as achieving a CGI-I score of 1 or 2, scores of 3 to 7 or missing are defined as Non Response</description>
        <time_frame>Visit 8 (Week 6)</time_frame>
        <population>Full Analysis Set</population>
        <group_list>
          <group group_id="O1">
            <title>AEVI-001</title>
            <description>100 mg, 200 mg or 400 mg of AEVI-001 b.i.d.</description>
          </group>
          <group group_id="O2">
            <title>Placebo</title>
            <description>Placebo b.i.d.</description>
          </group>
        </group_list>
        <measure>
          <title>Clinical Global Impression - Global Improvement (CGI -I) Response</title>
          <description>The CGI-I item is rated on a 7-point scale from 1 = &quot;Very much improved&quot;, 2 = &quot;Much improved&quot;, 3 = &quot;Minimally improved&quot;, 4 = &quot;No change&quot;, 5 = &quot;Minimally worse&quot;, 6 = &quot;Much worse&quot;, 7 = &quot;Very much worse&quot;.&#xD;
Response is defined as achieving a CGI-I score of 1 or 2, scores of 3 to 7 or missing are defined as Non Response</description>
          <population>Full Analysis Set</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="33"/>
                <count group_id="O2" value="34"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="9"/>
                    <measurement group_id="O2" value="11"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>1 year, 1 month, 4 days</time_frame>
      <desc>All AEs are collected from the time of the informed consent is signed until the follow-up call is completed. This includes events occurring during the screening phase of the study, regardless of whether investigational product (IP) is administered. An AE will be considered treatment emergent if it occurs after the first dose of IP and within 3 days of a subjects last dose of IP.</desc>
      <group_list>
        <group group_id="E1">
          <title>AEVI-001</title>
          <description>100 mg, 200 mg or 400 mg of AEVI-001 b.i.d.</description>
        </group>
        <group group_id="E2">
          <title>Placebo</title>
          <description>Placebo b.i.d.</description>
        </group>
      </group_list>
      <serious_events>
        <default_vocab>MedDRA version 19.0</default_vocab>
        <default_assessment>Non-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="34"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="35"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="34"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="35"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Psychiatric disorders</title>
            <event_list>
              <event>
                <sub_title>Irritability</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="34"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="35"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>5</frequency_threshold>
        <default_vocab>MedDRA version 19.0</default_vocab>
        <default_assessment>Non-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="12" subjects_at_risk="34"/>
                <counts group_id="E2" subjects_affected="11" subjects_at_risk="35"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Abdominal pain upper</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="34"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="35"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Pyrexia</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="34"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="35"/>
              </event>
              <event>
                <sub_title>Fatigue</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="34"/>
                <counts group_id="E2" events="3" subjects_affected="3" subjects_at_risk="35"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Upper respiratory tract infection</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="34"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="35"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Investigations</title>
            <event_list>
              <event>
                <sub_title>Weight increased</sub_title>
                <counts group_id="E1" events="4" subjects_affected="4" subjects_at_risk="34"/>
                <counts group_id="E2" events="4" subjects_affected="4" subjects_at_risk="35"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Nervous system disorders</title>
            <event_list>
              <event>
                <sub_title>Headache</sub_title>
                <counts group_id="E1" events="4" subjects_affected="3" subjects_at_risk="34"/>
                <counts group_id="E2" events="4" subjects_affected="4" subjects_at_risk="35"/>
              </event>
              <event>
                <sub_title>Sedation</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="34"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="35"/>
              </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>The information generated by this study is the property of Aevi Genomic Medicine, Inc. Publication or other public presentation of AEVI-001 data resulting from this study requires prior review and written approval of Aevi Genomic Medicine, Inc. Abstracts, manuscripts, and presentation materials should be provided to Aevi Genomic Medicine, Inc. for review at least 30 days prior to the relevant submission deadline.</restrictive_agreement>
    </certain_agreements>
    <point_of_contact>
      <name_or_title>Garry A. Neil, MD</name_or_title>
      <organization>Aevi Genomic Medicine</organization>
      <phone>610-254-4208</phone>
      <email>garry.neil@aevigenomics.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/NCT03265119
org study id: AEVI-001-ADHD-202
nct id: NCT03265119
lead sponsor: 
has dmc: No
is fda regulated drug: Yes
is fda regulated device: No
This is PART A of a 2-part, 6-week, double-blind, dose-optimization, parallel-group study in
      children and adolescents (ages 6-17 years) with ADHD with and without CNVs in specific genes
      implicated in glutamatergic signaling and neuronal activity. Parts A will include subjects
      determined to have a specific gene mutations implicated in glutamatergic signaling and
      neuronal connectivity.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Double (Participant, Investigator)
measure: Change From Baseline in Attention Deficit Hyperactivity Disorder Rating Scale, Version 5 (ADHD-RS-5) Total Score
time frame: Baseline to Visit 8 (Week 6)
description: The ADHD-RS-5 is comprised of 18 frequency items and 12 impairment items. Each frequency item was scored on a scale from 0 = "Never or rarely" to 3 = "Very often".
The ADHD-RS-5 total score was calculated as the sum of the 18 frequency item scores. The total score ranges from 0 to 54. Higher scores indicate greater symptom severity. Change from baseline value were calculated as the assessment value minus the baseline value.
measure: Clinical Global Impression - Global Improvement (CGI -I) Response
time frame: Visit 8 (Week 6)
description: The CGI-I item is rated on a 7-point scale from 1 = "Very much improved", 2 = "Much improved", 3 = "Minimally improved", 4 = "No change", 5 = "Minimally worse", 6 = "Much worse", 7 = "Very much worse".
Response is defined as achieving a CGI-I score of 1 or 2, scores of 3 to 7 or missing are defined as Non Response
arm group label: AEVI-001
arm group type: Experimental
arm group label: Placebo
arm group type: Placebo Comparator
intervention type: Drug
intervention name: AEVI-001
description: Oral doses of 100 mg, 200 mg or 400 mg of AEVI-001 will be administered twice daily during the treatment period.
arm group label: AEVI-001
other name: MDGN-001
other name: NFC-1
intervention type: Drug
intervention name: Placebo
description: Oral doses of 100 mg, 200 mg or 400 mg of Placebo will be administered twice daily during the treatment period.
arm group label: Placebo
criteria: 
gender: All
minimum age: 6 Years
maximum age: 17 Years
healthy volunteers: No
facility: 
country: United States
responsible party type: Sponsor
mesh term: Hyperkinesis
mesh term: Attention Deficit Disorder with Hyperactivity
sharing ipd: No
provided document: 
provided document: 
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
point of contact: 