<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/NCT02221869</url>
  </required_header>
  <id_info>
    <org_study_id>13-005</org_study_id>
    <nct_id>NCT02221869</nct_id>
  </id_info>
  <brief_title>A Multicenter Study of the Efficacy and Safety of Xyrem With an Open- Label Pharmacokinetic Evaluation and Safety Extension in Pediatric Subjects With Narcolepsy With Cataplexy</brief_title>
  <sponsors>
    <lead_sponsor>
      <agency>Jazz Pharmaceuticals</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Jazz Pharmaceuticals</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this trial is to assess the efficacy and safety of Xyrem in pediatrics&#xD;
      subjects with narcolepsy that includes cataplexy.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">October 1, 2014</start_date>
  <completion_date type="Actual">January 25, 2019</completion_date>
  <primary_completion_date type="Actual">February 2017</primary_completion_date>
  <phase>Phase 3</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>Double (Participant, Investigator)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Change in Weekly Number of Cataplexy Attacks</measure>
    <time_frame>From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)</time_frame>
    <description>Double-blind comparison of the change in weekly number of cataplexy attacks from the last 2 weeks of the Stable Dose Period to the 2 weeks of the Double-blind Treatment Period.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Clinical Global Impression of Change (CGIc) for Cataplexy Severity</measure>
    <time_frame>From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)</time_frame>
    <description>CGIc for cataplexy severity from the end of the Stable Dose Period to the end of the Double-blind Treatment Period.&#xD;
The CGIc is a 7-point scale ranging from &quot;very much improved&quot; to &quot;very much worse.&quot; A score of 0 = no change, a score of 3 = very much improved, and a score of -3 = very much worse.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change in the Epworth Sleepiness Scale (ESS) (CHAD) Score</measure>
    <time_frame>From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)</time_frame>
    <description>Change in the ESS (CHAD) score from the end of the Stable Dose Period to the end of the Double-blind Treatment Period.&#xD;
The ESS is a self-administered questionnaire with 8 questions. It provides a measure of a person's general level of daytime sleepiness, or their average sleep propensity in daily life. In the ESS for children and adolescents (CHAD), certain activities were modified. Each activity is scored on a scale ranging from 0-3, with 0 = would never fall asleep, and 3 = high chance of falling asleep. The total score ranges from 0-24, with a higher number representing an increased propensity for sleepiness.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>CGIc for Narcolepsy Overall</measure>
    <time_frame>From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)</time_frame>
    <description>CGIc for narcolepsy overall from the end of the Stable Dose Period to the end of the Double-blind Treatment Period.&#xD;
The CGIc is a 7-point scale ranging from &quot;very much improved&quot; to &quot;very much worse.&quot; A score of 0 = no change, a score of 3 = very much improved, and a score of -3 = very much worse.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change in Quality of Life (QoL; SF-10 Physical and Psychosocial Summary Score) From the End of the Stable Dose Period to the End of the Double-blind Treatment Period</measure>
    <time_frame>From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)</time_frame>
    <description>The SF-10 Health Survey for Children is a parent-completed survey that contains 10 questions adapted from the Child Health Questionnaire. The SF-10 is intended to produce physical and psychosocial health summary measures. Each of the 10 questions responses is scored with a point value from 1 to 6 (1 is the worst possible condition and 6 is the best possible condition). The SF-10 physical and psychosocial measures are scored such that higher scores indicate more favorable functioning.&#xD;
The questions and associated point values are separated into the Physical Health (PHS-10 domain) and Psychosocial Health (PSS-10 domain). The sums of the scores in each domain are standardized using the mean and standard deviation from a normal population (2006 sample). The standardized scores are transformed to norm based scoring (NBS) metric. Through NBS, scale scores are standardized to a mean of 50 and SD of 10 in the combined U.S. general population and clinical samples. NBS scores are reported</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">106</enrollment>
  <condition>Narcolepsy With Cataplexy</condition>
  <arm_group>
    <arm_group_label>Xyrem</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Active Xyrem at a dose ≤9 g/night</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Xyrem Placebo</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Xyrem placebo at a volume and regimen equivalent to the stable dose of Xyrem.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Xyrem</intervention_name>
    <arm_group_label>Xyrem</arm_group_label>
    <arm_group_label>Xyrem Placebo</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Male or female subjects aged 7-16 years at Visit 2 for subjects on Xyrem at study&#xD;
             entry and at Visit 1.1 for Xyrem-naïve subjects (to ensure subjects are &lt;18 years of&#xD;
             age at the end of the study)&#xD;
&#xD;
          2. Have a primary diagnosis of narcolepsy with cataplexy that meets International&#xD;
             Classification of Sleep Disorders (ICSD)-2 or ICSD-3 criteria, whichever was in effect&#xD;
             at the time of the diagnosis or, with the permission of the Medical Monitor, completes&#xD;
             a Multiple Sleep Latency Test (MSLT) during Screening to confirm the diagnosis of Type&#xD;
             1 narcolepsy by ICSD-3 criteria (i.e., the subject meets all other ICSD-3 criteria for&#xD;
             Type 1 narcolepsy)&#xD;
&#xD;
          3. Have given documented assent indicating that he/she was aware of the investigational&#xD;
             nature of the study and the required procedures and restrictions before participation&#xD;
             in any protocol-related activities&#xD;
&#xD;
          4. Have parent(s)/guardian(s) who have given informed consent for his/her/their child's&#xD;
             participation in the study&#xD;
&#xD;
          5. Be willing to spend the required number of nights (2 to 3) in a sleep laboratory for&#xD;
             PSG evaluations&#xD;
&#xD;
          6. If currently treated with Xyrem, must have been taking unchanged doses (twice nightly&#xD;
             dosing no higher than 9 g/night) of Xyrem, and stimulants, if applicable, for the&#xD;
             treatment of narcolepsy symptoms for at least 2 months prior to screening&#xD;
&#xD;
        In addition to the above inclusion criteria, subjects participating in the PK evaluation&#xD;
        must meet the following inclusion criteria:&#xD;
&#xD;
        7. Be willing to spend 2 additional nights in the clinic for PK evaluation&#xD;
&#xD;
        -&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Inability to understand assent or follow study instructions for any reason, in the&#xD;
             opinion of the Investigator&#xD;
&#xD;
          2. Parent(s) or guardian(s) unable to comply with the requirements of the study for any&#xD;
             reason, in the opinion of the Investigator&#xD;
&#xD;
          3. Other documented clinically significant condition (including an unstable medical&#xD;
             condition, chronic disease other than narcolepsy with cataplexy, or history or&#xD;
             presence of another neurological disorder) that might affect the subject's safety&#xD;
             and/or interfere with the conduct of the study in the opinion of the Investigator&#xD;
&#xD;
          4. Treatment with benzodiazepines, non-benzodiazepine anxiolytics/ hypnotics/sedatives,&#xD;
             neuroleptics, opioids, barbiturates, diclofenac, valproate, phenytoin, ethosuximide&#xD;
             within 2 weeks prior to enrollment (discontinuation for the purpose of study&#xD;
             enrollment is permitted only if considered safe by the Investigator and approved by&#xD;
             the Medical Monitor)&#xD;
&#xD;
          5. Treatment with any other medications that have anticataplectic effect (e.g.,&#xD;
             serotonin-norepinephrine reuptake inhibitors [SNRIs], selective serotonin reuptake&#xD;
             inhibitors [SSRIs], or tricyclic antidepressants [TCAs]) within 1 month before&#xD;
             Screening&#xD;
&#xD;
          6. Unsafe for the subject to receive placebo treatment for 2 weeks, in the opinion of the&#xD;
             Investigator&#xD;
&#xD;
        In addition to the above exclusion criteria, subjects participating in the PK evaluation&#xD;
        must not demonstrate the following:&#xD;
&#xD;
        -&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>7 Years</minimum_age>
    <maximum_age>17 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>Miller Children's Hospital - Long Beach</name>
      <address>
        <city>Long Beach</city>
        <state>California</state>
        <zip>90806</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>SDS Clinical Trials, Inc.</name>
      <address>
        <city>Orange</city>
        <state>California</state>
        <zip>92868</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Stanford Sleep Medicine Center</name>
      <address>
        <city>Redwood City</city>
        <state>California</state>
        <zip>94063</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Ann &amp; Robert H. Lurie Children's Hospital of Chicago</name>
      <address>
        <city>Chicago</city>
        <state>Illinois</state>
        <zip>60611</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The U-M Sleep Disorders Center</name>
      <address>
        <city>Ann Arbor</city>
        <state>Michigan</state>
        <zip>48109</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Montefiore Medical Center</name>
      <address>
        <city>Bronx</city>
        <state>New York</state>
        <zip>10467</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Duke Children's Hospital</name>
      <address>
        <city>Durham</city>
        <state>North Carolina</state>
        <zip>27710</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>ARSM Research, LLC</name>
      <address>
        <city>Huntersville</city>
        <state>North Carolina</state>
        <zip>28078</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Cincinnati Children's Hospital Medical Center</name>
      <address>
        <city>Cincinnati</city>
        <state>Ohio</state>
        <zip>45229</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University Hospitals Cleveland Medical Center</name>
      <address>
        <city>Cleveland</city>
        <state>Ohio</state>
        <zip>44106</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Nationwide Children's Hospital</name>
      <address>
        <city>Columbus</city>
        <state>Ohio</state>
        <zip>43205</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>SleepMed of SC</name>
      <address>
        <city>Columbia</city>
        <state>South Carolina</state>
        <zip>29201</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Greenville Health System</name>
      <address>
        <city>Greenville</city>
        <state>South Carolina</state>
        <zip>29615</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>UT/LeBonheur Neuroscience Institute</name>
      <address>
        <city>Memphis</city>
        <state>Tennessee</state>
        <zip>38105</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Todd Swick, MD, PA</name>
      <address>
        <city>Houston</city>
        <state>Texas</state>
        <zip>77063</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital of The King's Daughters</name>
      <address>
        <city>Norfolk</city>
        <state>Virginia</state>
        <zip>23507</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Seattle Children's Hospital</name>
      <address>
        <city>Seattle</city>
        <state>Washington</state>
        <zip>98105</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Robert Debre</name>
      <address>
        <city>Paris</city>
        <zip>75019</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Dipartimento di Scienze Biomediche e Biomotorie</name>
      <address>
        <city>Bologna</city>
        <zip>40139</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Sleep Wake Center SEIN Heemstede</name>
      <address>
        <city>Heemstede</city>
        <state>Noord Holland</state>
        <zip>2103 SW</zip>
        <country>Netherlands</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>France</country>
    <country>Italy</country>
    <country>Netherlands</country>
    <country>United States</country>
  </location_countries>
  <removed_countries>
    <country>Finland</country>
  </removed_countries>
  <verification_date>April 2019</verification_date>
  <study_first_submitted>July 28, 2014</study_first_submitted>
  <study_first_submitted_qc>August 19, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">August 21, 2014</study_first_posted>
  <results_first_submitted>November 23, 2018</results_first_submitted>
  <results_first_submitted_qc>April 26, 2019</results_first_submitted_qc>
  <results_first_posted type="Actual">April 30, 2019</results_first_posted>
  <disposition_first_submitted>January 19, 2018</disposition_first_submitted>
  <disposition_first_submitted_qc>January 19, 2018</disposition_first_submitted_qc>
  <disposition_first_posted type="Actual">January 25, 2018</disposition_first_posted>
  <last_update_submitted>April 26, 2019</last_update_submitted>
  <last_update_submitted_qc>April 26, 2019</last_update_submitted_qc>
  <last_update_posted type="Actual">April 30, 2019</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>Narcolepsy</mesh_term>
    <mesh_term>Cataplexy</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>February 23, 2017</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/69/NCT02221869/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>January 19, 2018</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/69/NCT02221869/SAP_001.pdf</document_url>
    </provided_document>
  </provided_document_section>

  <clinical_results>

    <participant_flow>
      <recruitment_details>106 subjects were enrolled. Xyrem-naïve subjects (n=74) entered the Dose Titration Period (3 to 10 weeks). Xyrem-naïve and on Xyrem subjects (n= 99) entered the Stable Dose Period (2 to 3 weeks). 96 subjects then entered the Double-Blind Randomized Withdrawal Period (2 weeks). 95 subjects then entered the Open-label Safety Period (38 to 47 weeks).</recruitment_details>
      <pre_assignment_details>Subjects aged 7-17 who were being treated with Xyrem or Xyrem naïve were eligible for the study. 63 subjects were randomized and 33 received open-label Xyrem during the Double-blind Treatment Period. 106 and 63 subjects comprised the Enrolled population and the Efficacy population respectively.</pre_assignment_details>
      <group_list>
        <group group_id="P1">
          <title>Randomized to Xyrem</title>
          <description>Active Xyrem continued as a double-blind treatment at the stable dose taken and regimen taken in the prior 2 weeks.</description>
        </group>
        <group group_id="P2">
          <title>Randomized to Xyrem Placebo</title>
          <description>Xyrem placebo was initiated as a double-blind treatment at a volume and regimen equivalent to the Xyrem dose taken in the prior 2 weeks.</description>
        </group>
        <group group_id="P3">
          <title>Open-label Xyrem</title>
          <description>Subjects who were not randomized or continued to receive open-label Xyrem during the Double-blind Treatment Period.</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="31"/>
                <participants group_id="P2" count="32"/>
                <participants group_id="P3" count="43"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="30"/>
                <participants group_id="P2" count="32"/>
                <participants group_id="P3" count="33"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
                <participants group_id="P2" count="0"/>
                <participants group_id="P3" count="10"/>
              </participants_list>
            </milestone>
          </milestone_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <population>106 subjects were enrolled. 63 subjects were randomized during the Double-blind Treatment Period. 106 subjects comprised the Enrolled population, and 63 subjects comprised the Efficacy population.</population>
      <group_list>
        <group group_id="B1">
          <title>Enrolled Population</title>
          <description>The Enrolled Population consists of all subjects who were dispensed study drug.</description>
        </group>
        <group group_id="B2">
          <title>Placebo (Efficacy Population)</title>
          <description>Xyrem placebo was initiated as a double-blind treatment at a volume and regimen equivalent to the Xyrem dose taken in the prior 2 weeks in the 2-week double-blind treatment period.</description>
        </group>
        <group group_id="B3">
          <title>Xyrem (Efficacy Population)</title>
          <description>Active Xyrem continued as a double-blind treatment at the stable dose taken and regimen taken in the prior 2 weeks in the 2-week double-blind treatment period.</description>
        </group>
        <group group_id="B4">
          <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="106"/>
            <count group_id="B2" value="32"/>
            <count group_id="B3" value="31"/>
            <count group_id="B4" value="169"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <population>Measure Analysis Population Description: Data analyzed separately for Enrolled and Efficacy populations</population>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <title>Enrolled Population</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="106"/>
                    <count group_id="B2" value="0"/>
                    <count group_id="B3" value="0"/>
                    <count group_id="B4" value="106"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <title>&lt;=18 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="106"/>
                    <measurement group_id="B4" value="106"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Between 18 and 65 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B4" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>&gt;=65 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B4" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Efficacy Population</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="0"/>
                    <count group_id="B2" value="32"/>
                    <count group_id="B3" value="31"/>
                    <count group_id="B4" value="63"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <title>&lt;=18 years</title>
                  <measurement_list>
                    <measurement group_id="B2" value="32"/>
                    <measurement group_id="B3" value="31"/>
                    <measurement group_id="B4" value="63"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Between 18 and 65 years</title>
                  <measurement_list>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>&gt;=65 years</title>
                  <measurement_list>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Sex: Female, Male</title>
          <population>Measure Analysis Population Description: Data analyzed separately for Enrolled and Efficacy populations</population>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <title>Enrolled Population</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="106"/>
                    <count group_id="B2" value="0"/>
                    <count group_id="B3" value="0"/>
                    <count group_id="B4" value="106"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <title>Female</title>
                  <measurement_list>
                    <measurement group_id="B1" value="43"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B4" value="43"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="63"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B4" value="63"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Efficacy Population</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="0"/>
                    <count group_id="B2" value="32"/>
                    <count group_id="B3" value="31"/>
                    <count group_id="B4" value="63"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <title>Female</title>
                  <measurement_list>
                    <measurement group_id="B2" value="15"/>
                    <measurement group_id="B3" value="13"/>
                    <measurement group_id="B4" value="28"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B2" value="17"/>
                    <measurement group_id="B3" value="18"/>
                    <measurement group_id="B4" value="35"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Ethnicity (NIH/OMB)</title>
          <population>Measure Analysis Population Description: Data analyzed separately for Enrolled and Efficacy populations</population>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <title>Enrolled Population</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="106"/>
                    <count group_id="B2" value="0"/>
                    <count group_id="B3" value="0"/>
                    <count group_id="B4" value="106"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <title>Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="6"/>
                    <measurement group_id="B4" value="6"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Not Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="100"/>
                    <measurement group_id="B4" value="100"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B4" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Efficacy Population</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="0"/>
                    <count group_id="B2" value="32"/>
                    <count group_id="B3" value="31"/>
                    <count group_id="B4" value="63"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <title>Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B2" value="2"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="2"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Not Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B2" value="30"/>
                    <measurement group_id="B3" value="31"/>
                    <measurement group_id="B4" value="61"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Region of Enrollment</title>
          <population>Measure Analysis Population Description: Data reported for the Enrolled Population</population>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <title>Netherlands</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="106"/>
                    <count group_id="B2" value="0"/>
                    <count group_id="B3" value="0"/>
                    <count group_id="B4" value="106"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="8"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="8"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>United States</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="106"/>
                    <count group_id="B2" value="0"/>
                    <count group_id="B3" value="0"/>
                    <count group_id="B4" value="106"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="62"/>
                    <measurement group_id="B4" value="62"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Italy</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="106"/>
                    <count group_id="B2" value="0"/>
                    <count group_id="B3" value="0"/>
                    <count group_id="B4" value="106"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="25"/>
                    <measurement group_id="B4" value="25"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>France</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="106"/>
                    <count group_id="B2" value="0"/>
                    <count group_id="B3" value="0"/>
                    <count group_id="B4" value="106"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="10"/>
                    <measurement group_id="B4" value="10"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Finland</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="106"/>
                    <count group_id="B2" value="0"/>
                    <count group_id="B3" value="0"/>
                    <count group_id="B4" value="106"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                    <measurement group_id="B4" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Region of Enrollment</title>
          <population>Measure Analysis Population Description: Data reported for Efficacy Population</population>
          <units>participants</units>
          <param>Number</param>
          <class_list>
            <class>
              <title>Netherlands</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="0"/>
                    <count group_id="B2" value="32"/>
                    <count group_id="B3" value="31"/>
                    <count group_id="B4" value="63"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B2" value="3"/>
                    <measurement group_id="B3" value="2"/>
                    <measurement group_id="B4" value="5"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>United States</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="0"/>
                    <count group_id="B2" value="32"/>
                    <count group_id="B3" value="31"/>
                    <count group_id="B4" value="63"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B2" value="17"/>
                    <measurement group_id="B3" value="16"/>
                    <measurement group_id="B4" value="33"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Italy</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="0"/>
                    <count group_id="B2" value="32"/>
                    <count group_id="B3" value="31"/>
                    <count group_id="B4" value="63"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B2" value="9"/>
                    <measurement group_id="B3" value="9"/>
                    <measurement group_id="B4" value="18"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>France</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="0"/>
                    <count group_id="B2" value="32"/>
                    <count group_id="B3" value="31"/>
                    <count group_id="B4" value="63"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B2" value="3"/>
                    <measurement group_id="B3" value="4"/>
                    <measurement group_id="B4" value="7"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Change in Weekly Number of Cataplexy Attacks</title>
        <description>Double-blind comparison of the change in weekly number of cataplexy attacks from the last 2 weeks of the Stable Dose Period to the 2 weeks of the Double-blind Treatment Period.</description>
        <time_frame>From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Xyrem</title>
            <description>Active Xyrem continued as a double-blind treatment at the stable dose taken and regimen used in the prior 2 weeks.</description>
          </group>
          <group group_id="O2">
            <title>Xyrem Placebo</title>
            <description>Xyrem placebo was initiated as a double-blind treatment at a volume and regimen equivalent to the Xyrem dose taken in the prior 2 weeks.</description>
          </group>
        </group_list>
        <measure>
          <title>Change in Weekly Number of Cataplexy Attacks</title>
          <description>Double-blind comparison of the change in weekly number of cataplexy attacks from the last 2 weeks of the Stable Dose Period to the 2 weeks of the Double-blind Treatment Period.</description>
          <units>number of attacks</units>
          <param>Median</param>
          <dispersion>Inter-Quartile Range</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="31"/>
                <count group_id="O2" value="32"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.27" lower_limit="-1.00" upper_limit="2.50"/>
                    <measurement group_id="O2" value="12.71" lower_limit="3.44" upper_limit="19.77"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Clinical Global Impression of Change (CGIc) for Cataplexy Severity</title>
        <description>CGIc for cataplexy severity from the end of the Stable Dose Period to the end of the Double-blind Treatment Period.&#xD;
The CGIc is a 7-point scale ranging from &quot;very much improved&quot; to &quot;very much worse.&quot; A score of 0 = no change, a score of 3 = very much improved, and a score of -3 = very much worse.</description>
        <time_frame>From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Xyrem</title>
            <description>Active Xyrem continued as a double-blind treatment at the stable dose taken and regimen used in the prior 2 weeks.</description>
          </group>
          <group group_id="O2">
            <title>Xyrem Placebo</title>
            <description>Xyrem placebo was initiated as a double-blind treatment at a volume and regimen equivalent to the Xyrem dose taken in the prior 2 weeks.</description>
          </group>
        </group_list>
        <measure>
          <title>Clinical Global Impression of Change (CGIc) for Cataplexy Severity</title>
          <description>CGIc for cataplexy severity from the end of the Stable Dose Period to the end of the Double-blind Treatment Period.&#xD;
The CGIc is a 7-point scale ranging from &quot;very much improved&quot; to &quot;very much worse.&quot; A score of 0 = no change, a score of 3 = very much improved, and a score of -3 = very much worse.</description>
          <units>score on a scale</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="29"/>
                <count group_id="O2" value="32"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.4" spread="1.12"/>
                    <measurement group_id="O2" value="-1.5" spread="1.19"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change in the Epworth Sleepiness Scale (ESS) (CHAD) Score</title>
        <description>Change in the ESS (CHAD) score from the end of the Stable Dose Period to the end of the Double-blind Treatment Period.&#xD;
The ESS is a self-administered questionnaire with 8 questions. It provides a measure of a person's general level of daytime sleepiness, or their average sleep propensity in daily life. In the ESS for children and adolescents (CHAD), certain activities were modified. Each activity is scored on a scale ranging from 0-3, with 0 = would never fall asleep, and 3 = high chance of falling asleep. The total score ranges from 0-24, with a higher number representing an increased propensity for sleepiness.</description>
        <time_frame>From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Xyrem</title>
            <description>Active Xyrem continued as a double-blind treatment at the stable dose taken and regimen used in the prior 2 weeks.</description>
          </group>
          <group group_id="O2">
            <title>Xyrem Placebo</title>
            <description>Xyrem placebo was initiated as a double-blind treatment at a volume and regimen equivalent to the Xyrem dose taken in the prior 2 weeks.</description>
          </group>
        </group_list>
        <measure>
          <title>Change in the Epworth Sleepiness Scale (ESS) (CHAD) Score</title>
          <description>Change in the ESS (CHAD) score from the end of the Stable Dose Period to the end of the Double-blind Treatment Period.&#xD;
The ESS is a self-administered questionnaire with 8 questions. It provides a measure of a person's general level of daytime sleepiness, or their average sleep propensity in daily life. In the ESS for children and adolescents (CHAD), certain activities were modified. Each activity is scored on a scale ranging from 0-3, with 0 = would never fall asleep, and 3 = high chance of falling asleep. The total score ranges from 0-24, with a higher number representing an increased propensity for sleepiness.</description>
          <units>score on a scale</units>
          <param>Median</param>
          <dispersion>Inter-Quartile Range</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="30"/>
                <count group_id="O2" value="31"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.0" lower_limit="-1.0" upper_limit="2.0"/>
                    <measurement group_id="O2" value="3.0" lower_limit="1.0" upper_limit="5.0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>CGIc for Narcolepsy Overall</title>
        <description>CGIc for narcolepsy overall from the end of the Stable Dose Period to the end of the Double-blind Treatment Period.&#xD;
The CGIc is a 7-point scale ranging from &quot;very much improved&quot; to &quot;very much worse.&quot; A score of 0 = no change, a score of 3 = very much improved, and a score of -3 = very much worse.</description>
        <time_frame>From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Xyrem</title>
            <description>Active Xyrem continued as a double-blind treatment at the stable dose taken and regimen used in the prior 2 weeks.</description>
          </group>
          <group group_id="O2">
            <title>Xyrem Placebo</title>
            <description>Xyrem placebo was initiated as a double-blind treatment at a volume and regimen equivalent to the Xyrem dose taken in the prior 2 weeks.</description>
          </group>
        </group_list>
        <measure>
          <title>CGIc for Narcolepsy Overall</title>
          <description>CGIc for narcolepsy overall from the end of the Stable Dose Period to the end of the Double-blind Treatment Period.&#xD;
The CGIc is a 7-point scale ranging from &quot;very much improved&quot; to &quot;very much worse.&quot; A score of 0 = no change, a score of 3 = very much improved, and a score of -3 = very much worse.</description>
          <units>score on a scale</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="29"/>
                <count group_id="O2" value="32"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.4" spread="0.95"/>
                    <measurement group_id="O2" value="-1.4" spread="1.13"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change in Quality of Life (QoL; SF-10 Physical and Psychosocial Summary Score) From the End of the Stable Dose Period to the End of the Double-blind Treatment Period</title>
        <description>The SF-10 Health Survey for Children is a parent-completed survey that contains 10 questions adapted from the Child Health Questionnaire. The SF-10 is intended to produce physical and psychosocial health summary measures. Each of the 10 questions responses is scored with a point value from 1 to 6 (1 is the worst possible condition and 6 is the best possible condition). The SF-10 physical and psychosocial measures are scored such that higher scores indicate more favorable functioning.&#xD;
The questions and associated point values are separated into the Physical Health (PHS-10 domain) and Psychosocial Health (PSS-10 domain). The sums of the scores in each domain are standardized using the mean and standard deviation from a normal population (2006 sample). The standardized scores are transformed to norm based scoring (NBS) metric. Through NBS, scale scores are standardized to a mean of 50 and SD of 10 in the combined U.S. general population and clinical samples. NBS scores are reported</description>
        <time_frame>From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Xyrem</title>
            <description>Active Xyrem continued as a double-blind treatment at the stable dose taken and regimen used in the prior 2 weeks.</description>
          </group>
          <group group_id="O2">
            <title>Xyrem Placebo</title>
            <description>Xyrem placebo was initiated as a double-blind treatment at a volume and regimen equivalent to the Xyrem dose taken in the prior 2 weeks.</description>
          </group>
        </group_list>
        <measure>
          <title>Change in Quality of Life (QoL; SF-10 Physical and Psychosocial Summary Score) From the End of the Stable Dose Period to the End of the Double-blind Treatment Period</title>
          <description>The SF-10 Health Survey for Children is a parent-completed survey that contains 10 questions adapted from the Child Health Questionnaire. The SF-10 is intended to produce physical and psychosocial health summary measures. Each of the 10 questions responses is scored with a point value from 1 to 6 (1 is the worst possible condition and 6 is the best possible condition). The SF-10 physical and psychosocial measures are scored such that higher scores indicate more favorable functioning.&#xD;
The questions and associated point values are separated into the Physical Health (PHS-10 domain) and Psychosocial Health (PSS-10 domain). The sums of the scores in each domain are standardized using the mean and standard deviation from a normal population (2006 sample). The standardized scores are transformed to norm based scoring (NBS) metric. Through NBS, scale scores are standardized to a mean of 50 and SD of 10 in the combined U.S. general population and clinical samples. NBS scores are reported</description>
          <units>score on a scale</units>
          <param>Median</param>
          <dispersion>Inter-Quartile Range</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="30"/>
                <count group_id="O2" value="30"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>SF-10 Physical Summary Score</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0" lower_limit="-2.730" upper_limit="2.500"/>
                    <measurement group_id="O2" value="0" lower_limit="-8.420" upper_limit="2.730"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>SF-10 Psychosocial Summary Score</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0" lower_limit="-6.240" upper_limit="2.680"/>
                    <measurement group_id="O2" value="-2.670" lower_limit="-8.020" upper_limit="2.670"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>Safety data are provided through the 120 day update.</time_frame>
      <desc>The Safety Population included all subjects who took study drug, and consisted of 104 subjects. There are 2 subjects included in the Enrolled Population who were dispensed study drug, but did not take the medication and were subsequently discontinued. As a result, safety analyses focused on the 104 subjects in the Safety Population. Adverse events are reported by the Safety Population in order to capture adverse events occurring across all treatment periods.</desc>
      <group_list>
        <group group_id="E1">
          <title>Safety Population</title>
          <description>Safety population who took study drug</description>
        </group>
        <group group_id="E2">
          <title>Randomized Placebo</title>
          <description>Subjects assigned to the Randomized Placebo group during the Double-blind Treatment Period. Adverse events with onset on or after the first dose in the Double-blind Treatment Period and prior to the date of first dose in the Open-label Safety Period are presented.</description>
        </group>
      </group_list>
      <serious_events>
        <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="104"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="32"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="104"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="32"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Psychiatric disorders</title>
            <event_list>
              <event>
                <sub_title>Acute Psychosis</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="104"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="32"/>
              </event>
              <event>
                <sub_title>Suicidal Ideation</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="104"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="32"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>5</frequency_threshold>
        <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="80" subjects_at_risk="104"/>
                <counts group_id="E2" subjects_affected="10" subjects_at_risk="32"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Nausea</sub_title>
                <counts group_id="E1" subjects_affected="20" subjects_at_risk="104"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="32"/>
              </event>
              <event>
                <sub_title>Vomiting</sub_title>
                <counts group_id="E1" subjects_affected="19" subjects_at_risk="104"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="32"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Nasopharyngitis</sub_title>
                <counts group_id="E1" subjects_affected="9" subjects_at_risk="104"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="32"/>
              </event>
              <event>
                <sub_title>Upper respiratory tract infection</sub_title>
                <counts group_id="E1" subjects_affected="9" subjects_at_risk="104"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="32"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Investigations</title>
            <event_list>
              <event>
                <sub_title>Weight decreased</sub_title>
                <counts group_id="E1" subjects_affected="12" subjects_at_risk="104"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="32"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Metabolism and nutrition disorders</title>
            <event_list>
              <event>
                <sub_title>Decreased appetite</sub_title>
                <counts group_id="E1" subjects_affected="9" subjects_at_risk="104"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="32"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Nervous system disorders</title>
            <event_list>
              <event>
                <sub_title>Headache</sub_title>
                <counts group_id="E1" subjects_affected="18" subjects_at_risk="104"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="32"/>
              </event>
              <event>
                <sub_title>Dizziness</sub_title>
                <counts group_id="E1" subjects_affected="8" subjects_at_risk="104"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="32"/>
              </event>
              <event>
                <sub_title>Somnolence</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="104"/>
                <counts group_id="E2" subjects_affected="7" subjects_at_risk="32"/>
              </event>
              <event>
                <sub_title>Cataplexy</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="104"/>
                <counts group_id="E2" subjects_affected="6" subjects_at_risk="32"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Psychiatric disorders</title>
            <event_list>
              <event>
                <sub_title>Sleep disorder</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="104"/>
                <counts group_id="E2" subjects_affected="2" subjects_at_risk="32"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Renal and urinary disorders</title>
            <event_list>
              <event>
                <sub_title>Enuresis</sub_title>
                <counts group_id="E1" subjects_affected="20" subjects_at_risk="104"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="32"/>
              </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 sponsor can review trial results communications prior to public release and can embargo such communications for a period of at least 60 days from the time submitted to sponsor for review. If requested by sponsor, the PI will withhold publication for up to an additional 30 days. Furthermore, the first publication of study results must be a joint publication of all study sites unless a joint manuscript has not been submitted for publication within 12 months of completion of the study.</restrictive_agreement>
    </certain_agreements>
    <point_of_contact>
      <name_or_title>Director, Disclosure &amp; Transparency</name_or_title>
      <organization>Jazz Pharmaceuticals</organization>
      <phone>215-970-7145</phone>
      <email>ClinicalTrialDisclosure@JazzPharma.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/NCT02221869
org study id: 13-005
nct id: NCT02221869
lead sponsor: 
has dmc: Yes
The purpose of this trial is to assess the efficacy and safety of Xyrem in pediatrics
      subjects with narcolepsy that includes cataplexy.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Double (Participant, Investigator)
measure: Change in Weekly Number of Cataplexy Attacks
time frame: From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)
description: Double-blind comparison of the change in weekly number of cataplexy attacks from the last 2 weeks of the Stable Dose Period to the 2 weeks of the Double-blind Treatment Period.
measure: Clinical Global Impression of Change (CGIc) for Cataplexy Severity
time frame: From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)
description: CGIc for cataplexy severity from the end of the Stable Dose Period to the end of the Double-blind Treatment Period.
The CGIc is a 7-point scale ranging from "very much improved" to "very much worse." A score of 0 = no change, a score of 3 = very much improved, and a score of -3 = very much worse.
measure: Change in the Epworth Sleepiness Scale (ESS) (CHAD) Score
time frame: From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)
description: Change in the ESS (CHAD) score from the end of the Stable Dose Period to the end of the Double-blind Treatment Period.
The ESS is a self-administered questionnaire with 8 questions. It provides a measure of a person's general level of daytime sleepiness, or their average sleep propensity in daily life. In the ESS for children and adolescents (CHAD), certain activities were modified. Each activity is scored on a scale ranging from 0-3, with 0 = would never fall asleep, and 3 = high chance of falling asleep. The total score ranges from 0-24, with a higher number representing an increased propensity for sleepiness.
measure: CGIc for Narcolepsy Overall
time frame: From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)
description: CGIc for narcolepsy overall from the end of the Stable Dose Period to the end of the Double-blind Treatment Period.
The CGIc is a 7-point scale ranging from "very much improved" to "very much worse." A score of 0 = no change, a score of 3 = very much improved, and a score of -3 = very much worse.
measure: Change in Quality of Life (QoL; SF-10 Physical and Psychosocial Summary Score) From the End of the Stable Dose Period to the End of the Double-blind Treatment Period
time frame: From the end of the Stable Dose Period to the end of the Double-blind Treatment Period (2 weeks)
description: The SF-10 Health Survey for Children is a parent-completed survey that contains 10 questions adapted from the Child Health Questionnaire. The SF-10 is intended to produce physical and psychosocial health summary measures. Each of the 10 questions responses is scored with a point value from 1 to 6 (1 is the worst possible condition and 6 is the best possible condition). The SF-10 physical and psychosocial measures are scored such that higher scores indicate more favorable functioning.
The questions and associated point values are separated into the Physical Health (PHS-10 domain) and Psychosocial Health (PSS-10 domain). The sums of the scores in each domain are standardized using the mean and standard deviation from a normal population (2006 sample). The standardized scores are transformed to norm based scoring (NBS) metric. Through NBS, scale scores are standardized to a mean of 50 and SD of 10 in the combined U.S. general population and clinical samples. NBS scores are reported
arm group label: Xyrem
arm group type: Experimental
description: Active Xyrem at a dose ≤9 g/night
arm group label: Xyrem Placebo
arm group type: Placebo Comparator
description: Xyrem placebo at a volume and regimen equivalent to the stable dose of Xyrem.
intervention type: Drug
intervention name: Xyrem
arm group label: Xyrem
arm group label: Xyrem Placebo
criteria: 
gender: All
minimum age: 7 Years
maximum age: 17 Years
healthy volunteers: No
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country: France
country: Italy
country: Netherlands
country: United States
country: Finland
responsible party type: Sponsor
mesh term: Narcolepsy
mesh term: Cataplexy
provided document: 
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participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
point of contact: 