<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/NCT01661335</url>
  </required_header>
  <id_info>
    <org_study_id>0413</org_study_id>
    <nct_id>NCT01661335</nct_id>
  </id_info>
  <brief_title>Efficacy of Aprepitant (Emend®) in Children</brief_title>
  <official_title>Efficacy of Aprepitant (Emend®) in Children Receiving Highly Emetogenic Chemotherapy</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>University of Oklahoma</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>University of Oklahoma</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this study is to find out whether or not adding aprepitant(Emend®) to the&#xD;
      standard therapy will help children who receive chemotherapy to have less nausea and&#xD;
      vomiting.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      1.1 Primary Aim To determine the efficacy of aprepitant (Emend®) in preventing and reducing&#xD;
      chemotherapy-induced nausea and vomiting (CINV) when added to standard antiemetic drug&#xD;
      regimens for children receiving highly emetogenic chemotherapy. The working hypothesis will&#xD;
      be that standard therapy + aprepitant is superior at preventing CINV than standard therapy +&#xD;
      placebo.&#xD;
&#xD;
      1.2 Secondary Aim To evaluate the safety and toxicity of aprepitant (Emend®) in children&#xD;
      receiving highly emetogenic chemotherapy when compared to standard antiemetic therapy +&#xD;
      placebo.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">June 1, 2012</start_date>
  <completion_date type="Actual">June 29, 2017</completion_date>
  <primary_completion_date type="Actual">June 29, 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>Crossover Assignment</intervention_model>
    <primary_purpose>Prevention</primary_purpose>
    <masking>Triple (Participant, Care Provider, Investigator)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Efficacy of aprepitant (Emend®) measured through a complete response</measure>
    <time_frame>Up to 11 weeks, or until 3 weeks after the second course of the study regimen</time_frame>
    <description>• Percentage of study subjects who demonstrate a complete response, defined as no episodes of emesis and no use of rescue medications during the investigational antiemetic cycles.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Efficacy of aprepitant (Emend®) measured through episodes of emesis and use of rescue medication.</measure>
    <time_frame>Up to 11 weeks, or until 3 weeks after the second course of the study regimen</time_frame>
    <description>The total episodes of emesis within 7 days of the first chemotherapy administration of each cycle.&#xD;
The total number of administrations of rescue medications given for breakthrough nausea or vomiting.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Efficacy of aprepitant (Emend®) measured through impact of chemotherapy induced nausea and vomiting on daily life</measure>
    <time_frame>Up to 11 weeks, or until 3 weeks after the second course of the study regimen</time_frame>
    <description>• A modified, 5-day recall version of the Functional Living Index-Emesis (FLIE) questionnaire</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Efficacy of aprepitant (Emend®) measured through a pictorial nausea scale</measure>
    <time_frame>Up to 11 weeks, or until 3 weeks after the second course of the study regimen</time_frame>
    <description>• A modified version of the Baxter Animated Retching Faces (BARF) scale, administered daily.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Safety of aprepitant (Emend®)</measure>
    <time_frame>Up to 11 weeks, or until 3 weeks after the second course of the study regimen</time_frame>
    <description>Occurrence of adverse events as per the NCI Common Terminology Criteria for Adverse Events (CTCAE) v.4.0. These will be reported spontaneously or on inquiry by the investigator/study nurse, and continuously monitored throughout the trial.&#xD;
Weekly complete blood count (CBC) for 3 weeks after each investigational antiemetic cycle.&#xD;
Weekly complete metabolic profile (CMP) for 3 weeks after each investigational antiemetic cycle.</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">19</enrollment>
  <condition>Nausea</condition>
  <condition>Vomiting</condition>
  <condition>Childhood Cancer</condition>
  <arm_group>
    <arm_group_label>Ondansetron, dexamethasone, aprepitant</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Arm A: Ondansetron 0.15 mg/kg (max 16 mg) IV or PO every 8 hours for at least 3 days, but no longer than 5 days; dexamethasone 0.2mg/kg (max 10 mg) IV or PO daily for at least 3 days, but no longer than 5 days; and aprepitant 3 mg/kg (max 125 mg) PO on day 1, and aprepitant 2 mg/kg (max 80 mg) PO on days 2 and 3 during the first investigational antiemetic cycle. During the next investigational antiemetic cycle, members of this arm will be crossed-over into the placebo arm, where the aprepitant will be replaced by placebo and the dexamethasone dose will be increased to 0.4 mg/kg (max 20 mg) daily.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Ondansetron, Dexamethasone, placebo</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Arm B: Ondansetron 0.15 mg/kg (max 16 mg) IV or PO every 8 hours for at least 3 days, but no more than 5 days; dexamethasone 0.4 mg/kg (max 20 mg) IV or PO daily for at least 3 days, but no more than 5 days; and a PO placebo for 3 days during the first investigational antiemetic cycle. During the second cycle, members this group will be crossed-over to the experimental arm, where the placebo will be replaced by aprepitant and the dexamethasone will be decreased by 50%.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Ondansetron, dexamethasone, aprepitant</intervention_name>
    <description>Ondansetron 0.15 mg/kg (max 16 mg) IV or PO every 8 hours for at least 3 days, but no longer than 5 days; dexamethasone 0.2mg/kg (max 10 mg) IV or PO daily for at least 3 days, but no longer than 5 days; and aprepitant 3 mg/kg (max 125 mg) PO on day 1, and aprepitant 2 mg/kg (max 80 mg) PO on days 2 and 3 during the first investigational antiemetic cycle. During the next investigational antiemetic cycle, members of this arm will be crossed-over into the placebo arm, where the aprepitant will be replaced by placebo and the dexamethasone dose will be increased to 0.4 mg/kg (max 20 mg) daily.</description>
    <arm_group_label>Ondansetron, dexamethasone, aprepitant</arm_group_label>
    <other_name>Aprepitant = Emend</other_name>
    <other_name>ARM A</other_name>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Ondansetron, Dexamethasone, placebo</intervention_name>
    <description>Ondansetron 0.15 mg/kg (max 16 mg) IV or PO every 8 hours for at least 3 days, but no more than 5 days; dexamethasone 0.4 mg/kg (max 20 mg) IV or PO daily for at least 3 days, but no more than 5 days; and a PO placebo for 3 days during the first investigational antiemetic cycle. During the second cycle, members this group will be crossed-over to the experimental arm, where the placebo will be replaced by aprepitant and the dexamethasone will be decreased by 50%.</description>
    <arm_group_label>Ondansetron, Dexamethasone, placebo</arm_group_label>
    <other_name>ARM B</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
        under 20.99 years of age at enrollment&#xD;
&#xD;
        Scheduled to receive two identical cycles of highly emetogenic[1] chemotherapy for&#xD;
        treatment of a primary malignancy, including:&#xD;
&#xD;
        Chemotherapy with any one or more of the following single agents in any combination:&#xD;
&#xD;
          -  Carboplatin&#xD;
&#xD;
          -  Carmustine &gt;250 mg/m2&#xD;
&#xD;
          -  Cisplatin&#xD;
&#xD;
          -  Cyclophosphamide ≥1 g/m2&#xD;
&#xD;
          -  Dactinomycin&#xD;
&#xD;
          -  High dose Methotrexate ≥ 5 g/m2&#xD;
&#xD;
        Or any of the following defined combinations:&#xD;
&#xD;
          -  Cyclophosphamide + anthracycline&#xD;
&#xD;
          -  Cyclophosphamide + etoposide&#xD;
&#xD;
          -  Cytarabine 150-200 mg/m2 + daunorubicin&#xD;
&#xD;
          -  Cytarabine 300 mg/m2 + etoposide&#xD;
&#xD;
          -  Cytarabine 300 mg/m2 + teniposide&#xD;
&#xD;
          -  Doxorubicin + ifosfamide&#xD;
&#xD;
          -  Doxorubicin + methotrexate 5 g/m2&#xD;
&#xD;
          -  Etoposide + ifosfamide&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Patients who have received aprepitant in the past.&#xD;
&#xD;
          -  Patients who demonstrate evidence of increased intracranial pressure.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>6 Months</minimum_age>
    <maximum_age>20 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Rene McNall-Knapp, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>University of Oklahoma</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Jimmy Everest Center for Cancer and Blood Disorders in Children</name>
      <address>
        <city>Oklahoma City</city>
        <state>Oklahoma</state>
        <zip>73104</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>March 2021</verification_date>
  <study_first_submitted>June 18, 2012</study_first_submitted>
  <study_first_submitted_qc>August 8, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">August 9, 2012</study_first_posted>
  <last_update_submitted>March 18, 2021</last_update_submitted>
  <last_update_submitted_qc>March 18, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">March 23, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Nausea</keyword>
  <keyword>Vomiting</keyword>
  <keyword>Children</keyword>
  <keyword>Aprepitant</keyword>
  <keyword>Emend®</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Nausea</mesh_term>
    <mesh_term>Vomiting</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Dexamethasone</mesh_term>
    <mesh_term>Dexamethasone acetate</mesh_term>
    <mesh_term>Ondansetron</mesh_term>
    <mesh_term>Aprepitant</mesh_term>
    <mesh_term>Fosaprepitant</mesh_term>
    <mesh_term>BB 1101</mesh_term>
  </intervention_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <pending_results>
    <submitted>March 24, 2021</submitted>
    <returned>April 20, 2021</returned>
    <submitted>May 3, 2022</submitted>
    <returned>May 26, 2022</returned>
  </pending_results>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT01661335
org study id: 0413
nct id: NCT01661335
lead sponsor: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
The purpose of this study is to find out whether or not adding aprepitant(Emend®) to the
      standard therapy will help children who receive chemotherapy to have less nausea and
      vomiting.
1.1 Primary Aim To determine the efficacy of aprepitant (Emend®) in preventing and reducing
      chemotherapy-induced nausea and vomiting (CINV) when added to standard antiemetic drug
      regimens for children receiving highly emetogenic chemotherapy. The working hypothesis will
      be that standard therapy + aprepitant is superior at preventing CINV than standard therapy +
      placebo.

      1.2 Secondary Aim To evaluate the safety and toxicity of aprepitant (Emend®) in children
      receiving highly emetogenic chemotherapy when compared to standard antiemetic therapy +
      placebo.
allocation: Randomized
intervention model: Crossover Assignment
primary purpose: Prevention
masking: Triple (Participant, Care Provider, Investigator)
measure: Efficacy of aprepitant (Emend®) measured through a complete response
time frame: Up to 11 weeks, or until 3 weeks after the second course of the study regimen
description: • Percentage of study subjects who demonstrate a complete response, defined as no episodes of emesis and no use of rescue medications during the investigational antiemetic cycles.
measure: Efficacy of aprepitant (Emend®) measured through episodes of emesis and use of rescue medication.
time frame: Up to 11 weeks, or until 3 weeks after the second course of the study regimen
description: The total episodes of emesis within 7 days of the first chemotherapy administration of each cycle.
The total number of administrations of rescue medications given for breakthrough nausea or vomiting.
measure: Efficacy of aprepitant (Emend®) measured through impact of chemotherapy induced nausea and vomiting on daily life
time frame: Up to 11 weeks, or until 3 weeks after the second course of the study regimen
description: • A modified, 5-day recall version of the Functional Living Index-Emesis (FLIE) questionnaire
measure: Efficacy of aprepitant (Emend®) measured through a pictorial nausea scale
time frame: Up to 11 weeks, or until 3 weeks after the second course of the study regimen
description: • A modified version of the Baxter Animated Retching Faces (BARF) scale, administered daily.
measure: Safety of aprepitant (Emend®)
time frame: Up to 11 weeks, or until 3 weeks after the second course of the study regimen
description: Occurrence of adverse events as per the NCI Common Terminology Criteria for Adverse Events (CTCAE) v.4.0. These will be reported spontaneously or on inquiry by the investigator/study nurse, and continuously monitored throughout the trial.
Weekly complete blood count (CBC) for 3 weeks after each investigational antiemetic cycle.
Weekly complete metabolic profile (CMP) for 3 weeks after each investigational antiemetic cycle.
arm group label: Ondansetron, dexamethasone, aprepitant
arm group type: Experimental
description: Arm A: Ondansetron 0.15 mg/kg (max 16 mg) IV or PO every 8 hours for at least 3 days, but no longer than 5 days; dexamethasone 0.2mg/kg (max 10 mg) IV or PO daily for at least 3 days, but no longer than 5 days; and aprepitant 3 mg/kg (max 125 mg) PO on day 1, and aprepitant 2 mg/kg (max 80 mg) PO on days 2 and 3 during the first investigational antiemetic cycle. During the next investigational antiemetic cycle, members of this arm will be crossed-over into the placebo arm, where the aprepitant will be replaced by placebo and the dexamethasone dose will be increased to 0.4 mg/kg (max 20 mg) daily.
arm group label: Ondansetron, Dexamethasone, placebo
arm group type: Placebo Comparator
description: Arm B: Ondansetron 0.15 mg/kg (max 16 mg) IV or PO every 8 hours for at least 3 days, but no more than 5 days; dexamethasone 0.4 mg/kg (max 20 mg) IV or PO daily for at least 3 days, but no more than 5 days; and a PO placebo for 3 days during the first investigational antiemetic cycle. During the second cycle, members this group will be crossed-over to the experimental arm, where the placebo will be replaced by aprepitant and the dexamethasone will be decreased by 50%.
intervention type: Drug
intervention name: Ondansetron, dexamethasone, aprepitant
description: Ondansetron 0.15 mg/kg (max 16 mg) IV or PO every 8 hours for at least 3 days, but no longer than 5 days; dexamethasone 0.2mg/kg (max 10 mg) IV or PO daily for at least 3 days, but no longer than 5 days; and aprepitant 3 mg/kg (max 125 mg) PO on day 1, and aprepitant 2 mg/kg (max 80 mg) PO on days 2 and 3 during the first investigational antiemetic cycle. During the next investigational antiemetic cycle, members of this arm will be crossed-over into the placebo arm, where the aprepitant will be replaced by placebo and the dexamethasone dose will be increased to 0.4 mg/kg (max 20 mg) daily.
arm group label: Ondansetron, dexamethasone, aprepitant
other name: Aprepitant = Emend
other name: ARM A
intervention type: Drug
intervention name: Ondansetron, Dexamethasone, placebo
description: Ondansetron 0.15 mg/kg (max 16 mg) IV or PO every 8 hours for at least 3 days, but no more than 5 days; dexamethasone 0.4 mg/kg (max 20 mg) IV or PO daily for at least 3 days, but no more than 5 days; and a PO placebo for 3 days during the first investigational antiemetic cycle. During the second cycle, members this group will be crossed-over to the experimental arm, where the placebo will be replaced by aprepitant and the dexamethasone will be decreased by 50%.
arm group label: Ondansetron, Dexamethasone, placebo
other name: ARM B
criteria: 
gender: All
minimum age: 6 Months
maximum age: 20 Years
healthy volunteers: No
last name: Rene McNall-Knapp, MD
role: Principal Investigator
affiliation: University of Oklahoma
facility: 
country: United States
responsible party type: Sponsor
mesh term: Nausea
mesh term: Vomiting
mesh term: Dexamethasone
mesh term: Dexamethasone acetate
mesh term: Ondansetron
mesh term: Aprepitant
mesh term: Fosaprepitant
mesh term: BB 1101
sharing ipd: No
submitted: March 24, 2021
returned: April 20, 2021
submitted: May 3, 2022
returned: May 26, 2022