<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/NCT04501757</url>
  </required_header>
  <id_info>
    <org_study_id>20-227</org_study_id>
    <nct_id>NCT04501757</nct_id>
  </id_info>
  <brief_title>Naxitamab and GM-CSF in People With Neuroblastoma</brief_title>
  <official_title>Expanded Access Use of Naxitamab/GM-CSF Immunotherapy for Consolidation of Complete Remission or Relapsed/Refractory High-Risk Neuroblastoma</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Memorial Sloan Kettering Cancer Center</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Memorial Sloan Kettering Cancer Center</source>
  <brief_summary>
    <textblock>
      This is an Expanded Access Program (EAP) that will give the participants access to the drug&#xD;
      naxitamab before it is approved by the FDA. Naxitamab will be combined with&#xD;
      granulocyte-macrophage colony stimulating factor (GM-CSF). Participants in this study will&#xD;
      have high-risk neuroblastoma that either went away completely after treatment (complete&#xD;
      remission) or has come back (relapsed/refractory).&#xD;
&#xD;
      Researchers think the combination of naxitamab and GM-CSF will be effective because naxitamab&#xD;
      and GM-CSF strengthen the immune system's response to cancer cells in different ways.&#xD;
      Naxitamab is an antibody, like the proteins made by the immune system to protect the body&#xD;
      from harm. Naxitamab helps the cells of the immune system to find and attack cancer cells.&#xD;
      GM-CSF is a protein that strengthens the immune system by increasing the number of immune&#xD;
      cells called granulocytes. Granulocytes are white blood cells that fight off cancer cells.&#xD;
      The combination of naxitamab and GM-CSF is a type of immunotherapy.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>No longer available</overall_status>
  <study_type>Expanded Access</study_type>
  <has_expanded_access>No</has_expanded_access>
  <expanded_access_info>
    <expanded_access_type_treatment>Yes</expanded_access_type_treatment>
  </expanded_access_info>
  <condition>High-Risk Neuroblastoma</condition>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>Naxitamab/GM-CSF</intervention_name>
    <description>naxitamab/GM-CSF for 5 Cycles</description>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Patients who are ineligible to participate in study NCT03363373 (IRB# 18-375), will be&#xD;
             considered for treatment under this expanded access protocol.&#xD;
&#xD;
          -  Diagnosis of NB as defined by a) histopathology (confirmed by the MSK Department of&#xD;
             Pathology), or b) BM metastases or MIBG-avid lesion(s) plus high urine catecholamine&#xD;
             levels&#xD;
&#xD;
          -  Patients must have HR-NB (MYCN-amplified stage 2/3/4/4S of any age and&#xD;
             MYCNnonamplified stage 4 in patients greater than 18 months of age).&#xD;
&#xD;
          -  Patients must be in first or greater CR or have refractory or relapsed disease&#xD;
&#xD;
          -  If previously treated with any anti-GD2 mAb, human anti-hu3F8 antibody (HAHA) titer&#xD;
             must be negative.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Active life-threatening infection&#xD;
&#xD;
          -  Pregnant women or women who are breastfeeding&#xD;
&#xD;
          -  HAHA titer positive&#xD;
&#xD;
          -  History of anaphylaxis CTCAE grade 4 to naxitamab&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Months</minimum_age>
    <maximum_age>N/A</maximum_age>
  </eligibility>
  <overall_official>
    <last_name>Brian Kushner, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Memorial Sloan Kettering Cancer Center</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Memorial Sloan Kettering Cancer Center</name>
      <address>
        <city>New York</city>
        <state>New York</state>
        <zip>10065</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <link>
    <url>http://www.mskcc.org/mskcc/html/44.cfm</url>
    <description>Memorial Sloan Kettering Cancer Center</description>
  </link>
  <verification_date>December 2022</verification_date>
  <study_first_submitted>July 30, 2020</study_first_submitted>
  <study_first_submitted_qc>August 4, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">August 6, 2020</study_first_posted>
  <last_update_submitted>December 6, 2022</last_update_submitted>
  <last_update_submitted_qc>December 6, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">December 8, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Remission High-Risk Neuroblastoma</keyword>
  <keyword>Relapsed/Refractory High-Risk Neuroblastoma</keyword>
  <keyword>Naxitamab</keyword>
  <keyword>GM-CSF</keyword>
  <keyword>20-227</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Neuroblastoma</mesh_term>
  </condition_browse>
  <!-- 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/NCT04501757
org study id: 20-227
nct id: NCT04501757
lead sponsor: 
This is an Expanded Access Program (EAP) that will give the participants access to the drug
      naxitamab before it is approved by the FDA. Naxitamab will be combined with
      granulocyte-macrophage colony stimulating factor (GM-CSF). Participants in this study will
      have high-risk neuroblastoma that either went away completely after treatment (complete
      remission) or has come back (relapsed/refractory).

      Researchers think the combination of naxitamab and GM-CSF will be effective because naxitamab
      and GM-CSF strengthen the immune system's response to cancer cells in different ways.
      Naxitamab is an antibody, like the proteins made by the immune system to protect the body
      from harm. Naxitamab helps the cells of the immune system to find and attack cancer cells.
      GM-CSF is a protein that strengthens the immune system by increasing the number of immune
      cells called granulocytes. Granulocytes are white blood cells that fight off cancer cells.
      The combination of naxitamab and GM-CSF is a type of immunotherapy.
expanded access type treatment: Yes
intervention type: Biological
intervention name: Naxitamab/GM-CSF
description: naxitamab/GM-CSF for 5 Cycles
criteria: 
gender: All
minimum age: 18 Months
maximum age: N/A
last name: Brian Kushner, MD
role: Principal Investigator
affiliation: Memorial Sloan Kettering Cancer Center
facility: 
country: United States
url: http://www.mskcc.org/mskcc/html/44.cfm
description: Memorial Sloan Kettering Cancer Center
responsible party type: Sponsor
mesh term: Neuroblastoma