<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/NCT03033303</url>
  </required_header>
  <id_info>
    <org_study_id>16-1643</org_study_id>
    <nct_id>NCT03033303</nct_id>
  </id_info>
  <brief_title>A Study of the Effect of Hu3F8/GM-CSF Immunotherapy Plus Isotretinoin in Patients in First Remission of High-Risk Neuroblastoma</brief_title>
  <official_title>Hu3F8/GM-CSF Immunotherapy Plus Isotretinoin for Consolidation of First Remission of Patients With High-Risk Neuroblastoma: A Phase II Study</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Memorial Sloan Kettering Cancer Center</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Y-mAbs Therapeutics</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
  </sponsors>
  <source>Memorial Sloan Kettering Cancer Center</source>
  <oversight_info>
    <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 test see the combined effects of the study drug called&#xD;
      Humanized 3F8 (Hu3F8) when used with granulocyte-macrophage colony stimulating factor&#xD;
      (GM-CSF). Hu3F8 plus GM-CSF could prevent your neuroblastoma from growing, but it could also&#xD;
      cause side effects.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Active, not recruiting</overall_status>
  <start_date type="Actual">January 23, 2017</start_date>
  <completion_date type="Anticipated">June 2024</completion_date>
  <primary_completion_date type="Anticipated">June 2024</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <intervention_model_description>In this phase II single arm trial, patients with HR-NB in first CR or VGPR undergo consolidation by using hu3F8/GM-CSF.</intervention_model_description>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>relapse-free survival (RFS)</measure>
    <time_frame>2 years</time_frame>
    <description>Patients are considered a response failure under this protocol if progressive disease is evident before two years. Deaths from toxicity attributable to protocol treatment will be counted as events. Patients who withdraw or are lost to followup before two years will be considered as progressions.</description>
  </primary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">59</enrollment>
  <condition>Neuroblastoma</condition>
  <arm_group>
    <arm_group_label>Hu3F8/GM-CSF Plus Isotretinoin</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>In this phase II single arm trial, patients with HR-NB in first CR or VGPR undergo consolidation by using hu3F8/GM-CSF x5 cycles and isotretinoin x6 cycles. Isotretinoin starts after cycle 2 of hu3F8/GM-CSF.</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>Hu3F8</intervention_name>
    <description>Day 1: hu3F8 infused iv over ~30 to 90 minutes. Day 3: hu3F8 infused iv over ~30 to 90 minutes. Day 5: hu3F8 infused iv over ~30 to 90 minutes.</description>
    <arm_group_label>Hu3F8/GM-CSF Plus Isotretinoin</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>GM-CSF</intervention_name>
    <description>Days -4 to 0: GM-CSF 250 mcg/m2/day, subcutaneously. Days 1 to 5: GM-CSF 500 mcg/m2/day, subcutaneously.</description>
    <arm_group_label>Hu3F8/GM-CSF Plus Isotretinoin</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Isotretinoin</intervention_name>
    <description>Isotretinoin is administered at 160 mg/m2/d, divided into two doses, x14 days.</description>
    <arm_group_label>Hu3F8/GM-CSF Plus Isotretinoin</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#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 high-risk NB (MYCN-amplified stage 2/3/4/4S of any age and&#xD;
             MYCN-nonamplified stage 4 in patients greater than 18 months of age).&#xD;
&#xD;
          -  Patients must be in first CR/VGPR&#xD;
&#xD;
          -  Patients must have a negative human anti-hu3F8 antibody (HAHA) titer&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Existing major organ dysfunction, i.e., renal., cardiac, hepatic, neurologic,&#xD;
             pulmonary, or gastrointestinal toxicity ≥ grade 3, with the exception of hearing loss&#xD;
             and hematologic toxicity.&#xD;
&#xD;
          -  Active life-threatening infection.&#xD;
&#xD;
          -  Inability to comply with protocol requirements&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>N/A</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Brian H. 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>July 2023</verification_date>
  <study_first_submitted>January 24, 2017</study_first_submitted>
  <study_first_submitted_qc>January 24, 2017</study_first_submitted_qc>
  <study_first_posted type="Estimate">January 26, 2017</study_first_posted>
  <last_update_submitted>July 3, 2023</last_update_submitted>
  <last_update_submitted_qc>July 3, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">July 5, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Hu3F8</keyword>
  <keyword>GM-CSF</keyword>
  <keyword>Isotretinoin</keyword>
  <keyword>16-1643</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Neuroblastoma</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Isotretinoin</mesh_term>
  </intervention_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/NCT03033303
org study id: 16-1643
nct id: NCT03033303
lead sponsor: 
collaborator: 
is fda regulated drug: Yes
is fda regulated device: No
The purpose of this study is to test see the combined effects of the study drug called
      Humanized 3F8 (Hu3F8) when used with granulocyte-macrophage colony stimulating factor
      (GM-CSF). Hu3F8 plus GM-CSF could prevent your neuroblastoma from growing, but it could also
      cause side effects.
allocation: N/A
intervention model: Single Group Assignment
intervention model description: In this phase II single arm trial, patients with HR-NB in first CR or VGPR undergo consolidation by using hu3F8/GM-CSF.
primary purpose: Treatment
masking: None (Open Label)
measure: relapse-free survival (RFS)
time frame: 2 years
description: Patients are considered a response failure under this protocol if progressive disease is evident before two years. Deaths from toxicity attributable to protocol treatment will be counted as events. Patients who withdraw or are lost to followup before two years will be considered as progressions.
arm group label: Hu3F8/GM-CSF Plus Isotretinoin
arm group type: Experimental
description: In this phase II single arm trial, patients with HR-NB in first CR or VGPR undergo consolidation by using hu3F8/GM-CSF x5 cycles and isotretinoin x6 cycles. Isotretinoin starts after cycle 2 of hu3F8/GM-CSF.
intervention type: Biological
intervention name: Hu3F8
description: Day 1: hu3F8 infused iv over ~30 to 90 minutes. Day 3: hu3F8 infused iv over ~30 to 90 minutes. Day 5: hu3F8 infused iv over ~30 to 90 minutes.
arm group label: Hu3F8/GM-CSF Plus Isotretinoin
intervention type: Drug
intervention name: GM-CSF
description: Days -4 to 0: GM-CSF 250 mcg/m2/day, subcutaneously. Days 1 to 5: GM-CSF 500 mcg/m2/day, subcutaneously.
arm group label: Hu3F8/GM-CSF Plus Isotretinoin
intervention type: Drug
intervention name: Isotretinoin
description: Isotretinoin is administered at 160 mg/m2/d, divided into two doses, x14 days.
arm group label: Hu3F8/GM-CSF Plus Isotretinoin
criteria: 
gender: All
minimum age: N/A
maximum age: N/A
healthy volunteers: No
last name: Brian H. 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
mesh term: Isotretinoin