<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/NCT03244306</url>
  </required_header>
  <id_info>
    <org_study_id>PLAT-04</org_study_id>
    <nct_id>NCT03244306</nct_id>
  </id_info>
  <brief_title>A Phase 1 Study of CD22-CAR TCell Immunotherapy for CD22+ Leukemia and Lymphoma</brief_title>
  <acronym>PLAT-04</acronym>
  <official_title>Pediatric and Young Adult Leukemia Adoptive Therapy (PLAT)-04: A Phase 1 Feasibility and Safety Study of CD22-CAR T Cell Immunotherapy for CD22+ Leukemia and Lymphoma</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Seattle Children's Hospital</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Seattle Children's Hospital</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>
      Patients with relapsed or refractory leukemia often develop resistance to chemotherapy and&#xD;
      some patients who relapse following CD19 directed therapy relapse with CD19 negative&#xD;
      leukemia. For this reason, the investigators are attempting to use T-cells obtained directly&#xD;
      from the patient, which can be genetically modified to express a chimeric antigen receptor&#xD;
      (CAR) to CD22, a different protein from CD19, expressed on the surface of the leukemic cell&#xD;
      in patients with CD22+ leukemia. The CAR enables the T-cell to recognize and kill the&#xD;
      leukemic cell through the recognition of CD22, a protein expressed on the surface of the&#xD;
      leukemic cell in patients with CD22+ leukemia. This is a Phase 1 study designed to determine&#xD;
      the safety and feasibility of the CAR+ T - cells and the feasibility of making enough to&#xD;
      treat patients with CD22+ leukemia.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Active, not recruiting</overall_status>
  <start_date type="Actual">July 27, 2017</start_date>
  <completion_date type="Anticipated">July 2035</completion_date>
  <primary_completion_date type="Actual">November 14, 2018</primary_completion_date>
  <phase>Phase 1</phase>
  <study_type>Interventional</study_type>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>The adverse events associated with one or multiple CAR T-cell product infusions will be assessed</measure>
    <time_frame>30 days</time_frame>
    <description>The type, frequency, severity, and duration of adverse events will be summarized</description>
  </primary_outcome>
  <primary_outcome>
    <measure>The number of successfully and unsuccessfully manufactured and infused CAR T-cell products will be assessed</measure>
    <time_frame>28 days</time_frame>
    <description>Proportion of products successfully manufactured and infused</description>
  </primary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">4</enrollment>
  <condition>Leukemia</condition>
  <arm_group>
    <arm_group_label>Autologous CD22-specific CAR T-cells expressing EGFRt</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>Patient-derived CD22-specific CAR T-cells also expressing an EGFRt</intervention_name>
    <description>Patient-derived CD22-specific chimeric antigen receptor T-cells expressing an EGFRt</description>
    <arm_group_label>Autologous CD22-specific CAR T-cells expressing EGFRt</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  First 3 subjects: male and female subjects age ≥ 18 years and &lt; 27 years&#xD;
&#xD;
          -  Subsequent subjects: 12 months of age and &lt;27 years of age at the time of study&#xD;
             enrollment&#xD;
&#xD;
          -  Disease status (one of the following):&#xD;
&#xD;
               1. If post-allogeneic hematopoetic cell transplant (HCT): confirmed CD22+ leukemia&#xD;
                  recurrence, defined as ≥0.01% disease&#xD;
&#xD;
               2. If Relapse/Refractory status with no prior history of allogeneic HCT, one of:&#xD;
&#xD;
                    -  2nd or grater marrow relapse, with or without extramedullary disease&#xD;
&#xD;
                    -  1st marrow relapse at end of 1st month of re-induction with marrow having&#xD;
                       ≥0.01% blasts by morphology and/or MPF&#xD;
&#xD;
                    -  Primary Refractory, defined as &gt;5% blasts by multi-parameter flow after ≥2&#xD;
                       separate induction regimens&#xD;
&#xD;
                    -  Subject has indication for HCT but is ineligible, inclusive of persistent&#xD;
                       minimal residual disease&#xD;
&#xD;
               3. CD22+ Lymphoma refractory or relapsed with no known curative therapies available&#xD;
&#xD;
          -  Asymptomatic from CNS involvement, if present, and have a reasonable expectation that&#xD;
             disease burden can be controlled in the interval between enrollment and T-cell&#xD;
             infusion. Subjects with significant neurologic deterioration will not be eligible for&#xD;
             T-cell infusion until stabilized.&#xD;
&#xD;
          -  Free from active GVHD and off immunosuppressive GVHD therapy for 4 weeks.&#xD;
&#xD;
          -  Lansky or Karnofsky performance score of ≥50&#xD;
&#xD;
          -  Life expectancy of &gt;8 weeks&#xD;
&#xD;
          -  Recovered from acute toxic effects of all prior chemotherapy, immunotherapy, and&#xD;
             radiotherapy&#xD;
&#xD;
          -  ≥7 days post last chemotherapy administration (excluding intrathecal or maintenance&#xD;
             chemotherapy)&#xD;
&#xD;
          -  ≥7 days post last systemic corticosteroid administration&#xD;
&#xD;
          -  No prior virotherapy&#xD;
&#xD;
          -  Adequate organ function&#xD;
&#xD;
          -  Adequate laboratory values&#xD;
&#xD;
          -  Patients of childbearing/fathering potential must agree to use highly effective&#xD;
             contraception&#xD;
&#xD;
          -  Signed a written consent&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Presence of active clinically significant CNS dysfunction&#xD;
&#xD;
          -  Pregnant or breastfeeding&#xD;
&#xD;
          -  Unable to tolerate apheresis procedure, including placement of temporary apheresis&#xD;
             line if required&#xD;
&#xD;
          -  Presence of active malignancy other than CD22+ leukemia or lymphoma&#xD;
&#xD;
          -  Presence of active severe infection&#xD;
&#xD;
          -  Presence of any concurrent medical condition that would prevent the patient from&#xD;
             undergoing protocol-based therapy&#xD;
&#xD;
          -  Presence of primary immunodeficiency/bone marrow failure syndrome&#xD;
&#xD;
          -  Unwilling to participate in 15-year follow-up period that is required if CAR T cell&#xD;
             therapy is administered&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>1 Year</minimum_age>
    <maximum_age>26 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Corinne Summers, MD</last_name>
    <role>Study Chair</role>
    <affiliation>Seattle Children's Hospital</affiliation>
  </overall_official>
  <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_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>June 2023</verification_date>
  <study_first_submitted>August 3, 2017</study_first_submitted>
  <study_first_submitted_qc>August 8, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">August 9, 2017</study_first_posted>
  <last_update_submitted>June 27, 2023</last_update_submitted>
  <last_update_submitted_qc>June 27, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">June 29, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Seattle Children's Hospital</investigator_affiliation>
    <investigator_full_name>Colleen Annesley</investigator_full_name>
    <investigator_title>Medical Director, Seattle Children's Therapeutics</investigator_title>
  </responsible_party>
  <keyword>leukemia</keyword>
  <keyword>CD 22</keyword>
  <keyword>CAR T cell</keyword>
  <keyword>pediatric</keyword>
  <keyword>young adult</keyword>
  <keyword>chimeric antigen receptor</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Leukemia</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/NCT03244306
org study id: PLAT-04
nct id: NCT03244306
lead sponsor: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
Patients with relapsed or refractory leukemia often develop resistance to chemotherapy and
      some patients who relapse following CD19 directed therapy relapse with CD19 negative
      leukemia. For this reason, the investigators are attempting to use T-cells obtained directly
      from the patient, which can be genetically modified to express a chimeric antigen receptor
      (CAR) to CD22, a different protein from CD19, expressed on the surface of the leukemic cell
      in patients with CD22+ leukemia. The CAR enables the T-cell to recognize and kill the
      leukemic cell through the recognition of CD22, a protein expressed on the surface of the
      leukemic cell in patients with CD22+ leukemia. This is a Phase 1 study designed to determine
      the safety and feasibility of the CAR+ T - cells and the feasibility of making enough to
      treat patients with CD22+ leukemia.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: The adverse events associated with one or multiple CAR T-cell product infusions will be assessed
time frame: 30 days
description: The type, frequency, severity, and duration of adverse events will be summarized
measure: The number of successfully and unsuccessfully manufactured and infused CAR T-cell products will be assessed
time frame: 28 days
description: Proportion of products successfully manufactured and infused
arm group label: Autologous CD22-specific CAR T-cells expressing EGFRt
arm group type: Experimental
intervention type: Biological
intervention name: Patient-derived CD22-specific CAR T-cells also expressing an EGFRt
description: Patient-derived CD22-specific chimeric antigen receptor T-cells expressing an EGFRt
arm group label: Autologous CD22-specific CAR T-cells expressing EGFRt
criteria: 
gender: All
minimum age: 1 Year
maximum age: 26 Years
healthy volunteers: No
last name: Corinne Summers, MD
role: Study Chair
affiliation: Seattle Children's Hospital
facility: 
country: United States
responsible party type: Principal Investigator
investigator affiliation: Seattle Children's Hospital
investigator full name: Colleen Annesley
investigator title: Medical Director, Seattle Children's Therapeutics
mesh term: Leukemia