<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/NCT03773393</url>
  </required_header>
  <id_info>
    <org_study_id>CK0801-101-1</org_study_id>
    <nct_id>NCT03773393</nct_id>
  </id_info>
  <brief_title>A Clinical Trial of CK0801 (a New Drug) in Patients With Bone Marrow Failure Syndrome (BMF)</brief_title>
  <official_title>Phase I Trial to Evaluate the Safety and Feasibility of CK0801 in Treatment of Bone Marrow Failure Syndrome</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Cellenkos, Inc.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Cellenkos, Inc.</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 goal of this clinical research study is to determine whether it is safe and practical to&#xD;
      give CK0801 (a Cord blood-derived T-regulatory cell product) to patients with bone marrow&#xD;
      failure syndrome. Researchers want to determine the highest possible dose that is safe to be&#xD;
      given. Researchers also want to learn if CK0801 may improve the symptoms of bone marrow&#xD;
      failure syndrome.&#xD;
&#xD;
      Patients enrolled in this study will all have been diagnosed with treatment refractory bone&#xD;
      marrow failure syndrome (which includes aplastic anemia, myelodysplastic syndrome, or&#xD;
      myelofibrosis). Participants eligible to participate in this study are unable or unwilling to&#xD;
      be treated with standard therapy or have failed standard therapy.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Primary Objective:&#xD;
&#xD;
      To determine dose-limiting toxicity of CK0801 as defined as any of the events each start at&#xD;
      the time of CK00801 infusion&#xD;
&#xD;
        1. Severe (grade 3 or 4) infusion toxicity within 24 hours (NCI-CTCAE V4.0)&#xD;
&#xD;
        2. Regimen related death within 30 days&#xD;
&#xD;
        3. Severe (grade 3 or 4) Cytokine Release Syndrome within 30 days&#xD;
&#xD;
      Secondary Objective:&#xD;
&#xD;
        1. Preliminary assessment of disease-specific response&#xD;
&#xD;
        2. Duration of disease-specific response&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Active, not recruiting</overall_status>
  <start_date type="Actual">May 30, 2019</start_date>
  <completion_date type="Anticipated">January 30, 2024</completion_date>
  <primary_completion_date type="Anticipated">January 25, 2024</primary_completion_date>
  <phase>Phase 1</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>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Number of Participants with Severe Infusion Toxicity as Assessed by CTCAE v4.0</measure>
    <time_frame>24 hours post-intervention</time_frame>
    <description>Number of Participants with Severe (Grade 3 or 4) Toxicity</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Number of Participants with Regimen Related Death</measure>
    <time_frame>30 days post-intervention</time_frame>
    <description>Number of Participants with Regimen Related Death</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Number of Participants with Severe Cytokine Release Syndrome (CRS)</measure>
    <time_frame>30 days post-intervention</time_frame>
    <description>Number of Participants with Severe (Grade 3 or 4) CRS</description>
  </primary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Anticipated">18</enrollment>
  <condition>Bone Marrow Disease</condition>
  <arm_group>
    <arm_group_label>CK0801</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>All subjects will receive adoptive therapy with an infusion of unrelated cord blood-derived regulatory T cells: CK0801. Subjects will receive one intravenous dose of CK0801 (Treg cells) on study Day 0.</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>CK0801</intervention_name>
    <description>CK0801 (a Cord blood-derived T-regulatory cell product)</description>
    <arm_group_label>CK0801</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Subjects who fulfill the diagnostic criteria of bone marrow failure syndrome&#xD;
             including: aplastic anemia, myelodysplastic syndrome, or myelofibrosis.&#xD;
&#xD;
          2. HLA matched (≥ 3/6) cord blood unit available for CK0801 generation.&#xD;
&#xD;
          3. Subjects age ≥ 18 years.&#xD;
&#xD;
          4. Bilirubin ≤ 2 x ULN and SGPT (ALT) ≤ 2 x ULN (unless Gilbert's syndrome is&#xD;
             documented).&#xD;
&#xD;
          5. Calculated creatinine clearance of &gt; 50mL/min using the Cockcroft-Gault equation.&#xD;
&#xD;
          6. Zubrod performance status ≤ 2.&#xD;
&#xD;
          7. Female subjects of child bearing potential (FPCP) must have a negative urine or serum&#xD;
             pregnancy test. NOTE: FPCP is defined as premenopausal and not surgically sterilized.&#xD;
             FPCP must agree to use maximally effective birth control or to abstain from&#xD;
             heterosexual activity throughout the study. Effective contraceptive methods include&#xD;
             intra-uterine device, oral and/or injectable hormonal; contraception, or 2 adequate&#xD;
             barrier methods (e.g., cervical cap with spermicide, diaphragm with spermicide).&#xD;
&#xD;
          8. Subject has agreed to abide by all protocol required procedures including&#xD;
             study-related assessments, visits and long term follow up.&#xD;
&#xD;
          9. Subject is willing and able to provide written informed consent.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Subject has received an investigational agent within 4 weeks prior to CK0801 infusion.&#xD;
&#xD;
          2. Subject has received radiation or chemotherapy within 21 days prior to CK0801&#xD;
             infusion.&#xD;
&#xD;
          3. Subject has received prior cord blood-derived T-regulatory therapy.&#xD;
&#xD;
          4. HIV seropositivity.&#xD;
&#xD;
          5. Subject has uncontrolled infection, not responding to appropriate antimicrobial agents&#xD;
             after seven days of therapy. The Protocol PI is the final arbiter of eligibility.&#xD;
&#xD;
          6. Subjects with uncontrolled inter-current illness that in the opinion of the&#xD;
             investigator would place the patient at greater risk of severe toxicity and/or impair&#xD;
             the activity of CK0801&#xD;
&#xD;
          7. Subjects is pregnant or breastfeeding.&#xD;
&#xD;
          8. Bone marrow failure caused by stem cell transplantation.&#xD;
&#xD;
          9. Subjects who are unable to provide consent or who, in the opinion of the Investigator&#xD;
             will be unlikely to fully comply with protocol requirements.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Tapan M Kadia, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>The University of Texas MD Anderson Cancer Center</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Sarcoma Oncology Research Center, Cancer Center of Southern California</name>
      <address>
        <city>Santa Monica</city>
        <state>California</state>
        <zip>90403</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The University of Texas MD Anderson Cancer Center</name>
      <address>
        <city>Houston</city>
        <state>Texas</state>
        <zip>77030</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>January 2023</verification_date>
  <study_first_submitted>October 23, 2018</study_first_submitted>
  <study_first_submitted_qc>December 10, 2018</study_first_submitted_qc>
  <study_first_posted type="Actual">December 12, 2018</study_first_posted>
  <last_update_submitted>August 28, 2023</last_update_submitted>
  <last_update_submitted_qc>August 28, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">August 29, 2023</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>Bone Marrow Failure Disorders</mesh_term>
    <mesh_term>Pancytopenia</mesh_term>
    <mesh_term>Bone Marrow Diseases</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <!-- 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/NCT03773393
org study id: CK0801-101-1
nct id: NCT03773393
lead sponsor: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
The goal of this clinical research study is to determine whether it is safe and practical to
      give CK0801 (a Cord blood-derived T-regulatory cell product) to patients with bone marrow
      failure syndrome. Researchers want to determine the highest possible dose that is safe to be
      given. Researchers also want to learn if CK0801 may improve the symptoms of bone marrow
      failure syndrome.

      Patients enrolled in this study will all have been diagnosed with treatment refractory bone
      marrow failure syndrome (which includes aplastic anemia, myelodysplastic syndrome, or
      myelofibrosis). Participants eligible to participate in this study are unable or unwilling to
      be treated with standard therapy or have failed standard therapy.
Primary Objective:

      To determine dose-limiting toxicity of CK0801 as defined as any of the events each start at
      the time of CK00801 infusion

        1. Severe (grade 3 or 4) infusion toxicity within 24 hours (NCI-CTCAE V4.0)

        2. Regimen related death within 30 days

        3. Severe (grade 3 or 4) Cytokine Release Syndrome within 30 days

      Secondary Objective:

        1. Preliminary assessment of disease-specific response

        2. Duration of disease-specific response
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Number of Participants with Severe Infusion Toxicity as Assessed by CTCAE v4.0
time frame: 24 hours post-intervention
description: Number of Participants with Severe (Grade 3 or 4) Toxicity
measure: Number of Participants with Regimen Related Death
time frame: 30 days post-intervention
description: Number of Participants with Regimen Related Death
measure: Number of Participants with Severe Cytokine Release Syndrome (CRS)
time frame: 30 days post-intervention
description: Number of Participants with Severe (Grade 3 or 4) CRS
arm group label: CK0801
arm group type: Experimental
description: All subjects will receive adoptive therapy with an infusion of unrelated cord blood-derived regulatory T cells: CK0801. Subjects will receive one intravenous dose of CK0801 (Treg cells) on study Day 0.
intervention type: Biological
intervention name: CK0801
description: CK0801 (a Cord blood-derived T-regulatory cell product)
arm group label: CK0801
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Tapan M Kadia, MD
role: Principal Investigator
affiliation: The University of Texas MD Anderson Cancer Center
facility: 
facility: 
country: United States
responsible party type: Sponsor
mesh term: Bone Marrow Failure Disorders
mesh term: Pancytopenia
mesh term: Bone Marrow Diseases
sharing ipd: No