<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/NCT04438083</url>
  </required_header>
  <id_info>
    <org_study_id>CRSP-ONC-003</org_study_id>
    <nct_id>NCT04438083</nct_id>
  </id_info>
  <brief_title>A Safety and Efficacy Study Evaluating CTX130 in Subjects With Relapsed or Refractory Renal Cell Carcinoma (COBALT-RCC)</brief_title>
  <official_title>A Phase 1 Dose Escalation and Cohort Expansion Study of the Safety and Efficacy of Allogeneic CRISPR-Cas9-Engineered T Cells (CTX130) in Subjects With Advanced, Relapsed or Refractory Renal Cell Carcinoma With Clear Cell Differentiation</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>CRISPR Therapeutics AG</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>CRISPR Therapeutics</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>
      This is a single-arm, open-label, multicenter, Phase 1 study evaluating the safety and&#xD;
      efficacy of CTX130 in subjects with relapsed or refractory renal cell carcinoma.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      The study may enroll approximately 107subjects in total.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Active, not recruiting</overall_status>
  <start_date type="Actual">June 16, 2020</start_date>
  <completion_date type="Anticipated">April 2027</completion_date>
  <primary_completion_date type="Anticipated">February 2027</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>Sequential Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Part A (dose escalation): Incidence of adverse events</measure>
    <time_frame>From CTX130 infusion up to 28 days post-infusion</time_frame>
    <description>Adverse events defined as dose-limiting toxicities</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Part B (cohort expansion): Objective response rate</measure>
    <time_frame>From CTX130 infusion up to 60 months post-infusion]</time_frame>
    <description>Objective response rate per Response Evaluation Criteria In Solid Tumors (RECIST) v1.1</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Progression Free Survival</measure>
    <time_frame>From date of CTX130 infusion until date of disease progression or death due to any cause, assessed up to 60 months</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Overall Survival</measure>
    <time_frame>From date of CTX130 until date of death due to any cause, assessed up to 60 months</time_frame>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Anticipated">107</enrollment>
  <condition>Renal Cell Carcinoma</condition>
  <arm_group>
    <arm_group_label>CTX130</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Administered by IV infusion following lymphodepleting chemotherapy.</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>CTX130</intervention_name>
    <description>CTX130 CD70-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components.</description>
    <arm_group_label>CTX130</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Abbreviated Inclusion Criteria:&#xD;
&#xD;
          1. Age ≥18 years and body weight ≥42 kg.&#xD;
&#xD;
          2. Unresectable or metastatic RCC that has exploited standard of care treatment.&#xD;
&#xD;
          3. Karnofsky performance status (KPS) ≥80%.&#xD;
&#xD;
          4. Adequate renal, liver, cardiac, and pulmonary organ function.&#xD;
&#xD;
          5. Female subjects of childbearing potential and male subjects must agree to use&#xD;
             acceptable method(s) of contraception from enrollment through at least 12 months after&#xD;
             CTX130 infusion.&#xD;
&#xD;
        Abbreviated Exclusion Criteria:&#xD;
&#xD;
          1. Prior treatment with any anti-CD70 targeting agents.&#xD;
&#xD;
          2. Prior treatment with any CAR T cells or any other modified T or natural killer (NK)&#xD;
             cells.&#xD;
&#xD;
          3. History of certain central nervous system (CNS), cardiac or pulmonary conditions.&#xD;
&#xD;
          4. Active HIV, hepatitis B virus or hepatitis C virus infection.&#xD;
&#xD;
          5. Previous or concurrent malignancy, except treated with curative approach not requiring&#xD;
             systemic therapy and in remission for &gt;12 months, or any other localized malignancy&#xD;
             with low risk of developing into metastatic disease.&#xD;
&#xD;
          6. Primary immunodeficiency disorder or active autoimmune disease requiring steroids&#xD;
             and/or other immunosuppressive therapy.&#xD;
&#xD;
          7. Prior solid organ transplantation or bone marrow transplant.&#xD;
&#xD;
          8. Pregnant or breastfeeding females.&#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>Alissa Keegan, MD</last_name>
    <role>Study Director</role>
    <affiliation>CRISPR Therapeutics</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Research Site 2</name>
      <address>
        <city>Duarte</city>
        <state>California</state>
        <zip>91010</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Research Site 5</name>
      <address>
        <city>Hartford</city>
        <state>Connecticut</state>
        <zip>06520</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Research Site 4</name>
      <address>
        <city>Houston</city>
        <state>Texas</state>
        <zip>77030</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Research Site 3</name>
      <address>
        <city>Salt Lake City</city>
        <state>Utah</state>
        <zip>84112</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Research Site 1</name>
      <address>
        <city>Melbourne</city>
        <state>Victoria</state>
        <zip>3000</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Research Site 6</name>
      <address>
        <city>Toronto</city>
        <state>Ontario</state>
        <zip>M5G 2M9</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Research Site 7</name>
      <address>
        <city>Amsterdam</city>
        <state>North Holland</state>
        <zip>1066</zip>
        <country>Netherlands</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Australia</country>
    <country>Canada</country>
    <country>Netherlands</country>
    <country>United States</country>
  </location_countries>
  <verification_date>May 2023</verification_date>
  <study_first_submitted>June 16, 2020</study_first_submitted>
  <study_first_submitted_qc>June 16, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 18, 2020</study_first_posted>
  <last_update_submitted>May 9, 2023</last_update_submitted>
  <last_update_submitted_qc>May 9, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">May 11, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>CAR T</keyword>
  <keyword>Allogeneic</keyword>
  <keyword>Renal cell carcinoma</keyword>
  <keyword>Renal cell carcinoma with clear cell differentiation</keyword>
  <keyword>CRISPR-Cas9</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Carcinoma</mesh_term>
    <mesh_term>Carcinoma, Renal Cell</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/NCT04438083
org study id: CRSP-ONC-003
nct id: NCT04438083
lead sponsor: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
This is a single-arm, open-label, multicenter, Phase 1 study evaluating the safety and
      efficacy of CTX130 in subjects with relapsed or refractory renal cell carcinoma.
The study may enroll approximately 107subjects in total.
allocation: N/A
intervention model: Sequential Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Part A (dose escalation): Incidence of adverse events
time frame: From CTX130 infusion up to 28 days post-infusion
description: Adverse events defined as dose-limiting toxicities
measure: Part B (cohort expansion): Objective response rate
time frame: From CTX130 infusion up to 60 months post-infusion]
description: Objective response rate per Response Evaluation Criteria In Solid Tumors (RECIST) v1.1
measure: Progression Free Survival
time frame: From date of CTX130 infusion until date of disease progression or death due to any cause, assessed up to 60 months
measure: Overall Survival
time frame: From date of CTX130 until date of death due to any cause, assessed up to 60 months
arm group label: CTX130
arm group type: Experimental
description: Administered by IV infusion following lymphodepleting chemotherapy.
intervention type: Biological
intervention name: CTX130
description: CTX130 CD70-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components.
arm group label: CTX130
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Alissa Keegan, MD
role: Study Director
affiliation: CRISPR Therapeutics
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: Australia
country: Canada
country: Netherlands
country: United States
responsible party type: Sponsor
mesh term: Carcinoma
mesh term: Carcinoma, Renal Cell