<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/NCT04409080</url>
  </required_header>
  <id_info>
    <org_study_id>R7257-RAA-1947</org_study_id>
    <secondary_id>2020-002031-29</secondary_id>
    <nct_id>NCT04409080</nct_id>
  </id_info>
  <brief_title>REGN7257 in Adult Patients With Severe Aplastic Anemia That Is Refractory to or Relapsed on Immunosuppressive Therapy</brief_title>
  <official_title>A Phase 1/2 Study of REGN7257 (Anti-Interleukin 2 Receptor Subunit Gamma [IL2RG] Monoclonal Antibody) in Patients With Severe Aplastic Anemia That Is Refractory to or Relapsed on Immunosuppressive Therapy</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Regeneron Pharmaceuticals</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Regeneron Pharmaceuticals</source>
  <oversight_info>
    <has_dmc>No</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 primary objective of this study is to assess the safety and tolerability of REGN7257 in&#xD;
      patients with immunosuppressive therapy (IST)-refractory or IST-relapsed severe aplastic&#xD;
      anemia (SAA). An additional primary objective (for Part B only) is to evaluate the clinical&#xD;
      efficacy of REGN7257 in patients with IST-refractory or IST-relapsed SAA.&#xD;
&#xD;
      The secondary objectives of this study are to assess the following for REGN7257:&#xD;
&#xD;
        -  Clinical response over time&#xD;
&#xD;
        -  Maintenance of response&#xD;
&#xD;
        -  Impact on transfusion requirements&#xD;
&#xD;
        -  Effect on blood counts and cell populations&#xD;
&#xD;
        -  Pharmacokinetics (PK)&#xD;
&#xD;
        -  Immunogenicity&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">January 13, 2021</start_date>
  <completion_date type="Anticipated">January 2, 2026</completion_date>
  <primary_completion_date type="Anticipated">January 2, 2026</primary_completion_date>
  <phase>Phase 1/Phase 2</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>Incidence of adverse events (AEs)</measure>
    <time_frame>12 months post-treatment, approximately 52 weeks</time_frame>
    <description>Part A</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Incidence of serious adverse events (SAEs)</measure>
    <time_frame>12 months post-treatment, approximately 52 weeks</time_frame>
    <description>Parts A and B</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Incidence and severity of treatment-emergent adverse events (TEAEs) up</measure>
    <time_frame>12 months post-treatment, approximately 52 weeks</time_frame>
    <description>Parts A and B</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Overall response rate (ORR)</measure>
    <time_frame>6 months post-treatment, approximately 26 weeks</time_frame>
    <description>Part B</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>ORR</measure>
    <time_frame>3 months post-treatment, approximately 12 weeks</time_frame>
    <description>Parts A and B</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Complete response (CR)</measure>
    <time_frame>3 months post-treatment, approximately 12 weeks</time_frame>
    <description>Parts A and B</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Partial response (PR)</measure>
    <time_frame>3 months post-treatment, approximately 12 weeks</time_frame>
    <description>Parts A and B</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Time to best response</measure>
    <time_frame>Up to 12 months</time_frame>
    <description>Parts A and B</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Time to first response</measure>
    <time_frame>Up to 12 months</time_frame>
    <description>Parts A and B</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Proportion of patients who maintain any clinical response</measure>
    <time_frame>Up to 12 months</time_frame>
    <description>Parts A and B</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Platelet transfusions per month over time</measure>
    <time_frame>Up to 12 months</time_frame>
    <description>Parts A and B</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Red blood cell transfusions per month over time</measure>
    <time_frame>Up to 12 months</time_frame>
    <description>Parts A and B</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Changes in lymphocyte cell counts</measure>
    <time_frame>Up to 12 months</time_frame>
    <description>Parts A and B</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Changes in neutrophil cell counts</measure>
    <time_frame>Up to 12 months</time_frame>
    <description>Parts A and B</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Changes in hemoglobin cell counts</measure>
    <time_frame>Up to 12 months</time_frame>
    <description>Parts A and B</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Changes in reticulocyte cell counts</measure>
    <time_frame>Up to 12 months</time_frame>
    <description>Parts A and B</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Changes in platelet cell counts</measure>
    <time_frame>Up to 12 months</time_frame>
    <description>Parts A and B</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Changes in the whole blood immune cell subsets (T cells)</measure>
    <time_frame>Up to 12 months</time_frame>
    <description>Parts A and B</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Changes in the whole blood immune cell subsets (B cells)</measure>
    <time_frame>Up to 12 months</time_frame>
    <description>Parts A and B</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Changes in the whole blood immune cell subsets (NK cells)</measure>
    <time_frame>Up to 12 months</time_frame>
    <description>Parts A and B</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Drug concentrations in serum over time</measure>
    <time_frame>Up to 12 months</time_frame>
    <description>Parts A and B</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Incidence of treatment-emergent anti-drug antibody (ADA) over time</measure>
    <time_frame>Up to 12 months</time_frame>
    <description>Parts A and B</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Anticipated">57</enrollment>
  <condition>Severe Aplastic Anemia (SAA)</condition>
  <arm_group>
    <arm_group_label>Part A and Part B</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Part A: Single ascending dose Part B: Preferred dose</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>REGN7257</intervention_name>
    <description>Single dose will be administered by intravenous IV infusion</description>
    <arm_group_label>Part A and Part B</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Key Inclusion Criteria:&#xD;
&#xD;
          -  SAA that is IST-refractory or IST-relapsed, as defined in the protocol&#xD;
&#xD;
          -  Hematopoietic stem cell transplantation (HSCT) is not available or suitable as a&#xD;
             treatment option or has been refused by the patient&#xD;
&#xD;
          -  Adequate hepatic and renal function as defined in the protocol&#xD;
&#xD;
        Key Exclusion Criteria:&#xD;
&#xD;
          -  Diagnosis of Fanconi anemia or other congenital bone marrow failure syndrome as&#xD;
             defined in the protocol&#xD;
&#xD;
          -  Evidence of myelodysplastic syndrome as defined in the protocol&#xD;
&#xD;
          -  Paroxysmal nocturnal hemoglobinuria (PNH) with evidence of clinically significant&#xD;
             hemolysis (eg, treatment indicated) or history of PNH-associated thrombosis&#xD;
&#xD;
          -  Treatment with a T cell-depleting agent (eg, ATG or alemtuzumab) within 6 months prior&#xD;
             to dosing&#xD;
&#xD;
          -  Treatment with a calcineurin inhibitor (eg, cyclosporine) within 4 weeks prior to&#xD;
             dosing as defined in the protocol&#xD;
&#xD;
          -  Treatment with eltrombopag or investigational thrombopoietin receptor agonist,&#xD;
             Granulocyte Colony-Stimulating Factor (G-CSF), or an androgen (eg, danazol), within 2&#xD;
             weeks prior to dosing&#xD;
&#xD;
          -  HIV, hepatitis B or hepatitis C positive by serological testing at the screening visit&#xD;
             as defined in the protocol&#xD;
&#xD;
          -  Active tuberculosis, latent tuberculosis infection (LTBI) or history&#xD;
             incompletely-treated tuberculosis or LTBI&#xD;
&#xD;
          -  Active infection as defined in the protocol including COVID-19&#xD;
&#xD;
        Note: Other protocol-defined inclusion/ exclusion criteria apply&#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>Clinical Trial Management</last_name>
    <role>Study Director</role>
    <affiliation>Regeneron Pharmaceuticals</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Clinical Trials Administrator</last_name>
    <phone>844-734-6643</phone>
    <email>clinicaltrials@regeneron.com</email>
  </overall_contact>
  <location>
    <facility>
      <name>Cleveland Clinic Foundation</name>
      <address>
        <city>Cleveland</city>
        <state>Ohio</state>
        <zip>44195</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Sophia Balderman</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </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>
    <status>Recruiting</status>
    <investigator>
      <last_name>Tapan Kadia</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Hopital Saint-Louis - APHP</name>
      <address>
        <city>Paris</city>
        <state>Ile-de-France</state>
        <zip>75010</zip>
        <country>France</country>
      </address>
    </facility>
    <status>Recruiting</status>
  </location>
  <location>
    <facility>
      <name>Gachon University Gil Hospital</name>
      <address>
        <city>Incheon</city>
        <state>Gyeonggi</state>
        <zip>21565</zip>
        <country>Korea, Republic of</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Hawk Kim</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Seoul National University Hospital</name>
      <address>
        <city>Seoul</city>
        <state>Seoul Capital Area</state>
        <zip>03080</zip>
        <country>Korea, Republic of</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Yoon Sung Soo</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Samsung Medical Center</name>
      <address>
        <city>Seoul</city>
        <state>Seoul Capital Area</state>
        <zip>06351</zip>
        <country>Korea, Republic of</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Jun Ho Yang</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>The Catholic University of Korea, Seoul St. Marys Hospital</name>
      <address>
        <city>Seoul</city>
        <state>Seoul Capital Area</state>
        <zip>06591</zip>
        <country>Korea, Republic of</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Jong Wook Lee</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Ewha Womans University Medical Centre</name>
      <address>
        <city>Seoul</city>
        <state>Seoul Capital Area</state>
        <zip>07985</zip>
        <country>Korea, Republic of</country>
      </address>
    </facility>
    <status>Completed</status>
  </location>
  <location>
    <facility>
      <name>St James's University Hospital</name>
      <address>
        <city>Leeds</city>
        <state>West Yorkshire</state>
        <zip>LS97TF</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Morag Griffin</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>King's College Hospital, London</name>
      <address>
        <city>London</city>
        <zip>SE5 9RS</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <investigator>
      <last_name>Austin Kulasekararaj</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location_countries>
    <country>France</country>
    <country>Korea, Republic of</country>
    <country>United Kingdom</country>
    <country>United States</country>
  </location_countries>
  <verification_date>June 2023</verification_date>
  <study_first_submitted>May 26, 2020</study_first_submitted>
  <study_first_submitted_qc>May 26, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 1, 2020</study_first_posted>
  <last_update_submitted>July 20, 2023</last_update_submitted>
  <last_update_submitted_qc>July 20, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">July 21, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Immunosuppressive therapy (IST)</keyword>
  <keyword>Refractory</keyword>
  <keyword>Relapsed</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Anemia</mesh_term>
    <mesh_term>Anemia, Aplastic</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>Yes</sharing_ipd>
    <ipd_description>All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing</ipd_description>
    <ipd_info_type>Study Protocol</ipd_info_type>
    <ipd_info_type>Statistical Analysis Plan (SAP)</ipd_info_type>
    <ipd_info_type>Informed Consent Form (ICF)</ipd_info_type>
    <ipd_info_type>Clinical Study Report (CSR)</ipd_info_type>
    <ipd_info_type>Analytic Code</ipd_info_type>
    <ipd_time_frame>Individual anonymized participant data will be considered for sharing once the indication has been approved by a regulatory body, if there is legal authority to share the data and there is not a reasonable likelihood of participant re-identification.</ipd_time_frame>
    <ipd_access_criteria>Qualified researchers may request access to anonymized patient level data or aggregate study data when Regeneron has received marketing authorization from major health authorities (e.g., FDA, European Medicines Agency [EMA], Pharmaceuticals and Medical Devices Agency [PMDA], etc) for the product and indication, has the legal authority to share the data, and has made the study results publicly available (eg, scientific publication, scientific conference, clinical trial registry).</ipd_access_criteria>
    <ipd_url>https://vivli.org/</ipd_url>
  </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/NCT04409080
org study id: R7257-RAA-1947
secondary id: 2020-002031-29
nct id: NCT04409080
lead sponsor: 
has dmc: No
is fda regulated drug: Yes
is fda regulated device: No
The primary objective of this study is to assess the safety and tolerability of REGN7257 in
      patients with immunosuppressive therapy (IST)-refractory or IST-relapsed severe aplastic
      anemia (SAA). An additional primary objective (for Part B only) is to evaluate the clinical
      efficacy of REGN7257 in patients with IST-refractory or IST-relapsed SAA.

      The secondary objectives of this study are to assess the following for REGN7257:

        -  Clinical response over time

        -  Maintenance of response

        -  Impact on transfusion requirements

        -  Effect on blood counts and cell populations

        -  Pharmacokinetics (PK)

        -  Immunogenicity
allocation: N/A
intervention model: Sequential Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Incidence of adverse events (AEs)
time frame: 12 months post-treatment, approximately 52 weeks
description: Part A
measure: Incidence of serious adverse events (SAEs)
time frame: 12 months post-treatment, approximately 52 weeks
description: Parts A and B
measure: Incidence and severity of treatment-emergent adverse events (TEAEs) up
time frame: 12 months post-treatment, approximately 52 weeks
description: Parts A and B
measure: Overall response rate (ORR)
time frame: 6 months post-treatment, approximately 26 weeks
description: Part B
measure: ORR
time frame: 3 months post-treatment, approximately 12 weeks
description: Parts A and B
measure: Complete response (CR)
time frame: 3 months post-treatment, approximately 12 weeks
description: Parts A and B
measure: Partial response (PR)
time frame: 3 months post-treatment, approximately 12 weeks
description: Parts A and B
measure: Time to best response
time frame: Up to 12 months
description: Parts A and B
measure: Time to first response
time frame: Up to 12 months
description: Parts A and B
measure: Proportion of patients who maintain any clinical response
time frame: Up to 12 months
description: Parts A and B
measure: Platelet transfusions per month over time
time frame: Up to 12 months
description: Parts A and B
measure: Red blood cell transfusions per month over time
time frame: Up to 12 months
description: Parts A and B
measure: Changes in lymphocyte cell counts
time frame: Up to 12 months
description: Parts A and B
measure: Changes in neutrophil cell counts
time frame: Up to 12 months
description: Parts A and B
measure: Changes in hemoglobin cell counts
time frame: Up to 12 months
description: Parts A and B
measure: Changes in reticulocyte cell counts
time frame: Up to 12 months
description: Parts A and B
measure: Changes in platelet cell counts
time frame: Up to 12 months
description: Parts A and B
measure: Changes in the whole blood immune cell subsets (T cells)
time frame: Up to 12 months
description: Parts A and B
measure: Changes in the whole blood immune cell subsets (B cells)
time frame: Up to 12 months
description: Parts A and B
measure: Changes in the whole blood immune cell subsets (NK cells)
time frame: Up to 12 months
description: Parts A and B
measure: Drug concentrations in serum over time
time frame: Up to 12 months
description: Parts A and B
measure: Incidence of treatment-emergent anti-drug antibody (ADA) over time
time frame: Up to 12 months
description: Parts A and B
arm group label: Part A and Part B
arm group type: Experimental
description: Part A: Single ascending dose Part B: Preferred dose
intervention type: Drug
intervention name: REGN7257
description: Single dose will be administered by intravenous IV infusion
arm group label: Part A and Part B
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Clinical Trial Management
role: Study Director
affiliation: Regeneron Pharmaceuticals
last name: Clinical Trials Administrator
phone: 844-734-6643
email: clinicaltrials@regeneron.com
facility: 
status: Recruiting
investigator: 
facility: 
status: Recruiting
investigator: 
facility: 
status: Recruiting
facility: 
status: Recruiting
investigator: 
facility: 
status: Recruiting
investigator: 
facility: 
status: Recruiting
investigator: 
facility: 
status: Recruiting
investigator: 
facility: 
status: Completed
facility: 
status: Recruiting
investigator: 
facility: 
status: Recruiting
investigator: 
country: France
country: Korea, Republic of
country: United Kingdom
country: United States
responsible party type: Sponsor
mesh term: Anemia
mesh term: Anemia, Aplastic
sharing ipd: Yes
ipd description: All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing
ipd info type: Study Protocol
ipd info type: Statistical Analysis Plan (SAP)
ipd info type: Informed Consent Form (ICF)
ipd info type: Clinical Study Report (CSR)
ipd info type: Analytic Code
ipd time frame: Individual anonymized participant data will be considered for sharing once the indication has been approved by a regulatory body, if there is legal authority to share the data and there is not a reasonable likelihood of participant re-identification.
ipd access criteria: Qualified researchers may request access to anonymized patient level data or aggregate study data when Regeneron has received marketing authorization from major health authorities (e.g., FDA, European Medicines Agency [EMA], Pharmaceuticals and Medical Devices Agency [PMDA], etc) for the product and indication, has the legal authority to share the data, and has made the study results publicly available (eg, scientific publication, scientific conference, clinical trial registry).
ipd url: https://vivli.org/