<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/NCT04423367</url>
  </required_header>
  <id_info>
    <org_study_id>IHBDH-IIT2020010</org_study_id>
    <nct_id>NCT04423367</nct_id>
  </id_info>
  <brief_title>Bortezomib Plus Dexamethasone for Acquired Pure Red Cell Aplasia Failure or Relapse After First-line Treatment</brief_title>
  <acronym>BID-PERAL</acronym>
  <official_title>Safety and Efficacy of Bortezomib Plus Dexamethasone for Acquired Pure Red Cell Aplasia Failure or Relapse After First-line Treatment: A Prospective Phase II Trial</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Institute of Hematology &amp; Blood Diseases Hospital, China</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Institute of Hematology &amp; Blood Diseases Hospital, China</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      This is an open-label, single-arm study to evaluate the safety and efficacy of bortezomib&#xD;
      plus dexamethasone for acquired pure red cell aplasia failure or relapse after first-line&#xD;
      treatment.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">September 13, 2020</start_date>
  <completion_date type="Anticipated">December 31, 2024</completion_date>
  <primary_completion_date type="Anticipated">November 30, 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>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Overall response rate</measure>
    <time_frame>within 12 weeks</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>Frequency and severity of adverse events and severe adverse events</measure>
    <time_frame>within 12 weeks</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Relapse free survival</measure>
    <time_frame>within 24 and 48 weeks</time_frame>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Anticipated">17</enrollment>
  <condition>Acquired Pure Red Cell Aplasia</condition>
  <arm_group>
    <arm_group_label>bortezomib/dexamethasone</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Enrolled patients will receive the combination therapy of bortezomib and dexamethasone.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>bortezomib/dexamethasone</intervention_name>
    <description>Enrolled patients would receive the combination therapy of bortezomib and dexamethasone.</description>
    <arm_group_label>bortezomib/dexamethasone</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  ECOG ≤ 2.&#xD;
&#xD;
          -  Age from 18 to 70.&#xD;
&#xD;
          -  Diagnosed with acquired pure red cell aplasia.&#xD;
&#xD;
          -  Meets the criteria of first-line treatment failure or relapse.&#xD;
&#xD;
          -  Organs in good function.&#xD;
&#xD;
          -  Signed informed consent.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Nursing woman.&#xD;
&#xD;
          -  Active bacterial, virus, fungal or parasitic infection, including HIV infection, HBsAg&#xD;
             or HBV DNA positive, HCV DNA positive, etc.&#xD;
&#xD;
          -  Secondary PRCA caused by lymphoproliferative disorders, including large granular&#xD;
             lymphocytic leukemia, waldenström macroglobulinemia, small lymphocytic&#xD;
             lymphoma/chronic lymphocytic leukemia, etc.&#xD;
&#xD;
          -  Secondary PRCA caused by either smoldering multiple myeloma or symptomatic multiple&#xD;
             myeloma.&#xD;
&#xD;
          -  Secondary PRCA caused by either ABO major mismatched stem cell transplantation or&#xD;
             organ transplantation.&#xD;
&#xD;
          -  Secondary PRCA caused by solid tumors except for thymoma.&#xD;
&#xD;
          -  Secondary PRCA caused by drugs or pregnancy.&#xD;
&#xD;
          -  Secondary PRCA caused by the B19 virus.&#xD;
&#xD;
          -  Have contraindications for glucocorticoids, or unable to tolerate glucocorticoids for&#xD;
             comorbidities.&#xD;
&#xD;
          -  Previously received treatment in other trials within 4 weeks before enrollment.&#xD;
&#xD;
          -  Previously treated with the proteasome inhibitor.&#xD;
&#xD;
          -  Experience active hemorrhage condition, including gastrointestinal bleeding,&#xD;
             respiratory tract bleeding and central nervous system bleeding within 2 months before&#xD;
             enrollment or during bortezomib/dexamethasone treatment.&#xD;
&#xD;
          -  Have a history of malignant tumors.&#xD;
&#xD;
          -  Have a history of mental illness.&#xD;
&#xD;
          -  Inability to understand or to follow study procedures.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>70 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Jun Shi, PhD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Institute of Hematology &amp; Blood Diseases Hospital, China</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Jun Shi, PhD</last_name>
    <phone>(86)2223900913</phone>
    <email>shijun@ihcams.ac.cn</email>
  </overall_contact>
  <overall_contact_backup>
    <last_name>Lele Zhang, MD</last_name>
    <phone>(86)15811139278</phone>
    <email>zhanglele@ihcams.ac.cn</email>
  </overall_contact_backup>
  <location>
    <facility>
      <name>Zhoukou Central Hospital</name>
      <address>
        <city>Zhoukou</city>
        <state>Henan</state>
        <country>China</country>
      </address>
    </facility>
    <status>Not yet recruiting</status>
    <contact>
      <last_name>Qian Liang, PhD</last_name>
      <phone>15703815972</phone>
      <email>lqw141230@163.com</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>The Second Affilated Hospital of Shandong First Medical University</name>
      <address>
        <city>Tai'an</city>
        <state>Shandong</state>
        <country>China</country>
      </address>
    </facility>
    <status>Not yet recruiting</status>
    <contact>
      <last_name>Hong Zhang</last_name>
      <phone>18505386878</phone>
      <email>tyfyzhh@163.com</email>
    </contact>
  </location>
  <location>
    <facility>
      <name>Regenerative Medicine Center</name>
      <address>
        <city>Tianjin</city>
        <country>China</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Jun Shi, PhD</last_name>
      <phone>(86)2223900913</phone>
      <email>shijun@ihcams.ac.cn</email>
    </contact>
    <contact_backup>
      <last_name>Jingyu Zhao, MPH</last_name>
      <phone>(86)13752253515</phone>
      <email>zhaojingyu@ihcams.ac.cn</email>
    </contact_backup>
  </location>
  <location_countries>
    <country>China</country>
  </location_countries>
  <reference>
    <citation>Zhang L, Chen N, Xu Z, Liang Q, Pan H, Zhao J, Fang L, Shi J. Good treatment-free survival of monoclonal gammopathy of undetermined significance associated pure red cell aplasia after bortezomib plus dexamethasone. Blood Cells Mol Dis. 2021 Jul;89:102573. doi: 10.1016/j.bcmd.2021.102573. Epub 2021 Apr 27.</citation>
    <PMID>33957358</PMID>
  </reference>
  <verification_date>December 2022</verification_date>
  <study_first_submitted>June 5, 2020</study_first_submitted>
  <study_first_submitted_qc>June 5, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 9, 2020</study_first_posted>
  <last_update_submitted>December 4, 2022</last_update_submitted>
  <last_update_submitted_qc>December 4, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">December 6, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Institute of Hematology &amp; Blood Diseases Hospital, China</investigator_affiliation>
    <investigator_full_name>Jun Shi</investigator_full_name>
    <investigator_title>Director, Regenerative Medicine Clinic Center</investigator_title>
  </responsible_party>
  <keyword>Acquired Pure Red Cell Aplasia</keyword>
  <keyword>PRCA</keyword>
  <keyword>Failure or Relapse after First-line Treatment</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Red-Cell Aplasia, Pure</mesh_term>
    <mesh_term>Recurrence</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Dexamethasone</mesh_term>
    <mesh_term>Bortezomib</mesh_term>
  </intervention_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/NCT04423367
org study id: IHBDH-IIT2020010
nct id: NCT04423367
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
This is an open-label, single-arm study to evaluate the safety and efficacy of bortezomib
      plus dexamethasone for acquired pure red cell aplasia failure or relapse after first-line
      treatment.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Overall response rate
time frame: within 12 weeks
measure: Frequency and severity of adverse events and severe adverse events
time frame: within 12 weeks
measure: Relapse free survival
time frame: within 24 and 48 weeks
arm group label: bortezomib/dexamethasone
arm group type: Experimental
description: Enrolled patients will receive the combination therapy of bortezomib and dexamethasone.
intervention type: Drug
intervention name: bortezomib/dexamethasone
description: Enrolled patients would receive the combination therapy of bortezomib and dexamethasone.
arm group label: bortezomib/dexamethasone
criteria: 
gender: All
minimum age: 18 Years
maximum age: 70 Years
healthy volunteers: No
last name: Jun Shi, PhD
role: Principal Investigator
affiliation: Institute of Hematology & Blood Diseases Hospital, China
last name: Jun Shi, PhD
phone: (86)2223900913
email: shijun@ihcams.ac.cn
last name: Lele Zhang, MD
phone: (86)15811139278
email: zhanglele@ihcams.ac.cn
facility: 
status: Not yet recruiting
contact: 
facility: 
status: Not yet recruiting
contact: 
facility: 
status: Recruiting
contact: 
contact backup: 
country: China
citation: Zhang L, Chen N, Xu Z, Liang Q, Pan H, Zhao J, Fang L, Shi J. Good treatment-free survival of monoclonal gammopathy of undetermined significance associated pure red cell aplasia after bortezomib plus dexamethasone. Blood Cells Mol Dis. 2021 Jul;89:102573. doi: 10.1016/j.bcmd.2021.102573. Epub 2021 Apr 27.
PMID: 33957358
responsible party type: Principal Investigator
investigator affiliation: Institute of Hematology & Blood Diseases Hospital, China
investigator full name: Jun Shi
investigator title: Director, Regenerative Medicine Clinic Center
mesh term: Red-Cell Aplasia, Pure
mesh term: Recurrence
mesh term: Dexamethasone
mesh term: Bortezomib
sharing ipd: No