<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/NCT04436380</url>
  </required_header>
  <id_info>
    <org_study_id>999920119</org_study_id>
    <secondary_id>20-H-N119</secondary_id>
    <nct_id>NCT04436380</nct_id>
  </id_info>
  <brief_title>Retrospective Study of Patients With Severe Aplastic Anemia Who Relapsed After Immunosuppressive Therapy</brief_title>
  <official_title>Retrospective Study of Patients With Severe Aplastic Anemia Who Relapsed After Immunosuppressive Therapy</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>National Heart, Lung, and Blood Institute (NHLBI)</agency>
      <agency_class>NIH</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>National Institutes of Health Clinical Center (CC)</source>
  <brief_summary>
    <textblock>
      Background:&#xD;
&#xD;
      Severe aplastic anemia (SAA) is a form of bone marrow failure. It usually results from a&#xD;
&#xD;
      cytotoxic T cell attack on the marrow stem cell. Two treatments can be used for most people&#xD;
      with SAA. One is allogeneic hematopoietic stem cell transplant (HSCT). The other is&#xD;
      immunosuppressive treatment (IST). For people who are treated with IST, relapse can occur. If&#xD;
      this happens, they can have HSCT or be re-treated with IST. The two most common IST regimes&#xD;
      used for relapsed SAA are rabbit ATG (rATG) and alemtuzumab. Both rATG and alemtuzumab have&#xD;
      similar response rates and survival rates. There is not much long-term data on people who&#xD;
      need repeat IST treatment due to relapse. Researchers want to look at data from past studies&#xD;
      to learn more.&#xD;
&#xD;
      Objective:&#xD;
&#xD;
      To compare the data of relapsed SAA patients between those who received alemtuzumab versus&#xD;
      rATG for repeat IST treatment.&#xD;
&#xD;
      Eligibility:&#xD;
&#xD;
      Adults and children with SAA who were enrolled on NHLBI protocol 12-H-0150, 06-H-0034,&#xD;
      05-H-0242, 03-H-0249, 03-H-0193, 00-H-0032, or 90-H-0146&#xD;
&#xD;
      Design:&#xD;
&#xD;
      This study uses data from past studies. The participants in those studies have allowed their&#xD;
      data to be used in future research.&#xD;
&#xD;
      Researchers will review participants medical records. They will collect clinical data, such&#xD;
      as notes, test results, and imaging scans. They will also collect the research data gathered&#xD;
      as part of the original study.&#xD;
&#xD;
      Researchers will enter the data into an in-house database. It is password protected. All data&#xD;
      will be kept in secure network drives or in secure sites.&#xD;
&#xD;
      Other studies may be added in the future....&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Severe aplastic anemia (SAA) is a form of bone marrow failure and in most cases is the result&#xD;
      of a cytotoxic T cell attack on the marrow stem cell. It is effectively treated in most&#xD;
      patients with either immunosuppressive treatment (IST) or upfront allogeneic hematopoietic&#xD;
      stem cell transplant (HSCT). However, in patients who are treated with IST, relapse remains&#xD;
      an issue with a rate of 30-40%. Once relapsed, patients can be either re-treated with IST or&#xD;
      undergo HSCT. The two most common IST regimes used for relapsed SAA are rabbit ATG (rATG) and&#xD;
      alemtuzumab. Both rATG and alemtuzumab have been shown to have a response rate of&#xD;
      approximately 60% in relapsed patients with similar overall survival rates of 83-85%.&#xD;
      Previous predictors of relapse include time from diagnosis to IST, and early response to IST.&#xD;
      Few long-term data exist on patients requiring repeat IST treatment due to relapse.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">June 15, 2020</start_date>
  <completion_date type="Actual">March 15, 2023</completion_date>
  <primary_completion_date type="Actual">March 15, 2023</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Cohort</observational_model>
    <time_perspective>Retrospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Assessment of long-term outcomes of relapsed patients who received repeat IST</measure>
    <time_frame>Between the Period of 1990 to 2020</time_frame>
    <description>Characteristics and outcomes of relapsed SAA patients between those who received alemtuzumab versus rATG for repeat IST</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Clinical predictors for the development of relapse</measure>
    <time_frame>Between the Period of 1990 to 2020</time_frame>
    <description>Clinical predictors for the development of relapse such as age, gender, baseline laboratory values, time from diagnosis to initial treatment, and initial treatment received.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Rates of clonal evolution between relapsed patients depending on type of IST received and number of IST rounds required</measure>
    <time_frame>Between the Period of 1990 to 2020</time_frame>
    <description>Compare rates of clonal evolution between relapsed patients depending on type of IST received and number of IST rounds required.</description>
  </secondary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Actual">173</enrollment>
  <condition>Severe Aplastic Anemia</condition>
  <arm_group>
    <arm_group_label>Relapsed SAA Patients</arm_group_label>
    <description>Patients with Severe Aplastic Anemia who Relapsed after Immunosuppressive Therapy</description>
  </arm_group>
  <eligibility>
    <study_pop>
      <textblock>
        Relapsed SAA Subjects who had a repeat IST&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        -  This is a retrospective chart review. The investigators will review the medical&#xD;
             records of adults and children with severe aplastic anemia (SAA) enrolled on&#xD;
             immunosuppressive therapy (IST) treatment protocols in the NHLBI.&#xD;
&#xD;
        Patients who opted out of future use of data on their prior studies will be excluded from&#xD;
        this study.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>2 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Emma M Groarke, M.D.</last_name>
    <role>Principal Investigator</role>
    <affiliation>National Heart, Lung, and Blood Institute (NHLBI)</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>National Heart, Lung and Blood Institute (NHLBI)</name>
      <address>
        <city>Bethesda</city>
        <state>Maryland</state>
        <zip>20892</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>March 2023</verification_date>
  <study_first_submitted>June 17, 2020</study_first_submitted>
  <study_first_submitted_qc>June 17, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 18, 2020</study_first_posted>
  <last_update_submitted>March 15, 2023</last_update_submitted>
  <last_update_submitted_qc>March 15, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">March 16, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Alemtuzumab</keyword>
  <keyword>rATG</keyword>
  <keyword>Natural History</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>
  <!-- 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/NCT04436380
org study id: 999920119
secondary id: 20-H-N119
nct id: NCT04436380
lead sponsor: 
Background:

      Severe aplastic anemia (SAA) is a form of bone marrow failure. It usually results from a

      cytotoxic T cell attack on the marrow stem cell. Two treatments can be used for most people
      with SAA. One is allogeneic hematopoietic stem cell transplant (HSCT). The other is
      immunosuppressive treatment (IST). For people who are treated with IST, relapse can occur. If
      this happens, they can have HSCT or be re-treated with IST. The two most common IST regimes
      used for relapsed SAA are rabbit ATG (rATG) and alemtuzumab. Both rATG and alemtuzumab have
      similar response rates and survival rates. There is not much long-term data on people who
      need repeat IST treatment due to relapse. Researchers want to look at data from past studies
      to learn more.

      Objective:

      To compare the data of relapsed SAA patients between those who received alemtuzumab versus
      rATG for repeat IST treatment.

      Eligibility:

      Adults and children with SAA who were enrolled on NHLBI protocol 12-H-0150, 06-H-0034,
      05-H-0242, 03-H-0249, 03-H-0193, 00-H-0032, or 90-H-0146

      Design:

      This study uses data from past studies. The participants in those studies have allowed their
      data to be used in future research.

      Researchers will review participants medical records. They will collect clinical data, such
      as notes, test results, and imaging scans. They will also collect the research data gathered
      as part of the original study.

      Researchers will enter the data into an in-house database. It is password protected. All data
      will be kept in secure network drives or in secure sites.

      Other studies may be added in the future....
Severe aplastic anemia (SAA) is a form of bone marrow failure and in most cases is the result
      of a cytotoxic T cell attack on the marrow stem cell. It is effectively treated in most
      patients with either immunosuppressive treatment (IST) or upfront allogeneic hematopoietic
      stem cell transplant (HSCT). However, in patients who are treated with IST, relapse remains
      an issue with a rate of 30-40%. Once relapsed, patients can be either re-treated with IST or
      undergo HSCT. The two most common IST regimes used for relapsed SAA are rabbit ATG (rATG) and
      alemtuzumab. Both rATG and alemtuzumab have been shown to have a response rate of
      approximately 60% in relapsed patients with similar overall survival rates of 83-85%.
      Previous predictors of relapse include time from diagnosis to IST, and early response to IST.
      Few long-term data exist on patients requiring repeat IST treatment due to relapse.
observational model: Cohort
time perspective: Retrospective
measure: Assessment of long-term outcomes of relapsed patients who received repeat IST
time frame: Between the Period of 1990 to 2020
description: Characteristics and outcomes of relapsed SAA patients between those who received alemtuzumab versus rATG for repeat IST
measure: Clinical predictors for the development of relapse
time frame: Between the Period of 1990 to 2020
description: Clinical predictors for the development of relapse such as age, gender, baseline laboratory values, time from diagnosis to initial treatment, and initial treatment received.
measure: Rates of clonal evolution between relapsed patients depending on type of IST received and number of IST rounds required
time frame: Between the Period of 1990 to 2020
description: Compare rates of clonal evolution between relapsed patients depending on type of IST received and number of IST rounds required.
arm group label: Relapsed SAA Patients
description: Patients with Severe Aplastic Anemia who Relapsed after Immunosuppressive Therapy
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 2 Years
maximum age: N/A
healthy volunteers: No
last name: Emma M Groarke, M.D.
role: Principal Investigator
affiliation: National Heart, Lung, and Blood Institute (NHLBI)
facility: 
country: United States
responsible party type: Sponsor
mesh term: Anemia
mesh term: Anemia, Aplastic