<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/NCT01701492</url>
  </required_header>
  <id_info>
    <org_study_id>XPD12-077 BMTPE3</org_study_id>
    <nct_id>NCT01701492</nct_id>
  </id_info>
  <brief_title>Cognitive, Academic and Psychosocial Functioning in Long-Term Survivors of Pediatric Stem Cell Transplantation</brief_title>
  <official_title>Cognitive, Academic and Psychosocial Functioning in Long-Term Survivors of Pediatric Stem Cell Transplantation</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>St. Jude Children's Research Hospital</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>St. Baldrick's Foundation</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>St. Jude Children's Research Hospital</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 observational study to collect information by use of performance-based measures&#xD;
      and survey questionnaires. It does not include interventions aimed at altering patient&#xD;
      outcome.&#xD;
&#xD;
      Advances in pediatric hematopoietic stem cell transplantation (SCT) have resulted in improved&#xD;
      survival and prompted increased attention to the potential adverse late effects of this&#xD;
      procedure. Survivors of SCT are thought to be at risk for neurocognitive deficits as a result&#xD;
      of their exposure to a number of potentially neurotoxic agents. Prior studies done by our&#xD;
      group and others have demonstrated generally stable cognitive function in the first 5 years&#xD;
      following transplant, with little evidence of significant declines. However, there has been&#xD;
      almost no research to date on the status of very long-term (&gt; 5 years post-transplant)&#xD;
      survivors. In this study, we will evaluate a large sample of long-term survivors of&#xD;
      allogeneic SCT using measures of intelligence, academic achievement, and specific cognitive&#xD;
      functions such as attention, working memory and processing speed. We will also obtain&#xD;
      measures of behavioral functioning and quality of life. We will examine how this group of&#xD;
      survivors are functioning relative to normative expectations, and in comparison to community&#xD;
      controls without a history of serious illness, matched on age, gender, race/ethnicity, and&#xD;
      socioeconomic status. We will also examine the relationship between cognitive function and&#xD;
      psychosocial function and quality of life in this population.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This study will involve a one-time assessment using both performance based measures as well&#xD;
      as both self- and parent-report questionnaires. During their yearly visit to St. Jude,&#xD;
      eligible patients who agree to participate will undergo a single neurocognitive evaluation.&#xD;
      This evaluation will take place in the Psychology Clinic. The administration time for the&#xD;
      assessment battery is estimated at 2.5-3.5 hours, with variability depending on the age of&#xD;
      the patient and the speed of completion of the tasks. All measures selected for the study&#xD;
      have well-established psychometric properties, including acceptable reliability and validity.&#xD;
      All measures were standardized on large, representative samples of children and adults and&#xD;
      have age-specific norms.&#xD;
&#xD;
      PRIMARY OBJECTIVE:&#xD;
&#xD;
      To examine and describe psychological outcomes in long-term (&gt; 5 years) survivors of&#xD;
      allogeneic bone marrow or stem cell transplantation (SCT) in childhood. This will be&#xD;
      conducted in a one-time cross-sectional assessment using both performance based measures and&#xD;
      survey questionnaires. Comparisons will be made to established normative data on well&#xD;
      validated standardized instruments and to control participants in the community without a&#xD;
      history of serious illness, matched on age, gender, race/ethnicity and socioeconomic status.&#xD;
&#xD;
        -  To describe global cognitive and academic outcomes in this SCT cohort using measures of&#xD;
           intelligence and academic achievement.&#xD;
&#xD;
        -  To examine performance in specific neuropsychologic domains, including working memory,&#xD;
           processing speed and other executive functions.&#xD;
&#xD;
        -  To examine psychosocial function and quality of life among long-term SCT survivors using&#xD;
           both self- and parent-report.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">October 19, 2012</start_date>
  <completion_date type="Actual">January 23, 2018</completion_date>
  <primary_completion_date type="Actual">January 23, 2018</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>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Intelligence and academic achievement</measure>
    <time_frame>1 day</time_frame>
    <description>To describe global cognitive and academic outcomes in this SCT cohort using measures of intelligence and academic achievement when compared to established normative data on well validated standardized instruments as well as to control participants in the community.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Performance in specific neuropsychologic domains.</measure>
    <time_frame>1 day</time_frame>
    <description>To examine performance in specific neuropsychologic domains, including working memory, processing speed and other executive functions compared to established normative data on well validated standardized instruments as well as to control participants in the community.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Psychosocial function</measure>
    <time_frame>1 day</time_frame>
    <description>To examine psychosocial function among long-term SCT survivors using both self- and parent-report compared to established normative data on well validated standardized instruments as well as to control participants in the community.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Quality of life</measure>
    <time_frame>1 day</time_frame>
    <description>To examine quality of life among long-term SCT survivors using both self- and parent-report compared to established normative data on well validated standardized instruments as well as to control participants in the community.</description>
  </primary_outcome>
  <number_of_groups>2</number_of_groups>
  <enrollment type="Actual">133</enrollment>
  <condition>Stem Cell Transplant During Childhood</condition>
  <condition>Allogeneic Bone Marrow Transplant During Childhood</condition>
  <arm_group>
    <arm_group_label>Stem Cell Transplant Survivors</arm_group_label>
    <description>All participants enrolled on this study will complete a questionnaire and undergo neurocognitive evaluation.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Normal control participants</arm_group_label>
    <description>Control participants in the community without a history of serious illness, matched on age, gender, race/ethnicity and socioeconomic status. They will complete a questionnaire and undergo neurocognitive evaluation.</description>
  </arm_group>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Questionnaire</intervention_name>
    <description>A one-time assessment using both performance-based measures as well as both self- and parent-report questionnaires.</description>
    <arm_group_label>Normal control participants</arm_group_label>
    <arm_group_label>Stem Cell Transplant Survivors</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Neurocognitive Evaluation</intervention_name>
    <description>A one-time neurocognitive evaluation conducted in the Psychology Clinic</description>
    <arm_group_label>Normal control participants</arm_group_label>
    <arm_group_label>Stem Cell Transplant Survivors</arm_group_label>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        Survivors of SCT who are &gt; 5 years since transplant and are returning for a transplant&#xD;
        clinic outpatient follow-up visit will be recruited to participate on study. We will&#xD;
        recruit patients beginning at age 8 years, with no upper age limit, although we anticipate&#xD;
        that most patients will be between 8 and 25 years at the time of assessment. For patients&#xD;
        in the age range 8-21 we will obtain data by both self- and parent-report. However, if&#xD;
        patients age 18-21 present to clinic without a parent, they will still be eligible for&#xD;
        study, and for these patients, as well as those ≤ 21 years, we will obtain performance&#xD;
        based and self-report measures only.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Treated with allogeneic bone marrow or stem cell transplant at St. Jude Children's&#xD;
             Research Hospital (SJCRH)&#xD;
&#xD;
          -  ≤ 21 years at time of transplant&#xD;
&#xD;
          -  &gt; 5 years from date of last transplant&#xD;
&#xD;
          -  Currently at least 8 years of age&#xD;
&#xD;
          -  English as primary language&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Transplanted for metabolic storage disorder, osteogenesis imperfecta, osteopetrosis,&#xD;
             or dyskeratosis congenita.&#xD;
&#xD;
          -  History of central nervous system (CNS) injury/disease predating or unrelated to&#xD;
             reason for SCT&#xD;
&#xD;
          -  Major sensory or motor impairment that would preclude valid cognitive assessment.&#xD;
             However, we will not exclude participants with mild cognitive impairments or special&#xD;
             educational needs.&#xD;
&#xD;
          -  Prior participation in BMTPE protocol at St. Jude Children's Research Hospital.&#xD;
&#xD;
        Control Group Inclusion Criteria&#xD;
&#xD;
          -  Age at least 8 years&#xD;
&#xD;
          -  No known history of serious illness&#xD;
&#xD;
          -  Demographic match to a St. Jude stem cell transplant patient&#xD;
&#xD;
        Control Group Exclusion Criteria&#xD;
&#xD;
          -  Major sensory or motor impairment that would preclude valid cognitive assessment.&#xD;
&#xD;
          -  History of specific CNS disease/injury (e.g., Down Syndrome, traumatic brain injury).&#xD;
             However, we will not exclude participants with mild cognitive impairments or special&#xD;
             educational needs.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>8 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Sean Phipps, PhD</last_name>
    <role>Principal Investigator</role>
    <affiliation>St. Jude Children's Research Hospital</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>St. Jude Children's Research Hospital</name>
      <address>
        <city>Memphis</city>
        <state>Tennessee</state>
        <zip>38105</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <link>
    <url>http://www.stjude.org</url>
    <description>St. Jude Children's Research Hospital</description>
  </link>
  <link>
    <url>http://www.stjude.org</url>
    <description>Clinical Trials Open at St. Jude</description>
  </link>
  <verification_date>May 2018</verification_date>
  <study_first_submitted>October 3, 2012</study_first_submitted>
  <study_first_submitted_qc>October 4, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">October 5, 2012</study_first_posted>
  <last_update_submitted>May 17, 2018</last_update_submitted>
  <last_update_submitted_qc>May 17, 2018</last_update_submitted_qc>
  <last_update_posted type="Actual">May 18, 2018</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>pediatric</keyword>
  <keyword>hematopoietic stem cell transplantation</keyword>
  <keyword>allogeneic bone marrow transplantation</keyword>
  <!-- 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/NCT01701492
org study id: XPD12-077 BMTPE3
nct id: NCT01701492
lead sponsor: 
collaborator: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
This is an observational study to collect information by use of performance-based measures
      and survey questionnaires. It does not include interventions aimed at altering patient
      outcome.

      Advances in pediatric hematopoietic stem cell transplantation (SCT) have resulted in improved
      survival and prompted increased attention to the potential adverse late effects of this
      procedure. Survivors of SCT are thought to be at risk for neurocognitive deficits as a result
      of their exposure to a number of potentially neurotoxic agents. Prior studies done by our
      group and others have demonstrated generally stable cognitive function in the first 5 years
      following transplant, with little evidence of significant declines. However, there has been
      almost no research to date on the status of very long-term (> 5 years post-transplant)
      survivors. In this study, we will evaluate a large sample of long-term survivors of
      allogeneic SCT using measures of intelligence, academic achievement, and specific cognitive
      functions such as attention, working memory and processing speed. We will also obtain
      measures of behavioral functioning and quality of life. We will examine how this group of
      survivors are functioning relative to normative expectations, and in comparison to community
      controls without a history of serious illness, matched on age, gender, race/ethnicity, and
      socioeconomic status. We will also examine the relationship between cognitive function and
      psychosocial function and quality of life in this population.
This study will involve a one-time assessment using both performance based measures as well
      as both self- and parent-report questionnaires. During their yearly visit to St. Jude,
      eligible patients who agree to participate will undergo a single neurocognitive evaluation.
      This evaluation will take place in the Psychology Clinic. The administration time for the
      assessment battery is estimated at 2.5-3.5 hours, with variability depending on the age of
      the patient and the speed of completion of the tasks. All measures selected for the study
      have well-established psychometric properties, including acceptable reliability and validity.
      All measures were standardized on large, representative samples of children and adults and
      have age-specific norms.

      PRIMARY OBJECTIVE:

      To examine and describe psychological outcomes in long-term (> 5 years) survivors of
      allogeneic bone marrow or stem cell transplantation (SCT) in childhood. This will be
      conducted in a one-time cross-sectional assessment using both performance based measures and
      survey questionnaires. Comparisons will be made to established normative data on well
      validated standardized instruments and to control participants in the community without a
      history of serious illness, matched on age, gender, race/ethnicity and socioeconomic status.

        -  To describe global cognitive and academic outcomes in this SCT cohort using measures of
           intelligence and academic achievement.

        -  To examine performance in specific neuropsychologic domains, including working memory,
           processing speed and other executive functions.

        -  To examine psychosocial function and quality of life among long-term SCT survivors using
           both self- and parent-report.
observational model: Cohort
time perspective: Prospective
measure: Intelligence and academic achievement
time frame: 1 day
description: To describe global cognitive and academic outcomes in this SCT cohort using measures of intelligence and academic achievement when compared to established normative data on well validated standardized instruments as well as to control participants in the community.
measure: Performance in specific neuropsychologic domains.
time frame: 1 day
description: To examine performance in specific neuropsychologic domains, including working memory, processing speed and other executive functions compared to established normative data on well validated standardized instruments as well as to control participants in the community.
measure: Psychosocial function
time frame: 1 day
description: To examine psychosocial function among long-term SCT survivors using both self- and parent-report compared to established normative data on well validated standardized instruments as well as to control participants in the community.
measure: Quality of life
time frame: 1 day
description: To examine quality of life among long-term SCT survivors using both self- and parent-report compared to established normative data on well validated standardized instruments as well as to control participants in the community.
arm group label: Stem Cell Transplant Survivors
description: All participants enrolled on this study will complete a questionnaire and undergo neurocognitive evaluation.
arm group label: Normal control participants
description: Control participants in the community without a history of serious illness, matched on age, gender, race/ethnicity and socioeconomic status. They will complete a questionnaire and undergo neurocognitive evaluation.
intervention type: Other
intervention name: Questionnaire
description: A one-time assessment using both performance-based measures as well as both self- and parent-report questionnaires.
arm group label: Normal control participants
arm group label: Stem Cell Transplant Survivors
intervention type: Other
intervention name: Neurocognitive Evaluation
description: A one-time neurocognitive evaluation conducted in the Psychology Clinic
arm group label: Normal control participants
arm group label: Stem Cell Transplant Survivors
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 8 Years
maximum age: N/A
healthy volunteers: No
last name: Sean Phipps, PhD
role: Principal Investigator
affiliation: St. Jude Children's Research Hospital
facility: 
country: United States
url: http://www.stjude.org
description: St. Jude Children's Research Hospital
url: http://www.stjude.org
description: Clinical Trials Open at St. Jude
responsible party type: Sponsor