<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/NCT03000244</url>
  </required_header>
  <id_info>
    <org_study_id>170027</org_study_id>
    <secondary_id>17-C-0027</secondary_id>
    <nct_id>NCT03000244</nct_id>
  </id_info>
  <brief_title>Long-Term Follow-up of People Undergoing Hematopoietic Stem Cell Transplantation</brief_title>
  <official_title>Long-Term Follow up of Patients Undergoing Hematopoietic Stem Cell Transplantation</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>National Cancer Institute (NCI)</agency>
      <agency_class>NIH</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>National Institutes of Health Clinical Center (CC)</source>
  <oversight_info>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      Background:&#xD;
&#xD;
      People who have had an allogeneic hematopoietic stem cell transplant (HCT) have bone marrow&#xD;
      or an immune system that is damaged. They get stem cells from a donor who is a relative.&#xD;
      Researchers want to study stem cell donors and recipients to learn about the long-term&#xD;
      effects of HCT. They want to learn how the stem cells change and how to improve their ability&#xD;
      to fight cancer.&#xD;
&#xD;
      Objective:&#xD;
&#xD;
      To provide long-term follow-up care for people who underwent or will undergo HCT. To collect&#xD;
      data, blood, and tissue samples to learn about late complications after HCT.&#xD;
&#xD;
      Eligibility:&#xD;
&#xD;
      Adults age 18 and older who will undergo HCT or underwent HCT and are surviving one year or&#xD;
      more from the date of HCT. The stem cell donors for these recipients are also needed.&#xD;
&#xD;
      Design:&#xD;
&#xD;
      Recipients will have 1 visit each year. They will have a physical exam. They will answer&#xD;
      questions about their medical history and health. They will receive screening and&#xD;
      surveillance testing. They will complete brief questionnaires.&#xD;
&#xD;
      Recipients will have blood tests. They may have tissue biopsies or specimens (such as tissue&#xD;
      in their cheek or skin or bone marrow biopsy).&#xD;
&#xD;
      Recipients will give their current address and phone number, and the same data for one or two&#xD;
      other people, who can get in contact with them.&#xD;
&#xD;
      After the first visit at the clinic, some recipients may see a doctor close to home to get&#xD;
      the necessary information and send it to NIH.&#xD;
&#xD;
      Donors will come to the clinic for 1 visit. They will answer questions about their medical&#xD;
      history. Blood samples will be taken.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Background:&#xD;
&#xD;
        -  Patients who survive more than 3 years after allogeneic hematopoietic stem cell&#xD;
           transplantation (HCT) have a high probability of being cured from their underlying&#xD;
           disease; however, mortality rates remain 4 to 9-fold higher than the general population&#xD;
           for at least 30 years after HCT.&#xD;
&#xD;
        -  The most common causes of late mortality in HCT survivors include second malignancies,&#xD;
           relapsed/recurrent disease, infections, chronic graft-versus-host disease (GVHD),&#xD;
           respiratory diseases, and cardiovascular diseases.&#xD;
&#xD;
        -  Guidelines for recommended screening and preventive practices for long-term survivors&#xD;
           after HCT have been developed, and can be implemented to detect or prevent late&#xD;
           complications after HCT.&#xD;
&#xD;
        -  Understanding the pattern of immune reconstitution and immune dysregulation after HCT&#xD;
           may help to elucidate the root mechanisms of late complications.&#xD;
&#xD;
      Objective:&#xD;
&#xD;
      -To collect clinical data on patients and late complications after HCT&#xD;
&#xD;
      Eligibility:&#xD;
&#xD;
        -  Patients who underwent HCT for any indication and are surviving one year or more from&#xD;
           the date of HCT OR patients who will be undergoing HCT on an ETIB protocol.&#xD;
&#xD;
        -  Related stem cell donors of patients meeting the above criteria who participated or will&#xD;
           be participating as a donor of stem cells or leukocytes.&#xD;
&#xD;
        -  Age greater than or equal to 4 years&#xD;
&#xD;
        -  Any active disease relapse or new hematologic malignancy including post-transplant&#xD;
           lymphoproliferative disorder (PTLD) are excluded from protocol enrollment.&#xD;
&#xD;
      Design:&#xD;
&#xD;
        -  Prospective, longitudinal study of the natural history of long-term survivors after HCT.&#xD;
&#xD;
        -  Clinical data on post-transplant complications and peripheral blood samples will be&#xD;
           collected at yearly evaluations by the Principal Investigator, LAI, AI or representative&#xD;
           investigator of the original HCT protocol.&#xD;
&#xD;
        -  If patient develops complications such as new cancers, relapsed disease, new hematologic&#xD;
           malignancy or PTLD they are exempt from required evaluations, and followed for survival&#xD;
           and causes of death (management of complications as per primary team).&#xD;
&#xD;
        -  As this is a longitudinal registry study without any planned intervention, there is no&#xD;
           formal statistical analysis plan or accrual goal; descriptive data will be reported.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">April 26, 2017</start_date>
  <completion_date type="Anticipated">August 12, 2050</completion_date>
  <primary_completion_date type="Anticipated">August 12, 2050</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>Basic characteristics of the patient population</measure>
    <time_frame>at death</time_frame>
    <description>Survival</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Provide a mechanism to ensure implementation of standard of care recommendations for post-transplant followup</measure>
    <time_frame>Ongoing throughout study</time_frame>
    <description>Observation of adherence and effects of standard of care recommendations</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Obtain blood samples from stem cell transplant donors to study how biological factors related to the donor are associated with late complications</measure>
    <time_frame>Ongoing throughout study</time_frame>
    <description>Studying donor samples for potential correlations to transplant recipient complications.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Obtain blood and tissue samples to study and document immunologic reconstitution after stem cell transplant</measure>
    <time_frame>Ongoing throughout study</time_frame>
    <description>Immune monitoring for analysis</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Monitoring of late effects after stem cell transplant for patients of ETIB protocols</measure>
    <time_frame>Ongoing throughout study</time_frame>
    <description>Survival and observation of long-term outcomes.</description>
  </secondary_outcome>
  <number_of_groups>3</number_of_groups>
  <enrollment type="Anticipated">2000</enrollment>
  <condition>Hematopoietic Stem Cell Transplantation</condition>
  <condition>Tissue Donors</condition>
  <arm_group>
    <arm_group_label>1/Patients</arm_group_label>
    <description>Patients who underwent hematopoietic stem cell transplant for any indication (malignant or non-malignant).</description>
  </arm_group>
  <arm_group>
    <arm_group_label>2/Donors</arm_group_label>
    <description>Related stem cell donors of those in Patients cohort.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>3/Parents of patients</arm_group_label>
    <description>Parents/guardians of minors enrolled in cohort 1</description>
  </arm_group>
  <eligibility>
    <study_pop>
      <textblock>
        Patients who have received an allogeneic stem cell transplant as well as donors of stem&#xD;
        cells who are related to the patients who are study participants. Parents of minor&#xD;
        recipients may also be enrolled to complete surveys. Participants can be from any community&#xD;
        or co-enrolled on another NIH protocol.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        -  INCLUSION CRITERIA FOR PATIENT SUBJECTS:&#xD;
&#xD;
          -  Patients who underwent HCT for any indication (malignant or non-malignant) and are&#xD;
             surviving one year or more from the date of HCT (patients may be at any time point&#xD;
             after HCT as long as it is greater than or equal to 1 year) OR patients who will be&#xD;
             undergoing HCT for any indication (malignant or non-malignant) on an ETIB protocol.&#xD;
             Patients do not need to have been transplanted under an ETIB protocol.&#xD;
&#xD;
          -  Age greater than or equal to 4 years&#xD;
&#xD;
          -  Ability of patient or patient s Legally Authorized Representative (LAR) to understand&#xD;
             and the willingness to sign a written informed consent document&#xD;
&#xD;
          -  Patients will need to have a primary care physician that will provide continued&#xD;
             comprehensive care throughout the patient s participation in the study.&#xD;
&#xD;
        EXCLUSION CRITERIA FOR PATIENT SUBJECTS:&#xD;
&#xD;
        -Patients with active disease relapse or new hematologic malignancy including&#xD;
        post-transplant lymphoproliferative disorder (PTLD) are excluded from protocol enrollment.&#xD;
&#xD;
        INCLUSION CRITERIA FOR DONOR SUBJECTS:&#xD;
&#xD;
          -  Related stem cell donors of patients meeting the above criteria as a donor of stem&#xD;
             cells or leukocytes&#xD;
&#xD;
          -  Age greater than or equal to 4 years&#xD;
&#xD;
          -  Ability of patient or patient s Legally Authorized Representative (LAR) to understand&#xD;
             and the willingness to sign a written informed&#xD;
&#xD;
        consent document&#xD;
&#xD;
        INCLUSION CRITERIA FOR PARENTS COMPLETING SURVEYS:&#xD;
&#xD;
          -  Parents/guardians of minors enrolled on the study who have undergone HCT&#xD;
&#xD;
          -  Willingness to complete surveys about the minor that underwent HCT&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>4 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>Accepts Healthy Volunteers</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Steven Z Pavletic, M.D.</last_name>
    <role>Principal Investigator</role>
    <affiliation>National Cancer Institute (NCI)</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Ashley E Carpenter</last_name>
    <phone>(240) 760-6009</phone>
    <email>carpentera@mail.nih.gov</email>
  </overall_contact>
  <overall_contact_backup>
    <last_name>Steven Z Pavletic, M.D.</last_name>
    <phone>(240) 760-6174</phone>
    <email>sp326h@nih.gov</email>
  </overall_contact_backup>
  <location>
    <facility>
      <name>National Institutes of Health Clinical Center</name>
      <address>
        <city>Bethesda</city>
        <state>Maryland</state>
        <zip>20892</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>For more information at the NIH Clinical Center contact National Cancer Institute Referral Office</last_name>
      <phone>888-624-1937</phone>
    </contact>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <link>
    <url>https://clinicalstudies.info.nih.gov/cgi/detail.cgi?A_2017-C-0027.html</url>
    <description>NIH Clinical Center Detailed Web Page</description>
  </link>
  <verification_date>August 25, 2023</verification_date>
  <study_first_submitted>December 21, 2016</study_first_submitted>
  <study_first_submitted_qc>December 21, 2016</study_first_submitted_qc>
  <study_first_posted type="Estimate">December 22, 2016</study_first_posted>
  <last_update_submitted>August 26, 2023</last_update_submitted>
  <last_update_submitted_qc>August 26, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">August 29, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Natural History</keyword>
  <keyword>HCT</keyword>
  <patient_data>
    <sharing_ipd>Yes</sharing_ipd>
    <ipd_description>.All collected IPD will be shared.@@@@@@All IPD recorded in the medical record will be shared with intramural investigators upon request. In addition, all large scale genomic sequencing data will be shared with subscribers to dbGaP.</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_time_frame>Clinical data available during the study and indefinitely.@@@@@@Genomic data are available once genomic data are uploaded per protocol GDS plan for as long as database is active.</ipd_time_frame>
    <ipd_access_criteria>Data from this study may be requested by contacting the PI.@@@@@@Genomic data are made available via dbGaP through requests to the data custodians.</ipd_access_criteria>
  </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/NCT03000244
org study id: 170027
secondary id: 17-C-0027
nct id: NCT03000244
lead sponsor: 
is fda regulated drug: No
is fda regulated device: No
Background:

      People who have had an allogeneic hematopoietic stem cell transplant (HCT) have bone marrow
      or an immune system that is damaged. They get stem cells from a donor who is a relative.
      Researchers want to study stem cell donors and recipients to learn about the long-term
      effects of HCT. They want to learn how the stem cells change and how to improve their ability
      to fight cancer.

      Objective:

      To provide long-term follow-up care for people who underwent or will undergo HCT. To collect
      data, blood, and tissue samples to learn about late complications after HCT.

      Eligibility:

      Adults age 18 and older who will undergo HCT or underwent HCT and are surviving one year or
      more from the date of HCT. The stem cell donors for these recipients are also needed.

      Design:

      Recipients will have 1 visit each year. They will have a physical exam. They will answer
      questions about their medical history and health. They will receive screening and
      surveillance testing. They will complete brief questionnaires.

      Recipients will have blood tests. They may have tissue biopsies or specimens (such as tissue
      in their cheek or skin or bone marrow biopsy).

      Recipients will give their current address and phone number, and the same data for one or two
      other people, who can get in contact with them.

      After the first visit at the clinic, some recipients may see a doctor close to home to get
      the necessary information and send it to NIH.

      Donors will come to the clinic for 1 visit. They will answer questions about their medical
      history. Blood samples will be taken.
Background:

        -  Patients who survive more than 3 years after allogeneic hematopoietic stem cell
           transplantation (HCT) have a high probability of being cured from their underlying
           disease; however, mortality rates remain 4 to 9-fold higher than the general population
           for at least 30 years after HCT.

        -  The most common causes of late mortality in HCT survivors include second malignancies,
           relapsed/recurrent disease, infections, chronic graft-versus-host disease (GVHD),
           respiratory diseases, and cardiovascular diseases.

        -  Guidelines for recommended screening and preventive practices for long-term survivors
           after HCT have been developed, and can be implemented to detect or prevent late
           complications after HCT.

        -  Understanding the pattern of immune reconstitution and immune dysregulation after HCT
           may help to elucidate the root mechanisms of late complications.

      Objective:

      -To collect clinical data on patients and late complications after HCT

      Eligibility:

        -  Patients who underwent HCT for any indication and are surviving one year or more from
           the date of HCT OR patients who will be undergoing HCT on an ETIB protocol.

        -  Related stem cell donors of patients meeting the above criteria who participated or will
           be participating as a donor of stem cells or leukocytes.

        -  Age greater than or equal to 4 years

        -  Any active disease relapse or new hematologic malignancy including post-transplant
           lymphoproliferative disorder (PTLD) are excluded from protocol enrollment.

      Design:

        -  Prospective, longitudinal study of the natural history of long-term survivors after HCT.

        -  Clinical data on post-transplant complications and peripheral blood samples will be
           collected at yearly evaluations by the Principal Investigator, LAI, AI or representative
           investigator of the original HCT protocol.

        -  If patient develops complications such as new cancers, relapsed disease, new hematologic
           malignancy or PTLD they are exempt from required evaluations, and followed for survival
           and causes of death (management of complications as per primary team).

        -  As this is a longitudinal registry study without any planned intervention, there is no
           formal statistical analysis plan or accrual goal; descriptive data will be reported.
observational model: Cohort
time perspective: Prospective
measure: Basic characteristics of the patient population
time frame: at death
description: Survival
measure: Provide a mechanism to ensure implementation of standard of care recommendations for post-transplant followup
time frame: Ongoing throughout study
description: Observation of adherence and effects of standard of care recommendations
measure: Obtain blood samples from stem cell transplant donors to study how biological factors related to the donor are associated with late complications
time frame: Ongoing throughout study
description: Studying donor samples for potential correlations to transplant recipient complications.
measure: Obtain blood and tissue samples to study and document immunologic reconstitution after stem cell transplant
time frame: Ongoing throughout study
description: Immune monitoring for analysis
measure: Monitoring of late effects after stem cell transplant for patients of ETIB protocols
time frame: Ongoing throughout study
description: Survival and observation of long-term outcomes.
arm group label: 1/Patients
description: Patients who underwent hematopoietic stem cell transplant for any indication (malignant or non-malignant).
arm group label: 2/Donors
description: Related stem cell donors of those in Patients cohort.
arm group label: 3/Parents of patients
description: Parents/guardians of minors enrolled in cohort 1
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 4 Years
maximum age: N/A
healthy volunteers: Accepts Healthy Volunteers
last name: Steven Z Pavletic, M.D.
role: Principal Investigator
affiliation: National Cancer Institute (NCI)
last name: Ashley E Carpenter
phone: (240) 760-6009
email: carpentera@mail.nih.gov
last name: Steven Z Pavletic, M.D.
phone: (240) 760-6174
email: sp326h@nih.gov
facility: 
status: Recruiting
contact: 
country: United States
url: https://clinicalstudies.info.nih.gov/cgi/detail.cgi?A_2017-C-0027.html
description: NIH Clinical Center Detailed Web Page
responsible party type: Sponsor
sharing ipd: Yes
ipd description: .All collected IPD will be shared.@@@@@@All IPD recorded in the medical record will be shared with intramural investigators upon request. In addition, all large scale genomic sequencing data will be shared with subscribers to dbGaP.
ipd info type: Study Protocol
ipd info type: Statistical Analysis Plan (SAP)
ipd info type: Informed Consent Form (ICF)
ipd time frame: Clinical data available during the study and indefinitely.@@@@@@Genomic data are available once genomic data are uploaded per protocol GDS plan for as long as database is active.
ipd access criteria: Data from this study may be requested by contacting the PI.@@@@@@Genomic data are made available via dbGaP through requests to the data custodians.