Long-Term Follow-up of People Undergoing Hematopoietic Stem Cell Transplantation

Sponsor
National Cancer Institute (NCI) (NIH)
Overall Status
Recruiting
CT.gov ID
NCT03000244
Collaborator
(none)
2,000
1
399.5
5

Study Details

Study Description

Brief Summary

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.

Condition or Disease Intervention/Treatment Phase

    Detailed Description

    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.

    Study Design

    Study Type:
    Observational
    Anticipated Enrollment :
    2000 participants
    Observational Model:
    Cohort
    Time Perspective:
    Prospective
    Official Title:
    Long-Term Follow up of Patients Undergoing Hematopoietic Stem Cell Transplantation
    Actual Study Start Date :
    Apr 26, 2017
    Anticipated Primary Completion Date :
    Aug 12, 2050
    Anticipated Study Completion Date :
    Aug 12, 2050

    Arms and Interventions

    Arm Intervention/Treatment
    1/Patients

    Patients who underwent hematopoietic stem cell transplant for any indication (malignant ornon-malignant).

    2/Donors

    Related stem cell donors of those in Patients cohort.

    3/Parents of patients

    Parents/guardians of minors enrolled in cohort 1

    Outcome Measures

    Primary Outcome Measures

    1. Basic characteristics of the patient population. [At death.]

      Survival

    Secondary Outcome Measures

    1. Monitoring of late effects after stem cell transplant for patients of ETIB protocols [Ongoing throughout study]

      Survival and observation of long-term outcomes.

    2. Provide a mechanism to ensure implementation of standard of carerecommendations for post-transplant followup [Ongoing throughout study]

      Observation of adherence and effects of standard of carerecommendations

    3. Obtain blood and tissue samples to study and document immunologicreconstitution after stem cell transplant [Ongoing throughout study]

      Immune monitoring for analysis

    4. Obtain blood samples from stem cell transplant donors to study howbiological factors related to the donor are associated with latecomplications [Ongoing throughout study]

      Studying donor samples for potential correlations to transplantrecipient complications.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    4 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    • INCLUSION CRITERIA FOR PATIENT SUBJECTS:

    • Patients who underwent HCT for any indication (malignant or non-malignant) and are surviving one year or more from the date of HCT (patients may be at any time point after HCT as long as it is greater than or equal to 1 year) OR patients who will be undergoing HCT for any indication (malignant or non-malignant) on an ETIB protocol. Patients do not need to have been transplanted under an ETIB protocol.

    • Age greater than or equal to 4 years

    • Ability of patient or patient s Legally Authorized Representative (LAR) to understand and the willingness to sign a written informed consent document

    • Patients will need to have a primary care physician that will provide continued comprehensive care throughout the patient s participation in the study.

    EXCLUSION CRITERIA FOR PATIENT SUBJECTS:

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

    INCLUSION CRITERIA FOR DONOR SUBJECTS:
    • Related stem cell donors of patients meeting the above criteria as a donor of stem cells or leukocytes

    • Age greater than or equal to 4 years

    • Ability of patient or patient s Legally Authorized Representative (LAR) to understand and the willingness to sign a written informed

    consent document

    INCLUSION CRITERIA FOR PARENTS COMPLETING SURVEYS:
    • Parents/guardians of minors enrolled on the study who have undergone HCT

    • Willingness to complete surveys about the minor that underwent HCT

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 National Institutes of Health Clinical Center Bethesda Maryland United States 20892

    Sponsors and Collaborators

    • National Cancer Institute (NCI)

    Investigators

    • Principal Investigator: Steven Z Pavletic, M.D., National Cancer Institute (NCI)

    Study Documents (Full-Text)

    None provided.

    More Information

    Additional Information:

    Publications

    None provided.
    Responsible Party:
    National Cancer Institute (NCI)
    ClinicalTrials.gov Identifier:
    NCT03000244
    Other Study ID Numbers:
    • 170027
    • 17-C-0027
    First Posted:
    Dec 22, 2016
    Last Update Posted:
    Aug 25, 2022
    Last Verified:
    Aug 3, 2022
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by National Cancer Institute (NCI)

    Study Results

    No Results Posted as of Aug 25, 2022