Cellular Immunotherapy, AdoptiveNCT03000244
Long-Term Follow up of Patients Undergoing Hematopoietic Stem Cell Transplantation, Cellular Therapy, or Gene Therapy
An observational study of Cellular Immunotherapy, Adoptive and Genetic Therapy, run by National Cancer Institute (NCI) at 1 site in United States.
Trial summary
Background: People who have had an allogeneic hematopoietic stem cell transplant (HCT), cellular therapy (CT), or gene therapy (GT) have bone marrow or an immune system that is damaged. They get stem cells from a donor who is a relative. Read more about this study
- Who is it for?
- Ages 4 to 120 yearsHealthy volunteers can take part
- Conditions the study is for
- Cellular Immunotherapy, Adoptive, Genetic Therapy, Tissue Donors
- How many people
- 2,000 participants
- Where
- 1 siteUnited States
- Study dates
- 26 Apr 2017to 12 Aug 2050
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 120 years
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: Patients who have received an allogeneic stem cell transplant, cellular therapy, or gene therapy as well as donors of stem cells who are related to the patients who are study participants. Parents of minor recipients may also be enrolled to complete surveys. Participants can be from any community or co-enrolled on another NIH protocol.
Who may be able to join
- Individuals who underwent HCT or received cellular or gene therapy for any indication (malignant or non-malignant) and are surviving one year or more from the date of therapy
- Age >= 4 years
- Ability of individual or individual s Legally Authorized Representative (LAR) to understand and the willingness to sign a written informed consent document
- Individuals will need to have a primary physician within the US (primary care, oncologist, hematologist, etc.) that will provide continued comprehensive care for the duration of participation in the study. PI may allow for established medical providers to be located outside of the US.
- Related stem cell donors of patients meeting the above criteria as a donor of hematopoietic progenitor and stem cells or leukocytes
- Age >= 4years
Show all 9Show fewer
- Ability of patient or patient s Legally Authorized Representative (LAR) to understand and the willingness to sign a written informed consent document
- Parents/guardians of minors enrolled on the study who have undergone HCT
- Willingness to complete surveys about the minor that underwent HCT
Who cannot join
- -Individuals with active disease relapse or new hematologic malignancy including post-transplant lymphoproliferative disorder (PTLD) are excluded from protocol enrollment.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
For more information at the NIH Clinical Center contact National Cancer Institute Referral Office, 888-624-1937
About this study
Background:
People who have had an allogeneic hematopoietic stem cell transplant (HCT), cellular therapy (CT), or gene therapy (GT) 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, CT, or GT. They want to learn how the stem cells change and how to improve their ability to fight cancer.
Read more
Objective:
To provide long-term follow-up care for people who underwent or will undergo HCT, CT, or GT. To collect data, blood, and tissue samples to learn about late complications after HCT, CT, or GT.
Eligibility:
Adults age 18 and older who will undergo HCT or underwent HCT, Cellular Therapy (CT), or Gene Therapy (GT) and are surviving one year or more from the date of therapy. 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.
Read the 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, cellular, or gene therapy for any indication and are surviving one year or more from the date of therapy.
- Related hematopoietic progenitor and stem cell donors of patients meeting the above criteria who participated or will be participating as a donor of stem cells or leukocytes, may undergo a one-time optional research blood sample collection. Donors will not remain on study for longitudinal follow-up.
- Age >= 4 years
- Recipients of HCT, cellular, or gene therapies for any indication.
- 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, cellular therapy, or gene therapy.
- 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.
- Conditions
- Cellular Immunotherapy, Adoptive, Genetic Therapy, Tissue Donors, Hematopoietic Stem Cell Transplantation
- Keywords
- Cellular Therapy, Gene Therapy, Natural History, HCT
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 1/Patients | Patients who underwent hematopoietic stem cell transplant or received cellular therapy or gene therapy for any indication (malignant or non-malignant). | |
| 2/Donors | Related stem cell donors of those in Patients cohort. | |
| 3/Parents of patients | Parents/guardians of minors enrolled in cohort 1 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Basic characteristics of the patient population
Survival
Measured: at death
Secondary outcomes (4)
Monitoring of late effects after stem cell transplant, cellular therapy, or gene therapy for patients of NIH transplantation and cellular therapy protocols
Survival and observation of long-term outcomes.
Measured: Ongoing throughout study
Obtain blood and tissue samples to study and document immunologic reconstitution after stem cell transplant
Immune monitoring for analysis
Measured: Ongoing throughout study
Obtain blood samples from stem cell transplant donors to study how biological factors related to the donor are associated with late complications
Studying donor samples for potential correlations to transplant recipient complications.
Measured: Ongoing throughout study
Provide a mechanism to ensure implementation of standard of care recommendations for post-transplant followup
Observation of adherence and effects of standard of care recommendations
Measured: Ongoing throughout study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Dec 2016 (estimated)
- Study start26 Apr 2017
- Primary completion12 Aug 2050 (estimated)
- Study completion12 Aug 2050 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cellular Immunotherapy, AdoptiveStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 2,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Najla El Jurdi, M.D., Principal investigator, National Cancer Institute (NCI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03000244
- Sponsor's protocol ID
- 170027
- Other IDs
- 17-C-0027
- Status last verified
- 13 Oct 2026
- First submitted
- 21 Dec 2016
- Last updated
- 26 Oct 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 26 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-Term Follow up of Patients Undergoing Hematopoietic Stem Cell Transplantation, Cellular Therapy, or Gene Therapy
- ClinicalTrials.gov ID
- NCT03000244
- Acronym / sponsor ID
- 170027
- Conditions
- Cellular Immunotherapy, Adoptive, Genetic Therapy, Tissue Donors, Hematopoietic Stem Cell Transplantation
- Keywords
- Cellular Therapy, Gene Therapy, Natural History, HCT
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 2,000 participants (estimated)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- 26 Apr 2017 (actual)
- Primary completion
- 12 Aug 2050 (estimated)
- Study completion
- 12 Aug 2050 (estimated)
- First posted
- 22 Dec 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Background:
People who have had an allogeneic hematopoietic stem cell transplant (HCT), cellular therapy (CT), or gene therapy (GT) 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, CT, or GT. 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, CT, or GT. To collect data, blood, and tissue samples to learn about late complications after HCT, CT, or GT.
Eligibility:
Adults age 18 and older who will undergo HCT or underwent HCT, Cellular Therapy (CT), or Gene Therapy (GT) and are surviving one year or more from the date of therapy. 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.
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, cellular, or gene therapy for any indication and are surviving one year or more from the date of therapy.
- Related hematopoietic progenitor and stem cell donors of patients meeting the above criteria who participated or will be participating as a donor of stem cells or leukocytes, may undergo a one-time optional research blood sample collection. Donors will not remain on study for longitudinal follow-up.
- Age >= 4 years
- Recipients of HCT, cellular, or gene therapies for any indication.
- 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, cellular therapy, or gene therapy.
- 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
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 1/Patients | – | –Patients who underwent hematopoietic stem cell transplant or received cellular therapy or gene therapy for any indication (malignant or non-malignant). |
| 2/Donors | – | –Related stem cell donors of those in Patients cohort. |
| 3/Parents of patients | – | –Parents/guardians of minors enrolled in cohort 1 |
Who may qualify
- Ages
- 4 to 120 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients who have received an allogeneic stem cell transplant, cellular therapy, or gene therapy as well as donors of stem cells who are related to the patients who are study participants. Parents of minor recipients may also be enrolled to complete surveys. Participants can be from any community or co-enrolled on another NIH protocol.
Key inclusion criteria (9 listed)
- Individuals who underwent HCT or received cellular or gene therapy for any indication (malignant or non-malignant) and are surviving one year or more from the date of therapy
- Age >= 4 years
- Ability of individual or individual s Legally Authorized Representative (LAR) to understand and the willingness to sign a written informed consent document
- Individuals will need to have a primary physician within the US (primary care, oncologist, hematologist, etc.) that will provide continued comprehensive care for the duration of participation in the study. PI may allow for established medical providers to be located outside of the US.
- Related stem cell donors of patients meeting the above criteria as a donor of hematopoietic progenitor and stem cells or leukocytes
Key exclusion criteria (1 listed)
- -Individuals with active disease relapse or new hematologic malignancy including post-transplant lymphoproliferative disorder (PTLD) are excluded from protocol enrollment.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Basic characteristics of the patient population
Time frame: at death
Survival
4 secondary outcomes
Monitoring of late effects after stem cell transplant, cellular therapy, or gene therapy for patients of NIH transplantation and cellular therapy protocols
Time frame: Ongoing throughout study
Obtain blood and tissue samples to study and document immunologic reconstitution after stem cell transplant
Time frame: Ongoing throughout study
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
Provide a mechanism to ensure implementation of standard of care recommendations for post-transplant followup
Time frame: Ongoing throughout study
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Oct 2026.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Najla El Jurdi, M.D. principal investigator
National Cancer Institute (NCI)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cellular Immunotherapy, Adoptive and Genetic Therapy, run by National Cancer Institute (NCI) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 26 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Danielle F Pinkiert, R.N. ((240) 858-7566, danielle.pinkiert@nih.gov), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Basic characteristics of the patient population (time frame: at death). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Oct 2026.