NCT03000244 · National Cancer Institute (NCI)
Long-Term Follow up of Patients Undergoing Hematopoietic Stem Cell Transplantation, Cellular Therapy, or Gene Therapy
What this study is about
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.
View original scientific description
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.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- FOR PATIENT SUBJECTS:
- 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.
Exclusion criteria
- FOR PATIENT SUBJECTS: -Individuals 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 hematopoietic progenitor and stem cells or leukocytes
- Age \>= 4years
- 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
Where
- Bethesda, Maryland
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jul 9, 2026 · Source of record for eligibility and locations