Allogeneic Blood or Bone Marrow Transplant for T-Cell Immunodeficiency Disorders
Trial Summary
What is the purpose of this trial?
Background: Blood stem cells in the bone marrow make all the cells to normally defend a body against disease. Allogeneic blood or marrow transplant is when these stem cells are transferred from one person to another. Researchers think this treatment can provide a new, healthy immune system to correct T-cell problems in some people. Objective: To see if allogeneic blood or bone marrow transplant is safe and effective in treating people with T-cell problems. Eligibility: Donors: Healthy people ages 4 and older Recipients: People the same age with abnormal T-cell function causing health problems Design: All participants will be screened with: * Medical history * Physical exam * Blood, heart, and urine tests Donors will also have an electrocardiogram and chest x-ray. They may have veins tested or a pre-anesthesia test. Recipients will also have lung tests. Some participants will have scans and/or bone marrow collected by needle in the hip bones. Donors will learn about medicines and activities to avoid and repeat some screening tests. Some donors will stay in the hospital overnight and have bone marrow collected with anesthesia. Other donors will get shots for several days to stimulate cells. They will have blood removed by plastic tube (IV) in an arm vein. A machine will remove stem cells and return the rest of the blood to the other arm. Recipients will have: * More bone marrow and a small fragment of bone removed * Dental, diet, and social worker consultations * Scans * Chemotherapy and antibody therapy for 2 weeks * Catheter inserted in a chest or neck vein to receive donor stem cells * A hospital stay for several weeks with more medicines and procedures * Multiple follow-up visits
Do I need to stop my current medications for this trial?
The trial protocol does not specify if you need to stop taking your current medications. However, if you are receiving standard therapy for your underlying disorder, you may continue it as a bridge to the transplant. It's best to discuss your specific medications with the trial team.
What data supports the idea that Allogeneic Blood or Bone Marrow Transplant for T-Cell Immunodeficiency Disorders is an effective treatment?
The available research shows that Allogeneic Blood or Bone Marrow Transplant is an effective treatment for T-Cell Immunodeficiency Disorders. It has been shown to be curative for both well-known and newly discovered immunodeficiencies. The treatment is especially successful when a matched donor is available, but even when a perfect match isn't possible, using a partially matched donor can still be beneficial. This treatment is preferred for several immune disorders because it can restore the immune system's function, which is crucial for patients with these conditions.12345
What safety data is available for allogeneic blood or bone marrow transplant in T-cell immunodeficiency disorders?
Allogeneic hematopoietic stem cell transplantation (HSCT) is associated with significant risks, including delayed immune reconstitution and graft-versus-host disease (GVHD). The treatment has been studied in various conditions, such as autoimmune diseases, aplastic anemia, and chronic myelogenous leukemia, showing potential for sustained remissions but also significant morbidity and mortality. Early complications occur within the first 100 days, while late complications arise after 100 days post-transplant. The risks may outweigh the benefits, and careful prospective studies are needed to better define target populations and optimize the technology used.26789
Is Allogeneic HSC a promising treatment for T-Cell Immunodeficiency Disorders?
Yes, Allogeneic HSC is a promising treatment for T-Cell Immunodeficiency Disorders. It has been shown to be effective in curing these disorders, especially when a matched donor is available. Even when a perfect match isn't possible, alternative donor options can still lead to successful outcomes. This treatment helps rebuild the immune system, which is crucial for patients with severe T-cell immunodeficiencies.123410
Research Team
Dimana Dimitrova, M.D.
Principal Investigator
National Cancer Institute (NCI)
Eligibility Criteria
This trial is for people aged 4 and older with T-cell disorders, such as immune deficiencies or lymphoproliferative diseases. Participants must have certain medical conditions like abnormal T-cell function or specific genetic mutations affecting the immune system. They need to be in a stable condition to undergo transplant procedures and agree to use contraception if of childbearing potential.Inclusion Criteria
Exclusion Criteria
Treatment Details
Interventions
- Allogeneic HSC (Cell Therapy)
Allogeneic HSC is already approved in Canada, Japan for the following indications:
- Acute leukemia
- Chronic leukemia
- Hodgkin's lymphoma
- Non-Hodgkin's lymphoma
- Multiple myeloma
- Myelodysplastic syndromes
- Myeloproliferative neoplasms
- Acute myeloid leukemia
- Acute lymphoblastic leukemia
- Chronic myeloid leukemia
- Hodgkin's lymphoma
- Non-Hodgkin's lymphoma
- Multiple myeloma
- Myelodysplastic syndromes
- Myeloproliferative neoplasms
Find a Clinic Near You
Who Is Running the Clinical Trial?
National Cancer Institute (NCI)
Lead Sponsor