Bone Marrow Transplant for Immune Deficiency
Trial Summary
Will I have to stop taking my current medications?
The trial information does not specify if you need to stop taking your current medications. However, since chemotherapy is involved, it's possible that some medications might need to be adjusted. Please discuss this with the trial team for specific guidance.
What data supports the effectiveness of the treatment Allo BMT for immune deficiency?
Allogeneic bone marrow transplantation (Allo BMT) is considered an effective treatment for various immune deficiency disorders, as it is the only cure for many primary immune deficiency disorders and inherited bone marrow failure syndromes. Studies have shown that it is also effective for severe combined immunodeficiency (SCID), with a high survival rate in patients receiving HLA-identical transplants.12345
Is allogeneic bone marrow transplantation generally safe for humans?
Allogeneic bone marrow transplantation (BMT) can be risky, with potential complications like graft failure, graft-versus-host disease (GVHD), and infections. Long-term effects may include lung problems, cataracts, and other health issues. While it can be life-saving for certain conditions, it is associated with significant risks and is usually done in specialized centers.16789
How is the treatment Allo BMT different from other treatments for immune deficiency?
Allo BMT is unique because it involves transplanting healthy bone marrow from a donor to replace the patient's faulty immune system, offering a potential cure for many immune deficiencies. Unlike other treatments that may only manage symptoms, this approach aims to restore normal immune function by using donor stem cells.210111213
What is the purpose of this trial?
Background:Allogeneic blood or marrow transplant is when stem cells are taken from one person s blood or bone marrow and given to another person. Researchers think this may help people with immune system problems.Objective:To see if allogeneic blood or bone marrow transplant is safe and effective in treating people with primary immunodeficiencies.Eligibility:Donors: Healthy people ages 4 or olderRecipients: People ages 4-75 with a primary immunodeficiency that may be treated with allogeneic blood or marrow transplantDesign:Participants will be screened with medical history, physical exam, and blood tests.Participants will have urine tests, EKG, and chest x-ray.Donors will have:Bone marrow harvest: With anesthesia, marrow is taken by a needle in the hipbone.ORBlood collection: They will have several drug injections over 5-7 days. Blood is taken by IV in one arm, circulates through a machine to remove stem cells, and returned by IV in the other arm.Possible vein assessment or pre-anesthesia evaluationRecipients will have:Lung test, heart tests, radiology scans, CT scans, and dental examPossible tissue biopsies or lumbar punctureBone marrow and a small piece of bone removed by needle in the hipbone.Chemotherapy 1-2 weeks before transplant dayDonor stem cell donation through a catheter put into a vein in the chest or neckSeveral-week hospital stay. They will take medications and may need blood transfusions and additional procedures.After discharge, recipients will:Remain near the clinic for about 3 months. They will have weekly visits and may require hospital readmission.Have multiple follow-up visits to the clinic in the first 6 months, and less frequently for at least 5 years.
Research Team
Dimana Dimitrova, M.D.
Principal Investigator
National Cancer Institute (NCI)
Eligibility Criteria
This trial is for people aged 4-75 with primary immunodeficiencies that could be treated by a blood or marrow transplant. Donors must be healthy and at least 4 years old. Participants need to have certain immune system issues, like severe infections or autoimmune diseases, and good organ function. Pregnant women can't join, and participants must agree to use contraception for one year post-transplant.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Pre-transplant Conditioning
Recipients undergo chemotherapy 1-2 weeks before transplant day
Transplantation
Donor stem cell donation and transplantation procedure
Post-transplant Hospitalization
Several-week hospital stay for monitoring and treatment post-transplant
Initial Follow-up
Participants remain near the clinic for about 3 months with weekly visits
Long-term Follow-up
Multiple follow-up visits to the clinic in the first 6 months, and less frequently for at least 5 years
Treatment Details
Interventions
- Allo BMT
Allo BMT is already approved in European Union, United States, Canada for the following indications:
- Primary Immunodeficiencies
- Severe Combined Immunodeficiency
- Wiskott-Aldrich Syndrome
- X-linked Lymphoproliferative Syndrome
- Primary Immunodeficiencies
- Severe Combined Immunodeficiency
- Wiskott-Aldrich Syndrome
- X-linked Lymphoproliferative Syndrome
- Chronic Granulomatous Disease
- Primary Immunodeficiencies
- Severe Combined Immunodeficiency
- Wiskott-Aldrich Syndrome
- X-linked Lymphoproliferative Syndrome
Find a Clinic Near You
Who Is Running the Clinical Trial?
National Cancer Institute (NCI)
Lead Sponsor