Actively Recruiting
Pilot Trial of Allogeneic Blood or Marrow Transplantation for Primary Immunodeficiencies
Led by National Cancer Institute (NCI) · Updated on 2026-05-11
354
Participants Needed
2
Research Sites
1101 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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 older Recipients: People ages 4-75 with a primary immunodeficiency that may be treated with allogeneic blood or marrow transplant Design: 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. OR Blood 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 evaluation Recipients will have: Lung test, heart tests, radiology scans, CT scans, and dental exam Possible tissue biopsies or lumbar puncture Bone marrow and a small piece of bone removed by needle in the hipbone. Chemotherapy 1-2 weeks before transplant day Donor stem cell donation through a catheter put into a vein in the chest or neck Several-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.
CONDITIONS
Official Title
Pilot Trial of Allogeneic Blood or Marrow Transplantation for Primary Immunodeficiencies
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients aged 4 to 75 years
- Primary immunodeficiency diagnosed by genetic test or clinical history
- Clinical history includes at least two of: life-threatening infection, recurrent infections, opportunistic infection, chronic viral elevation, immune dysregulation, hypogammaglobulinemia, hematologic malignancy, or virus-associated tumor
- Availability of at least one suitable HLA-matched or haploidentical donor
- Adequate organ function including heart, lung, liver, and kidney
- Performance status of 60% or higher (Karnofsky or Lansky) or ECOG 2 or less
- Ability to understand and sign informed consent
- Not pregnant or breastfeeding; use contraception for at least one year post-transplant
- Patients with malignancy must be in remission or evaluated appropriately
You will not qualify if you...
- Receiving other investigational agents except virus-specific cytotoxic T-cells
- Allergic reactions to study drugs (cyclophosphamide, busulfan, pentostatin, sirolimus, MMF, filgrastim)
- Active psychiatric disorder affecting protocol compliance or consent
- Active central nervous system malignancy except some virus-associated cases
- MAGT1 mutation requiring anticoagulation that cannot be interrupted
- HIV positive or other acquired immunodeficiency interfering with assessment
- Inadequate central venous access
- Unrelated donors failing to meet National Marrow Donor Program standards
AI-Screening
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Trial Site Locations
Total: 2 locations
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
2
National Marrow Donor Program
Minneapolis, Minnesota, United States, 55413-1753
Actively Recruiting
Research Team
A
Amy H Chai
CONTACT
D
Dimana Dimitrova, M.D.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
6
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