Pilot Trial of Allogeneic Blood or Marrow Transplantation for Primary Immunodeficiencies
Status:
Recruiting
Trial end date:
2028-12-21
Target enrollment:
Participant gender:
Summary
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.