Allogeneic Hematopoietic Stem Cell Transplant for People With Primary Immunodeficiency Diseases
Status:
Recruiting
Trial end date:
2025-11-30
Target enrollment:
Participant gender:
Summary
Background:
During a transplant, blood stem cells from one person are given to someone else. The cells
grow into the different cells that make up the immune system. This can cure people with
certain immunodeficiencies. But transplant has many risks and complications.
Objective:
To see if stem cell transplant can be successfully performed in people with primary
immunodeficiency disease and cure them.
Eligibility:
People ages 4-69 for whom a primary immunodeficiency (PID) or Primary Immune Regulatory
Disorder (PIRD), has caused significant health problems and either standard management has
not worked or there are no standard management options, along with their donors
Design:
Donors will be screened under protocol 01-C-0129. They will donate blood or bone marrow.
Participants will be screened with:
Medical history
Physical exam
Blood, urine, and heart tests
CT or PET scans
Before transplant, participants will have dental and eye exams. They will have a bone marrow
biopsy. For this, a needle will be inserted through the skin into the pelvis to remove
marrow.
Participants will be hospitalized before their transplant. They will have a central catheter
put into a vein in their chest or neck. They will get medications through the catheter to
prevent complications. Participants will get stem cells through the catheter. They will stay
in the hospital for at least 4 weeks. They will give blood, urine, bone marrow, and stool
samples. They may need blood transfusions. They may need more scans. They will take more
medications.
Participants will have visits on days 30, 60, 100, 180, and 360, and 24 months after the
transplant. Then they will have visits once a year for about 5 years