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Study identifier: NCT04339777 Synced from ClinicalTrials.gov · July 28, 2026
● Recruiting

Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of Immunity

Condition: Lymphoproliferative Disorders · Autoimmune Lymphoproliferative · Immune System Diseases  ·  Sponsor: National Cancer Institute (NCI)

PhasePhase 2
Planned participants66
Who can joinAll sexes, 4 Years to 69 Years
Healthy volunteersNo

About this study

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 …

This description comes directly from the study's public registry record.

Talk to the study team

Shannon L Knight, R.N.  ·  (240) 921-5872  ·  shannon.knight@nih.gov

Sung-Yun Pai, M.D.  ·  (240) 858-7284  ·  sung-yun.pai@nih.gov

Always discuss trial participation with your own doctor first.

Locations (1)

National Institutes of Health Clinical CenterBethesda, Maryland, United StatesRecruiting

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Source record: clinicaltrials.gov/study/NCT04339777