Actively Recruiting
Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels
Led by National Heart, Lung, and Blood Institute (NHLBI) · Updated on 2026-02-05
32
Participants Needed
1
Research Sites
944 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Background: Sickle cell disease can often be treated with blood stem cell transplants. But for some people the disease returns. This study will give a second transplant to people whose disease has returned but still have some donor cells in their body. Objective: To cure people s sickle cell disease by giving a second treatment that makes more room in their bone marrow for donor cells. Eligibility: People ages 4 and older with sickle cell disease who had a transplant but the disease returned, and their donor relatives. Donors can be 2 years of age or older. Design: Participants will be screened with medical history, physical exam, and blood tests. Recipients will also be screened with heart and breathing tests, x-rays, a bone marrow sample, and teeth and eye exams. They must have a caregiver. Donors will have 7-8 visits. They will take a drug for 5-6 days to prepare them for the donation. For the donation, blood is taken from a vein in the arm or groin. The stem cells are collected. The rest of the blood is returned. This may be repeated. Recipients will get a long IV line in their arm or chest for about 1-2 months. They will take drugs to help their body accept the donor cells. They will get the donor cells and red blood cell transfusions through the line. They will stay in the hospital about 30 days after the transfusion of donor cells. In first 3 months after the infusion, recipients will have many visits. Then they will have visits every 6 months to 1 year for 5 years. During those visits they will repeat some of the screening tests....
CONDITIONS
Official Title
Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patient with a history of sickle cell disease or beta-thalassemia who previously had allogeneic stem cell transplant
- Patient with recurrent sickle cell disease or beta-thalassemia showing clinical symptoms or specific lab criteria
- Persistent donor chimerism levels
- Age 4 years or older
- Negative pregnancy test (beta-HCG)
- Heart ejection fraction 35% or higher
- Lung diffusion capacity (DLCO) 35% or higher (may be waived in young children or those unable to perform test)
- Donor must be related to original transplant recipient, eligible, willing to donate, and age 2 to under 80 years
You will not qualify if you...
- Performance status of 3 or higher or Lansky score 50 or lower if under 16 years old
- Uncontrolled infections within one month before consent
- Current fever or suspected minor infection until symptoms resolve
- Major illness or organ failure that would prevent survival after transplant
- Pregnant or breastfeeding
- History of secondary cancers except localized skin cancer
- Donor-specific HLA antibodies with mean fluorescent intensity 2000 or higher
- No exclusion criteria listed for donors
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
Research Team
J
Julia M Varga, R.N.
CONTACT
C
Courtney F Joseph, M.D.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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