Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels
Recruiting now · Phase 1/Phase 2
Conditions studied: Myeloid Chimerism
In brief
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....
Key facts
- Study ID
- NCT04008368
- Run by
- National Heart, Lung, and Blood Institute (NHLBI)
- People needed
- 32
- Starts
- 2019-10-24
- Expected to finish
- 2037-12-01
- Last updated by the study team
- 2026-02-05
Who can join
Age: 2 and older, up to 80. Sex: any. Healthy volunteers: accepted.
You may not qualify if…
- Exclusion criteria- recipient
- ECOG performance status of 3 or more or if <16 years of age Lansky score of 50 or lower.
- Evidence of uncontrolled bacterial, viral, or fungal infections (currently taking medication and progression of clinical symptoms) within one month prior to signing the consent
- Patients with fever or suspected minor infection should await resolution of symptoms before signing the consent
- Major anticipated illness or organ failure incompatible with survival from PBSC transplant
- Pregnant or breastfeeding
- History of secondary malignancies (other than localized skin cancer)
- Donor-specific HLA antibodies with mean fluorescent intensity >= 2,000
- Exclusion- donor
- None
Where it is running
- National Institutes of Health Clinical Center — Bethesda, Maryland, United States (enrolling)
Full record on ClinicalTrials.gov
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