Bone marrow transplant using donor blood to treat sickle cell
Treatments studied
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This trial tests a specific type of bone marrow transplant for people with sickle cell disease (or certain other blood disorders) who have had serious complications despite standard care. It aims to see if removing the patient’s immune cells and using a matched donor’s cells can improve outcomes and reduce complications.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You (or your parent/guardian) can give written consent/assent to join the study.
- Your age is between 5 and 40 years at the time you agree to participate.
- You have sickle cell disease with serious past problems (for example frequent hospital-level pain crises or acute chest syndrome), or you have beta-thalassemia or Diamond-Blackfan anemia with frequent transfusions and iron overload.
- A suitable donor match must be found by special tissue typing (HLA): related donor at least a 4/8 match, or unrelated donor/cord blood search done but no better options available (and in some cases cord blood must meet a cell-dose limit).
- Your kidneys, liver, heart, and lungs must work well enough for transplant, and you must be medically cleared by the study team’s cardiology and lung specialists.
- You must be HIV-negative on a PCR test, and if you could become pregnant or get someone pregnant, you must use approved birth control during treatment and afterward.
- You must have tried and not benefited from hydroxyurea for sickle cell disease.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This trial tests a specialized donor stem cell transplant to treat high-risk problems in the blood and bone marrow, including some cancers and non-cancers. It may help by using donor immune cells designed to better control the disease after transplant.
This Phase 2 study tests a type of stem cell transplant that uses donor immune cells that have been specially “tweaked” to help treat sickle cell disease. It may help prevent serious sickle cell complications, but it requires careful matching and screening of organs and pregnancy status.
This early-phase study tests whether a “half-matched” donor bone marrow transplant, with special medicines to calm the immune system before transplant, can improve outcomes for people with sickle cell disease. It may help high-risk patients who have frequent complications and don’t have a fully matched donor.
This trial is testing a new, gentler type of bone marrow transplant for children and young adults with sickle cell disease who have a healthy sibling donor with a mismatched blood type or who have antibodies against their donor's red blood cells. The goal is to replace the faulty bone marrow with healthy donor cells, with fewer side effects than traditional transplants.
This study tests a way to do a bone marrow (stem cell) transplant from an HLA-matched sibling in children with sickle cell disease, aiming to lower the risk of serious side effects. You may be considered if you have certain past sickle complications and your body can safely handle the transplant plan.
This trial tests whether a stem cell transplant from a fully matched sibling donor can help people with serious blood disorders like sickle cell disease or thalassemia. It aims to replace the faulty blood system with a healthy one from the donor.
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