Acquired aplastic anemia

This trial tests whether using only blood stem cells (instead of blood stem cells plus bone marrow) for a half-matched transplant is safe and effective for severe aplastic anemia. 180 patients aged 18-40 will be randomly assigned to one of two graft sources. The main goal is to s…

This study tests a new way to do stem cell transplants for people with severe aplastic anemia or other bone marrow failure diseases. Doctors give a smaller number of donor white blood cells along with the stem cells to try to reduce serious side effects. Up to 120 patients aged 4…

This study tests whether a new cord blood stem cell product, Omidubicel, can improve transplant outcomes for people with severe aplastic anemia or a related bone marrow disorder. Participants receive a combination of stem cells from a family member and specially processed cord bl…

This study tests a stem cell transplant using blood stem cells from a half-matched family donor (haploidentical) for people with severe aplastic anemia, related MDS, or PNH. Participants receive chemotherapy and a single radiation dose before the transplant, plus a drug to preven…

This study tests a new combination of drugs (busulfan, fludarabine, cyclophosphamide, and r-ATG) as preparation for a stem cell transplant in people aged 40-60 with severe aplastic anemia. The goal is to see if this regimen helps the transplant succeed with fewer failures. The st…

This study provides unlicensed cord blood units for transplant to children and adults with blood cancers, immune disorders, and other serious diseases. The goal is to see how well patients recover their white blood cells after the transplant. By making more cord blood units avail…

This study tests a new type of stem cell transplant for children with severe blood disorders like sickle cell disease or aplastic anemia. The transplant uses stem cells from a parent or other half-matched family donor, which are specially processed to remove certain immune cells.…

This phase 2 trial tests a bone marrow transplant using lower doses of chemotherapy and radiation to prepare the body, along with a drug called cyclophosphamide after transplant to prevent graft-versus-host disease. It enrolls 60 people with severe aplastic anemia or related bone…

This study is for people with severe aplastic anemia, a rare condition where the bone marrow stops making enough blood cells, leading to serious infections and bleeding. It tests a bone marrow transplant from a half-matched family donor, followed by a drug to reduce complications…

This study tests whether adding memory immune cells (CD45RO) to a stem cell transplant can help children fight off dangerous viral and fungal infections after the procedure. The transplant uses donor stem cells that have been stripped of certain cells to prevent graft-versus-host…

This study tests whether starting two oral medications (cyclosporine and eltrombopag) earlier than usual is safe and feasible for people with severe aplastic anemia, a rare condition where the immune system attacks bone marrow. Participants aged 3 and older will take these pills …

This study tests a bone marrow transplant from a partially matched donor (like a parent or sibling) for people newly diagnosed with severe aplastic anemia, a condition where the bone marrow stops making enough blood cells. Participants receive chemotherapy and immune-suppressing …

This phase 2 trial tests a special stem cell transplant for people with inherited bone marrow failure disorders like Fanconi anemia. The transplant uses a technique to remove certain immune cells, aiming to reduce the need for long-term immune-suppressing drugs and lower infectio…

This study follows people with acquired or inherited bone marrow failure for up to 20 years to learn how the diseases and treatments affect the body over time. Participants, aged 2 and older, will have regular check-ups and tests every 1 to 3 years. The goal is to better understa…