Aplastic anemia

This trial tests a new, lower-intensity combination of chemotherapy and radiation before a donor stem cell transplant for people with high-risk blood cancers or aplastic anemia. The goal is to see if this approach is safe enough to allow the donor cells to take hold and cure the …

This study tests a pill called ruxolitinib for people whose immune system attacks their bone marrow, causing conditions like severe anemia. The drug aims to stop this attack and help the bone marrow recover. Participants take the pill twice daily for up to 6 months and are follow…

This phase 3 trial compares two standard treatments for severe aplastic anemia (SAA) in patients up to age 25 who lack a matched sibling donor: unrelated donor bone marrow transplant (URD BMT) versus immune suppressive therapy (IST). The study measures which approach better preve…

This early-phase trial tests whether adding specially grown umbilical cord stem cells to a standard bone marrow transplant can improve outcomes for people with severe aplastic anemia, a rare condition where the bone marrow stops making enough blood cells. About 40 participants ag…

This early-phase trial tests whether adding special immune cells called veto cells to a stem cell transplant can help donor cells grow in the patient without causing severe graft-versus-host disease (GVHD). The study includes 16 people with various blood cancers or bone marrow fa…

This study planned to test the drug hetrombopag in people with non-severe aplastic anemia, a condition where the bone marrow doesn't make enough blood cells. The goal was to see if the drug could safely raise platelet counts. However, the trial was withdrawn before enrolling any …

This study was designed to test whether combining cyclosporine with drugs that boost platelet production (eltrombopag, hetrombopag, or romiplostim) could help people with newly diagnosed severe aplastic anemia who need blood transfusions. The trial was withdrawn before enrolling …

This trial aimed to compare the safety and effectiveness of adding luspatercept to cyclosporine versus cyclosporine alone in adults newly diagnosed with non-severe aplastic anemia (NSAA). The goal was to see if the combination could improve blood cell counts better than the stand…

This study was designed to test whether romiplostim N01 could help people with aplastic anemia caused by chemotherapy or radiation therapy. The trial was withdrawn before enrolling any participants, so no results are available. It was planned as a small, early-stage study at a si…

This study was designed to test whether adding enarodustat to cyclosporine helps people with a type of aplastic anemia who need regular blood transfusions. The trial was withdrawn before enrolling any participants, so no results are available. It would have compared the combinati…

This study tests a stem cell transplant from a matched unrelated donor for people with advanced blood cancers or blood disorders. Patients get chemotherapy and radiation before the transplant to prepare their body, then take drugs to prevent the donor cells from attacking their o…

This phase 2 trial tests whether adding the drug ruxolitinib to standard care can prevent graft-versus-host disease (GVHD) in older adults with acquired aplastic anemia who receive a bone marrow transplant. GVHD is a common and serious complication where donor immune cells attack…

This study tests a stem cell transplant method for people with blood cancers like leukemia or lymphoma. The approach uses chemotherapy, total body radiation, and a drug called cyclophosphamide after the transplant to prevent graft-versus-host disease, where donor cells attack the…

This study tests whether adding eltrombopag to standard immunosuppressive therapy can improve blood cell counts in people with untreated severe aplastic anemia. Participants receive horse-ATG and cyclosporine plus eltrombopag for up to 6 months. The goal is to see if this combina…

This study tests a bone marrow transplant method that avoids harsh chemotherapy and radiation for people with dyskeratosis congenita, a genetic disorder causing bone marrow failure. The goal is to fix the blood system without worsening lung or liver disease or raising cancer risk…

This study tests a special treatment for red blood cells that aims to kill the Zika virus, making transfusions safer. About 692 people who need blood transfusions will receive either treated or standard blood. The goal is to see if the treated blood works just as well and is safe…

This study tests whether adding the drug abatacept to standard care can prevent graft-versus-host disease (GVHD) in children receiving stem cell transplants from unrelated donors. GVHD occurs when donor cells attack the patient's body, causing serious illness. The trial will enro…

This study tests a modified plan to prevent and treat cytomegalovirus (CMV) infection in people with blood cancers who have had a donor stem cell transplant. CMV is a common virus that can cause serious problems after transplant. The study involves 153 participants and looks at w…

This study tests whether the drug sirolimus can prevent severe aplastic anemia from coming back after patients stop taking cyclosporine. About 84 people aged 2 and older who have responded to initial treatment will be randomly assigned to either take sirolimus for 3 months or jus…

This study is testing the safety of a drug called ATGAM in one patient with moderate to severe aplastic anemia, a condition where the bone marrow doesn't make enough blood cells. The patient receives ATGAM by IV for 4 days and is monitored for 24 weeks for side effects. The goal …

This study tests a stem cell transplant method that removes certain immune cells from the donor's blood to lower the chance of graft-versus-host disease (GvHD), a serious complication. It involves 3 patients with blood cancers or non-cancerous blood disorders who lack a perfectly…

This study tests whether adding methadone to usual morphine-based pain medicine reduces severe mouth pain in children aged 6–18 who are getting a stem cell transplant for leukemia, aplastic anemia, or similar conditions. The mouth pain is a common side effect of the chemotherapy …

This study aims to understand the genetic and molecular changes in people with bone marrow failure disorders. Researchers will analyze blood and bone marrow samples from up to 1,400 participants to track how these changes evolve over time. The goal is to better predict disease ou…

This study was designed to see if a new monitoring algorithm could detect early signs of ovarian damage in women and girls who had cancer treatment. Researchers planned to compare a group using the new algorithm with a group that received standard care. However, the study was wit…

This study follows 36 patients with aplastic anemia who previously received romiplostim in earlier trials. Researchers will monitor blood cell counts and safety for up to 5 years to see if benefits last and to check for any new health issues. The goal is to better understand the …