Aplastic anemia

Researchers are testing a conditioning regimen built around thiotepa before a haploidentical, or half-matched, stem cell transplant in people aged 50 and older with severe aplastic anemia. The regimen also includes busulfan, fludarabine, anti-thymocyte globulin, and basiliximab, …

This trial tests whether the antioxidant N-acetylcysteine (NAC) can help platelets recover faster after a stem cell transplant in people with severe aplastic anemia. Participants take NAC capsules three times daily from two weeks before transplant until two months after. The stud…

This trial tests a combination of sirolimus, ruxolitinib, and mycophenolate mofetil to prevent acute graft-versus-host disease (aGVHD) in patients receiving a half-matched stem cell transplant. The study focuses on patients with blood cancers or severe aplastic anemia who cannot …

This trial tests an oral drug called avatrombopag in adults with aplastic anemia, a rare condition where the bone marrow fails to make enough blood cells. The study enrolls about 26 people whose disease has not improved with standard treatment, who cannot receive standard treatme…

This study tests an experimental drug called dibotatug (DR-01) in adults with bone marrow failure syndromes, including severe aplastic anemia. The goal is to see if the drug is safe and can improve blood cell counts. Participants receive dibotatug by IV infusion, and researchers …

This study tests whether a combination of dexamethasone, intravenous immunoglobulin (IVIG), and extra donor immune cells can safely lower harmful antibodies that attack donor stem cells in half-matched (haploidentical) transplants. The trial enrolls 60 adults aged 18–65 with bloo…

This study tests a drug called romiplostim N01 in 43 adults whose bone marrow has stopped making enough blood cells after chemotherapy or radiation. The goal is to see if the drug can improve blood counts and reduce the need for transfusions. Participants must have tried other tr…

This phase 2 trial tests a combination of three drugs—cyclosporine, a thrombopoietin receptor agonist (eltrombopag or hetrombopag), and romiplostim—in 43 adults newly diagnosed with severe aplastic anemia or transfusion-dependent non-severe aplastic anemia. The goal is to see if …

This study tests whether a single injection of goserelin, given just before a stem cell transplant, can help preserve ovarian function in women with severe aplastic anemia. The trial will enroll 132 women aged 14–50 and compare menstrual recovery rates six months after transplant…

This Phase 3 trial tests whether adding enarodustat to standard cyclosporine treatment helps people with transfusion-dependent non-severe aplastic anemia. The study will enroll 90 newly diagnosed adults who cannot have a stem cell transplant. The goal is to see if the combination…

This study tests a new treatment called CK0801 for adults with aplastic anemia who still need blood transfusions after standard therapy. CK0801 uses immune cells from donated cord blood to help the body make its own blood cells again. The trial will check if the treatment is safe…

This Phase 3 trial tests whether adding romiplostim (a platelet-boosting drug) to standard immunosuppressive therapy helps people with severe aplastic anemia achieve normal blood cell counts. About 210 participants will receive either romiplostim or a placebo alongside standard t…

This study compares two first-line treatments for severe aplastic anemia, a condition where the bone marrow stops making enough blood cells. One treatment is a stem cell transplant from a half-matched donor, and the other is drug therapy that suppresses the immune system. The stu…

This study tests a new drug called golidocitinib, combined with strong immune-suppressing therapy, for people with severe aplastic anemia—a condition where the bone marrow stops making enough blood cells. The trial has two parts: first, it checks safety in patients who didn't imp…

This phase II trial tests whether adding romiplostim, a drug that stimulates platelet production, to standard immunosuppressive therapy can improve blood cell counts in children and young adults with severe aplastic anemia. The study will enroll 15 participants aged 2 to 21 who a…

This study tests daratumumab, a drug that targets certain immune cells, in 37 people with aplastic anemia that hasn't improved with standard treatments. The first part checks safety and the right dose, and the second part looks at whether it can improve blood cell counts. The goa…