Aplastic anemia

Researchers are testing a stem cell transplant from a half-matched family member in children and young adults with severe aplastic anemia, a condition where the bone marrow stops making enough blood cells. Participants receive chemotherapy and radiation to prepare their body, the…

This study tests an oral drug called pacritinib in people aged 12 and older with myelodysplastic syndromes (MDS) or related blood cancers (MDS/MPN). The goal is to find the best dose and see if it can shrink or control the disease. Participants take capsules twice daily for up to…

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 trial tests isatuximab, an antibody that targets CD38, in adults with refractory severe aplastic anemia—a condition where the bone marrow fails to produce enough blood cells. Participants receive weekly infusions for six weeks. The study aims to see if the drug is safe and c…

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 combination of horse anti-thymocyte globulin, cyclosporine, steroids, and growth factors in 140 adults with aplastic anemia or low-risk myelodysplastic syndrome (MDS). The goal is to see if this immune-suppressing and blood-cell-boosting approach can im…

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 tests a stem cell transplant method for adults aged 40 to 60 with severe aplastic anemia that has not improved or has returned after standard drug therapy. The approach uses a special drug after transplant to lower the risk of graft-versus-host disease, a common and se…

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 program offers eltrombopag to patients with serious or life-threatening thrombocytopenia (low platelet counts) who have no other treatment options and cannot join a clinical trial. A licensed physician must request the drug, and the potential benefit must outweigh the risks.…

This study tests whether a once-weekly injection (romiplostim) can help platelets recover faster and more safely than a daily injection (rhTPO) after a donor stem cell transplant. It includes 66 adults with MDS or severe aplastic anemia. The goal is to reduce bleeding risk and tr…

This study tests whether adding umbilical cord blood to a half-matched stem cell transplant can help people with aplastic anemia, a serious condition where the bone marrow stops making enough blood cells. About 224 participants aged 14 and older will receive either the combined t…

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 is tracking 10 children aged 6 to 17 with aplastic anemia, a condition where the bone marrow stops making enough blood cells. The children are receiving eltrombopag (Revolade) for the first time, combined with anti-thymocyte globulin. Researchers are monitoring for ser…

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 study is observing how well a double umbilical cord blood transplant works for 40 adults with blood cancers like leukemia or lymphoma. The transplant uses two units of cord blood from unrelated donors to help rebuild the patient's blood system. Researchers are tracking survi…

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 early-stage trial tests a new way to do a stem cell transplant for people with severe aplastic anemia whose disease has come back or not improved with standard treatments. The approach uses a milder conditioning regimen and removes certain immune cells from the donor cells t…

This study tests whether giving emapalumab early can help children with newly diagnosed severe aplastic anemia recover their blood cell counts within 6 weeks. About 35 children will receive the drug before standard treatments. The goal is to see if this approach improves treatmen…

This study tests whether a tailored exercise program combined with a dietary supplement called nicotinamide riboside (NR) can improve muscle mass, strength, and fitness in adolescents and young adults who had a bone marrow transplant for leukemia or other blood disorders. About 8…

This program offers extra stem cells from a half-matched family donor to patients who are getting a cord blood transplant for serious blood cancers or immune disorders. The goal is to help the bone marrow recover more quickly while the cord blood cells take over permanently. It i…

This study tests a stem cell transplant approach for people with Fanconi Anemia who don't have a perfectly matched donor. The goal is to see if using lower doses of chemotherapy before the transplant can reduce serious side effects. About 70 participants will receive a transplant…

This phase 2 trial tests a two-step, lower-intensity transplant method for people with various blood cancers. The goal is to see if this gentler approach reduces the chance of dying from the transplant itself. About 63 participants will receive a combination of radiation, chemoth…

This study is testing whether umbilical cord blood stem cells from unrelated donors can safely and effectively restore blood cell production in people with serious conditions like leukemia, immune deficiencies, and inherited blood disorders. The main goal is to see how quickly th…

This study tests a specific order of antibiotics for cancer patients who develop fever due to low white blood cells (febrile neutropenia). Twenty adults with blood cancers will receive a carbapenem first, then add vancomycin or linezolid if fever persists, and finally an antifung…

This early-phase study tests whether Darzalex Faspro can safely lower donor-specific antibodies (DSAs) in 8 adults with blood cancers or bone marrow failure who are at high risk of rejecting a stem cell transplant. The goal is to reduce DSA levels enough to allow the transplant t…

This study tests whether a special cooling cap can prevent hair loss in children receiving chemotherapy for non-cancerous conditions or solid tumors. The cap is worn before, during, and after chemo to keep the scalp cool. Researchers want to see if it is safe and tolerable for 40…

This study uses a non-invasive brain imaging technique called fNIRS to see how anemia affects thinking and memory. Researchers will measure brain activity in 323 adults with different types of anemia and compare it to cognitive test scores. The goal is to identify which brain reg…

This natural history study follows up to 4,000 people with inherited bone marrow failure syndromes (IBMFS) and their families to learn why they are prone to certain cancers. Researchers will track health over time, collect genetic samples, and look for clues that separate those w…

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…

This study collects blood samples and health information from 375 people with non-cancerous blood disorders like aplastic anemia, sickle cell disease, or thalassemia who are receiving a stem cell transplant or gene therapy. The goal is to create a biobank that helps researchers u…

This study tests a new communication tool called 'Let's Get REAL' that helps children and teens (ages 8-17) and their families talk together about stem cell transplant or cellular therapy decisions. The goal is to see if the tool is easy to use and helpful for families. About 60 …

This research study collects blood, bone marrow, and tissue samples from people with cancer, blood disorders, and healthy volunteers. The goal is to study how the immune system changes during different treatments, including immunotherapy and chemotherapy. By comparing samples fro…

This study watches 200 people with aplastic anemia who are getting ATG/ALG treatment for the first time. Researchers will track side effects like serum sickness, organ damage, and infusion reactions to learn how common and severe they are. The goal is to create better guidelines …

This study is creating a large database of people in China with various blood diseases, such as leukemia, multiple myeloma, and hemophilia. Researchers will collect information from medical records and follow participants over time to learn about disease patterns, treatments, and…

This study aims to create a prediction tool for pregnant women with aplastic anemia, a rare blood disorder that raises risks of bleeding, infection, and complications like preterm birth. Researchers will follow 300 women to identify factors that lead to poor outcomes for mother a…

This study collects blood, tissue, and body fluid samples from up to 15,000 people with blood cancers or related disorders. The samples are stored in a central lab for future research on diagnosis, prognosis, and immune monitoring. Participants do not receive any treatment as par…

This study is a registry that tracks patients with blood diseases who receive stem cell transplants at a hospital on the Qinghai-Tibet Plateau. Researchers will collect information on recovery, complications, and costs over time. The goal is to understand how high altitude affect…