Bone marrow failure syndrome

This early-stage trial tests imatinib, a drug already used for certain cancers, in adults with a harmful RUNX1 gene mutation. The mutation causes easy bleeding and a high risk of blood cancers. The study aims to find the best dose and see if imatinib can improve platelet function…

This expanded access protocol provides stem cell transplants for people with serious blood diseases like leukemia, bone marrow failure, or immune deficiencies who do not have a fully matched sibling donor. The donated stem cells are processed to remove certain immune cells (alpha…

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 study tests a bone marrow transplant from a family donor for children under 21 with severe non-cancer blood disorders like sickle cell disease, bone marrow failure, or immune problems. The goal is to see if the transplant can replace the diseased cells with healthy donor cel…

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 giving two drugs, decitabine and filgrastim, after a stem cell transplant can help prevent blood cancers like AML and MDS from coming back in children and young adults. About 37 participants will receive the drug combination for up to 6 cycles. The main g…

This study tests a cord blood transplant in children and young adults (up to age 21) with life-threatening blood cancers or non-cancer blood disorders who do not have a matched family donor. The goal is to see if this approach lowers the risk of death from treatment one year afte…

This phase 2 trial tests a combination of three chemotherapy drugs (treosulfan, fludarabine, thiotepa) plus an immune-suppressing antibody before a donor stem cell transplant in up to 40 people under 50 with non-cancerous blood disorders. The goal is to see if this milder conditi…

This expanded access program offers a stem cell transplant using donor cells that have been specially processed to remove certain immune cells, reducing the risk of graft-versus-host disease. It is for patients with serious blood disorders, immune deficiencies, or metabolic disea…

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 is testing a new approach for people with severe lung disease (like pulmonary fibrosis or COPD) who also have bone marrow failure. First, participants receive a lung transplant to restore breathing. Then, about 8 weeks later, they get a bone marrow transplant using the…

This phase 2 trial is testing a stem cell transplant using a milder chemotherapy regimen to treat people with severe immune deficiencies and inherited bone marrow failure. The goal is to see if donor cells can safely take over and rebuild a healthy immune system. Up to 27 partici…

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 study tests whether giving blood transfusions at home is practical and acceptable for cancer patients who need regular transfusions. About 30 adults with blood cancers or bone marrow failure will receive 1-5 blood product units at home. Researchers will track how often trans…

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 provides ongoing check-ups for people who received a donor stem cell transplant at the NIH at least three years ago. Researchers will monitor for late side effects, disease return, and overall health. Participants will have yearly visits that may include blood tests, b…

This study interviews patients and families who have experience with gene therapy for rare pediatric diseases, including sickle cell disease. Researchers want to understand their beliefs, attitudes, and what information they need. The goal is to create an online platform with edu…

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 follows people with Fanconi anemia, a rare inherited condition that raises the risk of certain cancers, especially squamous cell carcinoma. Researchers will screen 200 participants aged 12 and older every year for up to 10 years using exams, blood tests, and imaging to…

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 collects blood, bone marrow, and other samples along with health information from up to 1,716 people with non-cancerous blood diseases and their family members. Researchers will analyze the participants' genes to find new genetic causes of these conditions and understa…

This study looks at how antibiotics given during a stem cell transplant change the bacteria in your gut. Researchers will track these changes in 255 adults getting a transplant for blood cancers or bone marrow failure. The goal is to understand the link between antibiotics and gu…

This study is looking at 2,000 people who have early warning signs of blood cancers or bone marrow failure. Researchers use a special genetic test (next generation sequencing) to find changes in cancer-related genes. The goal is to better understand who is at risk and to catch th…

This study is testing a virtual reality (VR) program called Life at 100.4 to help teens and young adults with cancer, along with their caregivers, learn about their diagnosis and treatment in an immersive way. About 45 participants will use the VR tool, and researchers will measu…

This study collects blood samples from up to 700 people, including stem-cell transplant patients and their donors, to understand how the immune system recovers after transplant. Researchers will analyze immune cells and T cell subsets to learn about immune reconstitution and graf…