Bone marrow failure syndrome

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 adding the drug vorinostat to standard care can prevent graft-versus-host disease (GVHD) in children and young adults (ages 1-26) with non-cancerous blood disorders who are getting a bone marrow transplant. GVHD is a serious complication where donor cells…

This study tests whether removing a specific type of immune cell (naive T cells) from a donor's stem cell graft can prevent graft-versus-host disease (GVHD), a serious complication where donor cells attack the recipient's body. The trial enrolls people with non-malignant blood di…

This early-phase trial tests whether giving healthy stem cells from the mother to a fetus diagnosed with Fanconi anemia can safely improve the baby's blood cell production. The treatment is a one-time injection into the fetus during pregnancy. The study will enroll 12 pregnant wo…

This study will test different methods to prevent infections and other problems from central lines (tubes placed in large veins) in 2,500 patients. It compares special antimicrobial caps and a skin anchor device against standard care. The goal is to find the best ways to keep pat…

This study screens up to 1,000 people with personal or family histories of certain cancers to see if they qualify for ongoing genetics research at the National Cancer Institute. Participants fill out a 15-20 minute online survey about their health and family history. No treatment…

This study aims to create a score that uses a special blood test to predict bleeding risk in people with blood cancers and very low platelet counts. Researchers will take an extra blood sample from 410 participants and analyze it with viscoelastometric tests. The goal is to ident…

This early-phase study at St. Jude Children's Research Hospital will test whether it is safe and possible to collect blood stem cells from 12 young adults with inherited bone marrow failure syndromes. Participants will receive two drugs (filgrastim and plerixafor) to move stem ce…