HEMATOLOGIC DISEASES

This phase 3 trial tests a single dose of CTX001, a CRISPR gene therapy made from the child's own blood stem cells, for children with transfusion-dependent beta-thalassemia. The goal is to help them become transfusion-free for at least 12 months. Sixteen children will receive the…

This study follows 160 children and adults with beta-thalassemia or sickle cell disease who received a one-time treatment of their own CRISPR-edited stem cells (CTX001). The goal is to monitor long-term safety, including any new cancers or blood disorders, and to see how well the…

This study tests whether adding the drug vorinostat to standard care can prevent graft-versus-host disease (GVHD) in children, adolescents, and young adults receiving a bone marrow or blood stem cell transplant for blood cancers. Researchers are first finding the best dose of vor…

This experimental study tests whether frozen-thawed testis tissue, when grafted under the skin, can produce sperm in men with infertility. Five men will receive their own stored tissue. The goal is to see if viable sperm can be retrieved, potentially offering a future fertility o…

This study was designed to test the safety and tolerability of a new drug called HMPL-523 (sovleplenib) in adults with immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding problems. The trial was withdrawn before enrolling any pa…

This study follows over 32,000 people who helped clean up the 2010 Deepwater Horizon oil spill in the Gulf of Mexico. Researchers want to see if exposure to crude oil and chemical dispersants leads to long-term health problems like heart disease, cancer, or lung issues. Participa…

This study gathers medical information from up to 10,000 people with blood or heart conditions, as well as stem cell donors, treated at the National Institutes of Health. Researchers will review participants' medical records, test results, and treatments to build a database for g…

This study tests a new digital platform that helps doctors and nurses share important patient information when a person is moved from one hospital to another. About 1,000 adults being transferred to a major hospital will take part. The goal is to see if better information sharing…

This study collects blood and tissue samples from people receiving or donating bone marrow transplants for leukemia, lymphoma, or other blood diseases. Researchers will analyze these samples to find genes and proteins linked to cancer development and transplant complications. The…

This study follows 144 blood cancer patients undergoing stem cell transplants to see how muscle function changes and whether it predicts complications like infections, relapse, or survival. Researchers will measure muscle mass and track medical events over time. The goal is to un…