SICKLE CELL ANEMIA

This study tests whether adding a new antibody drug (briquilimab, JSP191) to a standard blood stem cell transplant can help more people with sickle cell disease or beta-thalassemia achieve a full donor cell takeover, potentially curing their condition. About 40 participants aged …

This study tests a stem cell transplant using a half-matched family donor for adults with severe sickle cell disease or beta-thalassemia. Instead of high-dose chemotherapy, patients receive low-dose radiation and immune-suppressing drugs to help the donor cells take hold. The goa…

This study follows 160 children and adults with beta-thalassemia or sickle cell disease who already received an experimental CRISPR gene therapy called CTX001. Researchers will track participants for years to check for long-term side effects, new cancers, and how well the treatme…

This study looks at whether acceptance and commitment therapy (ACT) can improve sleep in adults with sickle cell disease. Participants will have weekly video chats with a coach for 8 weeks and wear a wrist device to track sleep. The goal is to see if the therapy is helpful and ea…

This study looks at how common strokes and silent brain injuries are in young adults (ages 16-25) with sickle cell anemia in northern Nigeria. Researchers will use brain scans and ultrasound to check for damage and also look at other risk factors like high blood pressure. The goa…

This observational study looks at how the drug mitapivat changes red blood cells in people with sickle cell disease. Researchers will measure how well the cells carry oxygen and how long they survive. The study involves about 6 people already enrolled in a larger trial, with extr…