Thalassemia

This trial tests a modified stem cell transplant for people with severe sickle cell disease or beta-thalassemia who are at higher risk of transplant failure. The approach uses low-dose radiation and a mix of immunosuppressive drugs to help the body accept donor stem cells without…

This study looks at growth problems in children with thalassemia, a blood disorder. Researchers will test a program that includes health education, nutrition plans, exercise, and support to help kids grow better. The goal is to improve height, weight, and overall well-being in 36…

This study follows patients who have already been taking ruxolitinib alone or with panobinostat in earlier trials and are still benefiting. Researchers will track side effects over time to see how safe these drugs are for long-term use. About 296 participants with conditions like…

This study offers continued access to the drug mitapivat for adults with pyruvate kinase deficiency who completed an earlier Agios-sponsored trial and cannot get the drug commercially. Only 6 participants are enrolled, and the main goal is to monitor side effects. The study does …

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 trial investigates whether a 12- to 36-week program of aerobic and strength exercises can improve body composition, muscle function, and bone density in adolescents and adults with thalassemia. Participants will follow a self-directed routine of 30 minutes of exercise, 5 day…

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 tests a stem cell transplant using cells from a half-matched family donor, combined with low-dose radiation and drugs to prevent rejection, for adults with severe sickle cell disease or beta-thalassemia. The goal is to see if this gentler approach can replace the disea…

This study tested a drug called AG-348 in 20 adults with a type of thalassemia that doesn't require regular blood transfusions. The goal was to see if the drug could safely raise hemoglobin levels, which carry oxygen in the blood. Participants took the pill twice a day for up to …

This study tests a special treatment for red blood cells that aims to kill the Zika virus, making transfusions safer. About 692 people who need blood transfusions will receive either treated or standard blood. The goal is to see if the treated blood works just as well and is safe…

This early-phase study tests a new type of stem cell transplant for people with severe sickle cell disease who don't have a fully matched sibling donor. It uses a half-matched family donor and a milder chemotherapy regimen to prepare the body, along with removing certain immune c…

This study tests whether adding the drug abatacept to standard care can prevent graft-versus-host disease (GVHD) in children receiving stem cell transplants from unrelated donors. GVHD occurs when donor cells attack the patient's body, causing serious illness. The trial will enro…