HEMOGLOBINOPATHIES

This study tests a reduced intensity conditioning regimen for children and young adults with non-malignant blood disorders like sickle cell disease and immune deficiencies. The goal is to achieve stable donor cell engraftment after a stem cell transplant while causing fewer side …

This study tests a new way to choose cord blood for transplant in people with blood cancers like leukemia or lymphoma. Patients get stem cells from a family member plus a cord blood unit, matched to target certain inherited traits. The goal is to find the lowest safe cord blood d…

This phase 3 trial tests a single dose of CTX001, a CRISPR-edited stem cell therapy, in 13 children with severe sickle cell disease who have not responded to standard treatments. The goal is to see if it can prevent severe pain crises for at least a year. Participants receive the…

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 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 a stem cell transplant method that removes certain immune cells from the donor's blood to lower the chance of graft-versus-host disease (GvHD), a serious complication. It involves 3 patients with blood cancers or non-cancerous blood disorders who lack a perfectly…