This is a multi-center, longterm safety and efficacy follow-up study for subjects who have been treated with the LentiGlobin BB305 drug product on HGB-206 sickle cell gene therapy parent study. This study is observational only, and no investigational treatment will be given on this follow-up study. Participants for this study are requested to follow-up every six months for the first three years and then once every year for the remaining Year 4 through Year 13, totaling thirteen years in this longterm study. Study assessments will include exams, laboratory studies, imaging, ECG, surveys, and record review.
This study is being conducted to assess safety and efficacy of bone marrow transplant in severe and less severely affected children. In this study, we will transplant bone marrow or cord blood from a HLA-matched brother or sister instead of the currently more common method with a HLA-identical sibling donor. This is a multicenter, single arm trial for patients between 2 and 10 years of age who have less severe HbSS or sickle beta zero thalassemia. Patient must have an HLA identical sibling donor who is less than 10 years old and who does not have HbSS, SC or sickle beta zero thalassemia.
This study is evaluating an experimental procedure for treating sickle cell disease called gene transfer. In sickle cell disease, a malfunctioning hemoglobin gene causes red blood cells to change shape and get rigid, which results in organ damage and other problems. This research will study whether modifying the gene through gene transfer will correct the abnormalities and eliminate or reduce the effects of sickle cell disease. Blood stem cells will be harvested from a subject with sickle cell disease, and the modified gene will be inserted into the cells. These stem cells will be given back to the subject in a procedure called an autologous stem cell transplant. The subject will be closely monitored for side effects of the procedure and signs that the modified stem cells are producing normal red blood cells.
This study if for patients that have a blood disease and it's been determined that the best option for treating that blood disease is a cord blood transplant. Cord blood (CB) is blood that is taken from the umbilical cord and placenta of healthy newborn babies after childbirth. The cord blood collected from a newborn baby is called a cord blood unit. The United States Food and Drug Administration (FDA) considers cord blood to be a biological drug. These are considered "investigational" products. This study will evaluate the safety of administration of the investigational cord blood units by carefully documenting all infusion-related problems.