- enrolling by invitationPHASE3
A Long-term Follow-up Study in Participants Who Received CTX001
This study is looking at the long-term safety and how well a treatment called CTX001 works for children and adults with serious blood conditions like beta-thalassemia and sickle cell disease. It's for people who have already had CTX001 in a previous study.
United States · Belgium · Canada - recruitingPHASE2
A Trial to Assess Haploidentical T-depleted Stem Cell Transplantation in Patients With SCD
This study is testing a new type of stem cell transplant for people with sickle cell disease. It aims to see if this new transplant, using cells from a partially matched family member, is as safe and effective as the current best transplant option.
Austria · Germany