- 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 - active not recruitingPHASE3
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
This study is testing a new 'gene-editing' treatment called CTX001 for children aged 2-11 with a blood condition called beta-thalassemia. It aims to see how safe and effective this single treatment is, hoping to reduce their need for regular blood transfusions.
United States · Canada · Germany - recruitingPHASE3
A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials
This study looks at the long-term safety of a medicine called luspatercept for people with certain blood disorders. It's for those already taking part in a luspatercept study to see if continuing the treatment is safe over a longer period.
United States · Australia · Belgium