- 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.
For: Beta-Thalassemia · Thalassemia · Genetic Diseases, InbornUnited States · Canada · Germany - Active not recruitingPHASE3
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)
This study is testing a new gene therapy, CTX001, for children aged 2-11 with severe sickle cell disease. It's for those whose current medicine, hydroxyurea, isn't working well or can't be taken. The aim is to see if this new treatment is safe and effective.
For: Sickle Cell Disease · Hydroxyurea Failure · Hydroxyurea IntoleranceUnited States · Germany · Italy - 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.
For: Beta-Thalassemia · Thalassemia · Sickle Cell DiseaseUnited States · Belgium · Canada