Phase 3b Study of a Single Dose of CTX001 Gene-Modified Stem Cell Therapy for Beta-Thalassemia and Severe Sickle Cell Disease in Patients Aged 12 to 35

What this Trial Is About

Researchers are evaluating the safety and effectiveness of a single dose of CTX001, an autologous CRISPR-Cas9 modified CD34 human hematopoietic stem and progenitor cell therapy, in participants aged 12 to 35 years with transfusion-dependent beta-thalassemia TDT or severe sickle cell disease SCD. This Phase 3b open-label study aims to understand how this gene-edited therapy may impact these serious blood disorders that require frequent transfusions or cause severe complications. Participants will receive one infusion of CTX001 through a central venous catheter after undergoing myeloablative conditioning with busulfan. The study focuses on a single-dose administration of these genetically modified stem cells targeting the BCL11A gene enhancer to potentially improve hemoglobin production. No placebo or comparison group is involved, and the intervention is delivered intravenously. During the study, participants will be closely monitored for up to 12 months after infusion, including assessments of fetal and total hemoglobin levels over time. Safety evaluations will track adverse events, serious adverse events, and transplant-related mortality. Researchers will also measure engraftment success, genetic modification presence in blood and bone marrow, transfusion needs, and rates of vaso-occlusive crises and hospitalizations for SCD. Participants will undergo laboratory tests and clinical evaluations to gather comprehensive data on treatment impact and safety throughout the study period.

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease