A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Subjects With Transfusion-Dependent β-Thalassemia

This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 18 subjects ≤50 years of age with transfusion-dependent β-thalassemia (TDT), who have a β0/β0, β0/IVS-I-110, or IVS-I-110/IVS-I-110 genotype. The study will evaluate the efficacy and safety of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug Product.

NCT: NCT03207009

Locations: Ann & Robert H. Lurie Children’s Hospital of Chicago 

Link: https://clinicaltrials.gov/ct2/show/NCT03207009?recrs=ad&cond=Thalassemia&locn=Ann+%26+Robert+H.+Lurie+Children%E2%80%99s+Hospital+of+Chicago&cntry=US&age=0&draw=2&rank=1