This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 15 subjects ≤50 years of age with transfusion-dependent β-thalassemia (TDT), who have a β0 mutation at both alleles of the β-globin (HBB) gene (i.e. β0/β0). The study will evaluate the efficacy and safety of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug Product.
Name: LentiGlobin BB305 Drug ProductDescription: LentiGlobin BB305 Drug Product is administered by IV infusion following myeloablative conditioning with busulfan.Type: Genetic
LentiGlobin BB305 Drug Product
Description: TR is defined as demonstration of reduction in volume of RBC transfusion requirements (in mL/kg) in the post-treatment time period of Months 12 to 24 compared to the average annual transfusion requirement in the 24 months prior to enrollment.Measure: The proportion of subjects who meet the definition of "transfusion reduction" (TR). Time: 12-24 months post-transplant
Single Group Assignment
There is one SNP
A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects With Transfusion-dependent β-Thalassemia, Who Have a β0/β0 Genotype, by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector in Subjects ≤50 Years of Age. --- T87Q ---