This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 23 subjects ≤50 years of age with transfusion-dependent β-thalassemia (TDT), also known as β-thalassemia major, who do not have a β0 mutation at both alleles of the hemoglobin β (HBB) gene. 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: TI is defined as a weighted average Hb ≥9g/dL without any RBC transfusions for a continuous period of ≥12 months at any time during the study after drug product infusion.Measure: The proportion of treated subjects who meet the definition of "transfusion independence" (TI). 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 do Not 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 ---