Evaluating Efficacy and Safety of Gene Therapy in Transfusion-dependent Beta-Thal Beta0/Beta0 Genotype by Autologous Transplantation of CD34+ Stem Cells Transduced Ex Vivo with Lentiviral BA-T87Q Globin Vector in Subjects < 50 Years

NCT#/Phase: NCT02906202
Phase 3
IRB#: 2017-050
Description: This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 15 subjects with transfusion-dependent Beta thalassemia (TDT), also known as Beta Thalassemia Major, who do not have a Beta 0 mutation at both alleles of the hemoglobin Beta (HBB) gene. The study will evaluate the efficacy and safety of autologous hematopoietic stem cell transplantation (HSCT) using mobilized peripheral blood stem cells transduced with LentiGlobin BB305 drug product.
Inclusion: Individuals who are less than 50 Years of age, with transfusion-dependent Beta-thalassemia (TDT).
Enrollment Status: Open for enrollment
Sponsor: Bluebird Bio
Principal Investigator(s): Ashutosh Lal, MD
Contact(s): Marci Moriarty, RN (510-428-3885 x5396)
Julia Klein (510-428-3885 x5427)
Cyrus Bascon (510-428-3885 x6953
Study Link: https://clinicaltrials.gov/ct2/show/NCT02906202