A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Subjects With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype
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
Doctors at research locations in the US and abroad seek adult patients with transfusion-dependent β-thalassemia, who do not have a β0/β0 genotype to trial a gene therapy of their own stem cells, which is performed outside the body in a lab and then the cells are returned to the patient with a functional human beta-globin gene. This hematopoetic stem cell transplant therapy uses a disabled virus to transfer a functional copy of the gene, and it requires that the patient receive leukapheresis to collect the stem cells, then chemotherapy to prepare their immune system before they are re-infused.
Follow up after the studies involved lab assessments and lasts 24 months to confirm the gene is producing βA-T87Q-globin and that the patients require fewer transfusions.
- Be up to 50 years of age
- Diagnosis of TDT with a history of at least 100 mL/kg/year of packed red blood cells (pRBCs) in the 2 years preceding enrollment (all subjects), or be managed under standard thalassemia guidelines with ≥8 transfusions of pRBCs per year in the 2 years preceding enrollment (subjects ≥12 years)
- Not have received hematopoetic stem cell transplant
THE STUDY INVOLVES:
- Screening before the treatment.
- Leukapheresis to collect blood stem cells.
- No treatment while the stem cells are gene-modified in the lab.
- Chemotherapy to prepare the immune system to receive the stem cells.
- Infusion of the gene-modified stem cells.
- Follow-up for a period of 24 months from the treatment.
LOCATIONS AND CONTACTS:
Study site locations in both the US and abroad are listed here.
Contact: bluebird bio | (339) 499-9300 | [email protected]
Or go online: