Clinical Trials

Date: 2017-11-16

Type of information: Treatment of the first patient

phase: 3

Announcement: treatment of the first patient

Company: Bluebird bio (USA - MA)

Product: LentiGlobin® BB305 (autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human beta A-T87Q-globin gene)

Action mechanism:

• gene therapy/stem cell therapy. LentiGlobin BB305 Drug Product consists of autologous CD34+ hematopoietic stem cells transduced with lentiviral vector LentiGlobin BB305 encoding the human Beta A-T87Q-globin gene and suspended in cryopreservative solution.
• LentiGlobin is also being investigated in Northstar-2 (HGB-207), a Phase 3 study in patients with TDT and non- Beta 0/ Beta 0 genotypes, Northstar (HGB-204), a Phase 1/2 study in patients with TDT and all genotypes, HGB-205, a Phase 1 study in patients with TDT and severe sickle cell disease (SCD), and HGB-206, a Phase 1 study in patients with SCD.

Disease: transfusion-dependent beta-thalassemia

Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases

Country: France, Germany, Greece, Italy, UK, USA

Trial details:

• Northstar-3 (HGB-212) is a Phase 3, global, multi-center study designed to evaluate the safety and efficacy of LentiGlobin in patients with transfusion-dependent beta-thalassemia with a Beta 0/ Beta 0 genotype. In this study, the manufacturing process by which the patient’s cells are transduced with the LentiGlobin viral vector has been modified, with the intent of increasing the percentage of cells successfully transduced and the average vector copy number per diploid genome.

• The study’s primary endpoint is the proportion of patients who meet the definition of "transfusion reduction" (TR). TR is defined as demonstration of reduction of at least 60% in volume of red blood cell (RBC) transfusion requirements (in mL/kg) in the post-treatment time period of Months 12 to 24, as compared to the average annual transfusion requirement in the 24 months prior to enrollment. The target enrollment of the study is 15 adult, adolescent and pediatric patients. (NCT03207009)

Latest news:

• • On November 16, 2017, bluebird bio announced that the first patient has been treated in Northstar-3 (HGB-212), the company’s Phase 3 study of LentiGlobin in patients with transfusion-dependent Beta -thalassemia (TDT) with the Beta 0/ Beta 0 genotype.

     

Is general: Yes