Clinical Trials

Date: 2015-06-09

Type of information: Submission of a clinical trial application

phase: 1

Announcement:

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.

Disease: beta-thalassemia major

Therapeutic area: Genetic diseases - Hematological diseases - Rare diseases

Country:

Trial details: HGB-208 Pediatric Study Protocol

Latest news:

• • On June 9, 2015, bluebird bio, a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and rare diseases and T cell-based immunotherapies, announced the completion of the National Institutes of Health (NIH) Recombinant DNA Advisory Committee’s (RAC) public review of the HGB-208 pediatric study protocol for bluebird bio’s LentiGlobin BB305 product candidate in beta-thalassemia major. The RAC recommendation was to delay initiation of the study in the United States for one to two years. This recommendation has no effect on the HGB-207 protocol plan. In May, bluebird bio announced that it had reached general agreement with the FDA on the design of its planned clinical trials HGB-207 and HGB-208. Based on its discussions with the FDA, bluebird bio believes that data from these trials, together with data from the ongoing beta-thalassemia major clinical studies (Northstar and HGB-205), could form the basis for a Biologics License Application (BLA) submission for LentiGlobin BB305. HGB-207 and HGB-208 share similar trial designs and are differentiated primarily by patient age. HGB-207 will enroll adult and adolescent patients; HGB-208 is planned to enroll pediatric patients.
• Highlights:
• Sample size: 15 patients per trial
• Duration: 24 months of follow-up per patient
• Primary endpoint: 12 months of transfusion independence
• The RAC had previously notified bluebird bio that only HGB-208 required a public RAC discussion.
• bluebird bio also announced in May that it is one of the first companies to participate in the European Medicines Agency’s (EMA) Adaptive Pathways (formerly referred to as Adaptive Licensing) pilot program, which is part of the EMA’s efforts to improve timely access for patients to new medicines. Based on several discussions involving the EMA, European Health Technology Assessment (HTA) agencies and patient advocacy organizations as part of this program, bluebird bio believes it is possible to seek conditional approval for the treatment of adults and adolescents with beta-thalassemia major on the basis of the totality of clinical data, in particular reduction in transfusion need, from the ongoing Northstar Study and supportive HGB-205 study. Conversion to full approval will be subject to the successful completion of the HGB-207 and HGB-208 clinical trials, supportive long-term follow-up data and “real-life” post-approval monitoring data.

Is general: Yes