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Clinical Trials

Date: 2018-06-15

Type of information: Presentation of results at a congress

phase: 1-2

Announcement: presentation of results at the 23rd Annual Congress of the European Hematology Association

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 ?A-T87Q-globin gene and suspended in cryopreservative solution.

Disease: beta-thalassemia

Therapeutic area: Hematologic diseases - Genetic diseases - Rare diseases

Country: USA

Trial details:

  • The Northstar (HGB-204) is a non randomized, open label, multi-site, single-dose, Phase 1/2 study in up to 15 adults with beta-thalassemia major. The study will evaluate the safety and efficacy of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin® BB305 Drug Product [autologous CD34+ hematopoietic stem cells transduced with LentiGlobin® BB305 Lentiviral Vector encoding the human Beta-A(T87Q)-globin gene. (NCT01745120)

Latest news:

  • • On June 15, 2018, bluebird bio announced that new data from the completed Phase 1/2 Northstar (HGB-204) study in adolescents and adults with transfusion-dependent ?-thalassemia (TDT) and any genotype, and its ongoing, Phase 3 Northstar-2 (HGB-207) multicenter clinical study of LentiGlobin™ investigational gene therapy in patients with TDT and non-?0/?0 genotypes, will be presented in an oral session on June 16 at the 23rd Annual Congress of the European Hematology Association by Franco Locatelli, M.D., Ph.D., of the IRCCS Ospedale Pediatrico Bambino Gesù of Rome, Italy (LentiGlobin Gene Therapy for Transfusion-Dependent ?-Thalassemia (TDT) in Patients with Non-?0/?0 Genotypes: Updated Results from Northstar-2  - Abstract #1510). bluebird bio is now on track to submit a marketing authorization application in the European Union later this year, and continues to work closely with clinical investigators and regulatory authorities to complete its ongoing clinical trials.
  • • On December 5, 2015, bluebird bio announced that new data from the ongoing Northstar Study evaluating its LentiGlobin® BB305 product candidate in patients with beta-thalassemia major will be presented at the 57th American Society of Hematology Annual Meeting. The data from the Northstar Study will be highlighted on Sunday, December 6th, in an oral presentation by Mark C. Walters, M.D., UCSF Benioff Children’s Hospital, Oakland, Calif. This ongoing study is evaluating the safety and efficacy of LentiGlobin BB305 drug product for the treatment of subjects with beta-thalassemia major. As of October 28, 2015, 13 subjects with beta-thalassemia major have undergone infusion with LentiGlobin BB305 product candidate. As of October 28, 2015, nine of these subjects had at least six months follow up. Results in these patients as of October 28, 2015 include:
  • Median HbAT87Q production at six months follow up was 4.9 g/dL among patients of all genotypes (n=9), 4.9 g/dL among patients with non-Beta0/Beta0 genotypes (n=5) and 5.0 g/dL among patients with the Beta0/Beta0 genotype (n=4). All of the patients with non-Beta0/Beta0 genotypes with at least six months follow up (n=5) have achieved sustained transfusion independence as of the data cut-off, ranging from 7.1 to 16.4 months of ongoing transfusion independence; total hemoglobin at last follow up for these patients ranged from 9.1 to 12.2 g/dL. Patients with the Beta0/Beta0 genotype (n=4) demonstrated a reduction in transfusion volume ranging from 33 percent to 100 percent. The safety profile was consistent with autologous transplantation. No Grade 3 or higher drug-product related adverse events have been observed, and there is no evidence of clonal dominance.
  • • On March 27, 2014, bluebird bio, a US clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and orphan diseases, has announced that the first subject with beta-thalassemia major has been enrolled in its phase 1/2 Northstar Study (HGB-204) in the United States and has undergone infusion with bluebird bio’s LentiGlobin® drug product in an autologous hematopoietic stem cell transplantation.
  • LentiGlobin® is currently in another phase 1/2 study in France (HGB-205), for the treatment of beta-thalassemia major. The phase 1/2 HGB-205 study also allows enrollment of patient(s) with sickle cell disease and bluebird bio is planning to enroll its first sickle cell patient in a separate U.S. trial in 2014.

Is general: Yes