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 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

Trial Profile

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 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

Recruiting
Phase of Trial: Phase III

Latest Information Update: 10 Dec 2017

At a glance

  • Drugs BB305 beta-globin gene therapy bluebird bio (Primary)
  • Indications Beta-thalassaemia
  • Focus Registrational; Therapeutic Use
  • Acronyms Northstar-2
  • Sponsors bluebird bio
  • Most Recent Events

    • 10 Dec 2017 According to a bluebird bio media release, Janet L. Kwiatkowski, MD, MSCE, Director of the Thalassemia Program at the Children's Hospital of Philadelphia is the primary investigator of this study. Data from this study and other study (238788) is used for matketing approval in TDT.
    • 10 Dec 2017 Results published in the bluebird Media Release
    • 10 Dec 2017 According to a bluebird bio media release, results are presented at the 59th Annual Meeting of the American Society of Hematology (ASH) Annual Meeting 2017.
Restricted Access

Oops, it looks like you don’t have a valid subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • with a username/password associated to an account with a valid subscription
  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Request a trial

If you are a subscriber to this content then contact us at AsktheExpert.AdisInsight@springer.com so we can help.

Back to top