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

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 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

Recruiting
Phase of Trial: Phase III

Latest Information Update: 06 Mar 2018

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

    • 21 Feb 2018 According to a bluebird bio media release, clinical data from this trial will be presented at the European Hematology Association Annual Meeting 2018.
    • 12 Dec 2017 Longer follow-up on the 3 treated patients and data from approximately 5 additional treated patients will be presented.
    • 12 Dec 2017 Initial results (n=6; data cut off June 2017, 17) evaluating safety and efficacy presented at the 59th Annual Meeting and Exposition of the American Society of Hematology
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