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: 17 Sep 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

    • 02 Aug 2017 According to a bluebird bio media release, recruitment completed for the adult and adolescent patient cohort in Jun 2017.
    • 25 Jun 2017 Trial design and recent update (n=2) on this study, presented at the 22nd Congress of the European Haematology Association.
    • 23 Jun 2017 According to a bluebird bio media release, Alexis Thompson is a primary investigator on the study. As of June 2, 2017, drug product had been manufactured for six patients.
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