A Phase 1/2 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy in Subjects With β-Thalassemia Major by Transplantation of Autologous CD34+ Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector (LentiGlobin® BB305 Drug Product)

Trial Profile

A Phase 1/2 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy in Subjects With β-Thalassemia Major by Transplantation of Autologous CD34+ Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector (LentiGlobin® BB305 Drug Product)

Completed
Phase of Trial: Phase I/II

Latest Information Update: 02 Apr 2018

At a glance

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

    • 29 Mar 2018 Status changed from active, no longer recruiting to completed.
    • 12 Dec 2017 Results presented at the 59th Annual Meeting and Exposition of the American Society of Hematology
    • 10 Dec 2017 Results published in the bluebird Media Release
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