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

Active, no longer recruiting
Phase of Trial: Phase I/II

Latest Information Update: 10 Dec 2017

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

    • 10 Dec 2017 Results published in the bluebird Media Release
    • 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 (257159) is used for matketing approval in TDT.
    • 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.
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