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A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects With Transfusion-dependent β-Thalassemia by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector in Subjects ≤50 Years of Age

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A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects With Transfusion-dependent β-Thalassemia by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector in Subjects ≤50 Years of Age

Status: Completed
Phase of Trial: Phase III

Latest Information Update: 02 Jan 2024

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At a glance

  • Drugs Betibeglogene-autotemcel (Primary)
  • Indications Beta-thalassaemia
  • Focus Registrational; Therapeutic Use
  • Acronyms Northstar-3
  • Sponsors bluebird bio

    • Most Recent Events

    • 12 Dec 2023 Results of an analysis reporting iron management outcomes in patients who completed either a phase 1/2 (NCT01745120, NCT02151526) or phase 3 beti-cel parent studies (NCT02906202 and NCT03207009) and subsequently enrolled in the long-term follow-up study (NCT02633943) up to 9 years of follow up presented at the 65th American Society of Hematology Annual Meeting and Exposition
    • 12 Dec 2023 Results of an analysis reporting report efficacy, safety, and quality of life (QOL) data from following clinical studies: NCT01745120, NCT02151526, NCT02906202, NCT03207009 and NCT02633943 presented at the 65th American Society of Hematology Annual Meeting and Exposition
    • 10 Dec 2023 Results published in the bluebird bio Media Release

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