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A Phase 1 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy in Subjects With β-Thalassemia Major by Transplantation of Autologous CD34+Stem Cells Transduced With a Lentiviral Vector Encoding βA-T87Q-Globin

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A Phase 1 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy in Subjects With β-Thalassemia Major by Transplantation of Autologous CD34+Stem Cells Transduced With a Lentiviral Vector Encoding βA-T87Q-Globin

Status: Recruiting
Phase of Trial: Clinical Phase Unknown

Latest Information Update: 24 Apr 2024

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At a glance

  • Drugs BD 211 (Primary) ; Busulfan (Primary)
  • Indications Beta-thalassaemia
  • Focus Adverse reactions; Pharmacokinetics; Therapeutic Use
  • Sponsors Shanghai BDgene

    • Most Recent Events

    • 19 Apr 2024 Planned End Date changed from 23 Feb 2024 to 23 Feb 2025.
    • 19 Apr 2024 Planned primary completion date changed from 23 Feb 2024 to 23 Feb 2025.
    • 24 Oct 2022 Planned End Date changed from 1 Jul 2023 to 23 Feb 2024.

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