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

Status: Completed
Phase of Trial: Phase I/II

Latest Information Update: 13 Jun 2019

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At a glance

  • Drugs BB305 beta-globin gene therapy bluebird bio (Primary)
  • Indications Beta-thalassaemia; Haemoglobinopathies; Sickle cell anaemia
  • Focus Adverse reactions; Pharmacodynamics; Proof of concept; Therapeutic Use
  • Sponsors bluebird bio
  • Most Recent Events

    • 10 Jun 2019 Status changed from active, no longer recruiting to completed.
    • 03 Jun 2019 The European Commission (EC) has granted conditional marketing authorization for ZYNTEGLO for patients 12 years and older with transfusion-dependent beta-thalassemia for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available. This approval is supported by data from HGB-205 ,HGB-204, HGB-207, HGB-212 and LTF-303 studies.
    • 16 May 2019 This trial has been completed in France.
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