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Long-term Follow-up of Subjects with Hemoglobinopathies Treated with Ex Vivo Gene Therapy using Autologous Hematopoietic Stem Cells Transduced with a Lentiviral Vector

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Trial Profile

Long-term Follow-up of Subjects with Hemoglobinopathies Treated with Ex Vivo Gene Therapy using Autologous Hematopoietic Stem Cells Transduced with a Lentiviral Vector

Status: Active, no longer recruiting
Phase of Trial: Phase III

Latest Information Update: 11 Apr 2025

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At a glance

  • Drugs Betibeglogene-autotemcel (Primary) ; Busulfan
  • Indications Beta-thalassaemia; Haemoglobinopathies; Sickle cell anaemia
  • Focus Adverse reactions; Pharmacodynamics; Registrational; Therapeutic Use
  • Sponsors bluebird bio

Most Recent Events

  • 05 Nov 2024 According to a bluebird bio media release, results in durable Hemoglobin A (HbA) production with up to 10 years of follow-up, will be presented at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition. The meeting will take place December 7-10, 2024 at the San Diego Convention Center and online.
  • 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

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