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A Safety and Efficacy Study of β-globin Restored Autologous Hematopoietic Stem Cells for β-thalassemia Major Patients With CVS-654 Mutation

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Trial Profile

A Safety and Efficacy Study of β-globin Restored Autologous Hematopoietic Stem Cells for β-thalassemia Major Patients With CVS-654 Mutation

Status: Discontinued
Phase of Trial: Phase I/II

Latest Information Update: 17 Jan 2024

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At a glance

  • Drugs BRL 101 (Primary)
  • Indications Beta-thalassaemia
  • Focus Adverse reactions; Therapeutic Use

    • Most Recent Events

    • 17 Jan 2024 New trial record
    • 12 Dec 2023 Results of pooled analysis (NCT05577312, NCT04211480 and NCT04205435) assessing adverse events, neutrophil and platelet engraftment, Efficacy assessments included proportion of transfusion-independent (TI) subjects, levels of total hemoglobin and HbF, and proportion of red blood cells expressing HbF in peripheral blood, were presented at the 65th American Society of Hematology Annual Meeting and Exposition.

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