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A Phase 1/2, Open-label, Single-arm Study to Assess the Safety, Tolerability, and Efficacy of ST-400 Autologous Hematopoietic Stem Cell Transplant for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)

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Trial Profile

A Phase 1/2, Open-label, Single-arm Study to Assess the Safety, Tolerability, and Efficacy of ST-400 Autologous Hematopoietic Stem Cell Transplant for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)

Status: Completed
Phase of Trial: Phase I/II

Latest Information Update: 19 Dec 2023

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At a glance

  • Drugs ST 400 (Primary) ; Busulfan
  • Indications Beta-thalassaemia
  • Focus Adverse reactions
  • Acronyms THALES
  • Sponsors Sangamo Therapeutics

    • Most Recent Events

    • 26 Jan 2023 Status changed from active, no longer recruiting to completed.
    • 16 Dec 2021 Planned End Date changed from 1 Mar 2023 to 1 Nov 2022.
    • 14 Dec 2021 Updated results assessing HbF expression in edited erythroid cells Thales trial (NCT03432364) presented at the 63rd American Society of Hematology Annual Meeting and Exposition
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