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A Phase I/II Study Evaluating Safety and Efficacy of Autologous Hematopoietic Stem Cells Genetically Modified With GLOBE Lentiviral Vector Encoding for the Human Beta-globin Gene for the Treatment of Patients Affected by Transfusion Dependent Beta-thalassemia

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Trial Profile

A Phase I/II Study Evaluating Safety and Efficacy of Autologous Hematopoietic Stem Cells Genetically Modified With GLOBE Lentiviral Vector Encoding for the Human Beta-globin Gene for the Treatment of Patients Affected by Transfusion Dependent Beta-thalassemia

Status: Completed
Phase of Trial: Phase I/II

Latest Information Update: 05 Sep 2023

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At a glance

  • Drugs OTL 300 (Primary)
  • Indications Beta-thalassaemia
  • Focus Adverse reactions; First in man; Therapeutic Use
  • Acronyms TIGET-BTHAL

Most Recent Events

  • 03 Apr 2022 Status changed from active, no longer recruiting to completed.
  • 03 May 2018 Status changed from recruiting to active, no longer recruiting.
  • 12 Dec 2017 Preliminary results (n=7, data cut off: Aug 2017) presented at the 59th Annual Meeting and Exposition of the American Society of Hematology

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