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A Phase 1 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy in Subjects With Transfusion-dependent α-Thalassemia by Transplantation of Autologous CD34+ Cells Transduced Ex Vivo With a Lentiviral Vector (GMCN-508A Drug Product)

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Trial Profile

A Phase 1 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy in Subjects With Transfusion-dependent α-Thalassemia by Transplantation of Autologous CD34+ Cells Transduced Ex Vivo With a Lentiviral Vector (GMCN-508A Drug Product)

Status: Recruiting
Phase of Trial: Phase I

Latest Information Update: 21 Apr 2023

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At a glance

  • Drugs GMCN 508A (Primary)
  • Indications Alpha-thalassaemia
  • Focus Therapeutic Use

Most Recent Events

  • 16 Apr 2023 Planned primary completion date changed from 1 Jun 2028 to 1 Aug 2028.
  • 16 Apr 2023 Status changed from not yet recruiting to recruiting.
  • 14 Mar 2023 New trial record

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