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Efficacy and Safety of a Cryopreserved Formulation of Autologous CD34+ Haematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1α Short Form (EFS) Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency (SCID) Due to Adenosine Deaminase Deficiency

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Trial Profile

Efficacy and Safety of a Cryopreserved Formulation of Autologous CD34+ Haematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1α Short Form (EFS) Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency (SCID) Due to Adenosine Deaminase Deficiency

Status: Completed
Phase of Trial: Phase I/II

Latest Information Update: 03 Feb 2023

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At a glance

  • Drugs Simoladagene autotemcel (Primary)
  • Indications Adenosine deaminase deficiency
  • Focus Registrational; Therapeutic Use
  • Most Recent Events

    • 31 Jan 2023 Status changed from active, no longer recruiting to completed.
    • 10 Jun 2021 Planned End Date changed from 1 Jul 2022 to 1 Sep 2022.
    • 10 Jun 2021 Planned primary completion date changed from 1 Jul 2021 to 1 Sep 2021.
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