Efficacy and Safety of a Cryopreserved Formulation of Autologous CD34+ Hematopoietic Stem Cells Transduced Ex Vivo With EFS Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency

Trial Profile

Efficacy and Safety of a Cryopreserved Formulation of Autologous CD34+ Hematopoietic Stem Cells Transduced Ex Vivo With EFS Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency

Active, no longer recruiting
Phase of Trial: Phase I/II

Latest Information Update: 07 Feb 2018

At a glance

  • Drugs Adenosine deaminase transduced bone marrow cells (Primary)
  • Indications Adenosine deaminase deficiency
  • Focus Therapeutic Use
  • Most Recent Events

    • 01 Feb 2018 Planned primary completion date changed from 1 Jun 2020 to 1 Oct 2019.
    • 01 Feb 2018 Status changed from recruiting to active, no longer recruiting.
    • 26 Dec 2016 New trial record
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