Phase I/II, Non-controlled, Open-label, Non-randomised, Single-centre Trial to Assess the Safety and Efficacy of EF1αS-ADA Lentiviral Vector Mediated Gene Modification of Autologous CD34+ Cells From ADA-deficient Individuals

Trial Profile

Phase I/II, Non-controlled, Open-label, Non-randomised, Single-centre Trial to Assess the Safety and Efficacy of EF1αS-ADA Lentiviral Vector Mediated Gene Modification of Autologous CD34+ Cells From ADA-deficient Individuals

Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 08 Jul 2017

At a glance

  • Drugs ADA-SCID gene therapy (Primary)
  • Indications Adenosine deaminase deficiency
  • Focus First in man; Therapeutic Use
  • Most Recent Events

    • 18 Oct 2016 New trial record
    • 22 Sep 2016 Pooled clinical data (n=32) from this and another study (Profile 277538) published in an Orchard Therapeutics media release.
    • 21 Sep 2016 According to Orchard Media release, clinical data from this study will be presented at the 17th biennial meeting of the European Society for Immunodeficiencies (ESID) 2016 and at the European Society of Gene and Cell Therapy (ESGCT) conference.
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