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ADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCID

Trial Profile

ADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCID

Status: Completed
Phase of Trial: Phase II

Latest Information Update: 12 Aug 2019

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At a glance

  • Drugs GSK 2696273 (Primary) ; Busulfan
  • Indications Adenosine deaminase deficiency
  • Focus Therapeutic Use
  • Sponsors GlaxoSmithKline; Orchard Therapeutics
  • Most Recent Events

    • 07 Aug 2019 Status changed from active, no longer recruiting to completed.
    • 30 May 2016 According to a MolMed media release, the European Commission approved GSK-2696273 (Strimvelis), the first ex-vivo stem cell gene therapy to treat patients with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID).
    • 27 May 2016 Primary endpoint (Survival) has been met, according to a GlaxoSmithKline media release.
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