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A Single Arm, Open Label, Clinical Study of Cryopreserved Autologous CD34+ Cells Transduced With Lentiviral Vector Containing Human ARSA cDNA (OTL-200), for the Treatment of Early Onset Metachromatic Leukodystrophy (MLD)

Trial Profile

A Single Arm, Open Label, Clinical Study of Cryopreserved Autologous CD34+ Cells Transduced With Lentiviral Vector Containing Human ARSA cDNA (OTL-200), for the Treatment of Early Onset Metachromatic Leukodystrophy (MLD)

Recruiting
Phase of Trial: Phase II

Latest Information Update: 27 Dec 2018

At a glance

  • Drugs OTL 200 (Primary)
  • Indications Metachromatic leukodystrophy
  • Focus Therapeutic Use
  • Sponsors GlaxoSmithKline; Orchard Therapeutics
  • Most Recent Events

    • 17 Dec 2018 Phase has been changed
    • 31 Aug 2018 Biomarkers information updated
    • 10 Jan 2018 New trial record
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