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A Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Treatment of Metachromatic Leukodystrophy

Trial Profile

A Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Treatment of Metachromatic Leukodystrophy

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

Latest Information Update: 05 Mar 2023

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At a glance

  • Drugs Atidarsagene autotemcel (Primary) ; Busulfan
  • Indications Metachromatic leukodystrophy
  • Focus Adverse reactions; Therapeutic Use
  • Acronyms TIGET-MLD
  • Sponsors GlaxoSmithKline; GSK; Orchard Therapeutics
  • Most Recent Events

    • 24 Feb 2023 According to an Orchard Therapeutics media release, data of this trial used for Long-term integrated analysis are being presented at the ongoing 19th Annual WORLDSymposium™ taking place February 22-26, 2023, in Orlando, Florida.
    • 24 Feb 2023 Long-term results of updated integrated analysis in early-onset MLD patients enrolled in two prospective non-randomized clinical studies (n=30) or treated under expanded access frameworks (n=9) presented in an Orchard Therapeutics media release
    • 04 Nov 2022 Planned End Date changed from 9 Apr 2023 to 15 Mar 2025.
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