Either you have JavaScript disabled or your browser does not support Javascript . To work properly, this page requires JavaScript to be enabled.
How to enable JavaScript in your browser?

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

Next Previous
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

Price : $35 *
  • Adis is an information provider.
  • Final gross price and currency may vary according to local VAT and billing address.
  • Your purchase entitles you to full access to the information contained in our trial profile at the time of purchase.
  • A link to download a PDF version of the trial profile will be included in your email receipt.

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.
Subscriber content

You need to be a logged in subscriber to view this content.

If your organization has a subscription then there are several options available to help you access AdisInsight, even while working remotely.

  • IP authentication when working within your organization’s network.
  • with username/password or try via your institution
  • Persisted access using your organization’s identifier stored in your user browser for 90 days.
  • Contact us at AsktheExpert.AdisInsight@springer.com for assistance.

If your organization does not have a subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Buy a PDF version of the profile.
  • Request a free trial
Legal
Back to top