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 Single Center, Open-label, Non-randomized, Uncontrolled, Multiple-dose, Dose Escalation Study of the Safety, Pharmacokinetics and Efficacy of Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)

Trial Profile

A Single Center, Open-label, Non-randomized, Uncontrolled, Multiple-dose, Dose Escalation Study of the Safety, Pharmacokinetics and Efficacy of Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)

Status: Completed
Phase of Trial: Phase I

Latest Information Update: 15 Jun 2022

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 Cerebroside-sulfatase (Primary)
  • Indications Metachromatic leukodystrophy
  • Focus Adverse reactions; Pharmacokinetics; Therapeutic Use
  • Sponsors Shire; Shire Pharmaceuticals Ireland
  • Most Recent Events

    • 26 Apr 2022 Using data from 3 studies (NCT00418561; NCT00633139; NCT00681811) were used to evaluate the toxic effects of sulfatide and lysosulfatide on the central (CNS) and peripheral (PNS) nervous systems in children with metachromatic leukodystrophy (MLD) and to explore the impact of treatment with intravenous recombinant human arylsulfatase A (rhASA) on myelin turnover, presented at the 74th Annual Meeting of the American Academy of Neurology 2022
    • 20 Jun 2012 New trial record
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
Back to top