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?

An Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12 Month Treatment With Migalastat in Pediatric Subjects (Aged 12 to <18 Years) With Fabry Disease and Amenable GLA Variants

Next Previous
Trial Profile

An Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12 Month Treatment With Migalastat in Pediatric Subjects (Aged 12 to <18 Years) With Fabry Disease and Amenable GLA Variants

Status: Completed
Phase of Trial: Phase III

Latest Information Update: 04 Sep 2024

Price :
$35 *
Note:
  • 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 Migalastat (Primary)
  • Indications Fabry's disease
  • Focus Adverse reactions; Pharmacokinetics; Registrational
  • Acronyms ASPIRE
  • Sponsors Amicus Therapeutics

Most Recent Events

  • 04 Sep 2024 According to an Amicus Therapeutics media release, data from the study will be presented at the Society for the Study of Inborn Errors of Metabolism (SSIEM) 2024 Annual Symposium, being held September 3-6, 2024 in Porto, Portugal.
  • 02 Aug 2021 According to an Amicus Therapeutics media release, the company announced the European Commission has approved Galafold (migalastat) for use in adolescents aged 12 to <16 years weighing = 45 kg with a confirmed diagnosis of Fabry disease and who have an amenable mutation. The extension of the indication was supported by 1-month interim safety and pharmacokinetics data from this study
  • 12 Feb 2021 Status changed from active, no longer recruiting to completed.

You need to be a logged in or subscribed to view this content

Request demo (opens in a new window)
If your organization or you do not have a subscription, try one of the following: If your organization has a subscription, there are several access options, even while working remotely:
  • Working within your organization’s network
  • with username/password or try to via your institution
  • Persisted access using your organization’s identifier stored in your user browser for 90 days
Back to top