An Open-label, Escalating Dose Study to Assess the Safety, Tolerability and Dose-range Finding of a Single Intrathecal Dose of ISIS 396443 in Patients With Spinal Muscular Atrophy.

Trial Profile

An Open-label, Escalating Dose Study to Assess the Safety, Tolerability and Dose-range Finding of a Single Intrathecal Dose of ISIS 396443 in Patients With Spinal Muscular Atrophy.

Completed
Phase of Trial: Phase I

Latest Information Update: 14 Apr 2017

At a glance

  • Drugs Nusinersen (Primary)
  • Indications Spinal muscular atrophy
  • Focus Adverse reactions; Pharmacokinetics
  • Sponsors Ionis Pharmaceuticals
  • Most Recent Events

    • 20 Mar 2013 Status changed from active, no longer recruiting to completed, based on results reported in an Isis Pharmaceuticals media release.
    • 14 Mar 2013 Phase I data was presented at the 2013 American Academy of Neurology Annual Meeting (AAN), according to an Isis Pharmaceuticals media release.
    • 01 Nov 2012 This trial was ongoing at the end of October 2012, according to an Isis Pharmaceuticals media release.
Restricted Access

Oops, it looks like you don’t have a valid subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • with a username/password associated to an account with a valid subscription
  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Request a trial

If you are a subscriber to this content then contact us at AsktheExpert.AdisInsight@springer.com so we can help.

Back to top