A Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Infantile-Onset Spinal Muscular Atrophy

Trial Profile

A Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Infantile-Onset Spinal Muscular Atrophy

Active, no longer recruiting
Phase of Trial: Phase II

Latest Information Update: 03 Aug 2017

At a glance

  • Drugs Nusinersen (Primary)
  • Indications Spinal muscular atrophy
  • Focus Therapeutic Use
  • Sponsors Biogen; Ionis Pharmaceuticals
  • Most Recent Events

    • 28 Jul 2017 Planned End Date changed from 1 Jun 2017 to 31 Aug 2017.
    • 28 Jul 2017 Planned primary completion date changed from 1 May 2017 to 31 Aug 2017.
    • 03 Jan 2017 Planned End Date changed from 1 Nov 2016 to 1 Jun 2017.
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