An Open-Label, Dose Escalation Study to Assess the Safety, Tolerability and Dose-Range Finding of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Spinal Muscular Atrophy

Trial Profile

An Open-Label, Dose Escalation Study to Assess the Safety, Tolerability and Dose-Range Finding of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Spinal Muscular Atrophy

Completed
Phase of Trial: Phase I/II

Latest Information Update: 22 Mar 2017

At a glance

  • Drugs Nusinersen (Primary)
  • Indications Spinal muscular atrophy
  • Focus Adverse reactions
  • Acronyms SMNRx-CS2
  • Sponsors Biogen; Ionis Pharmaceuticals; Isis Pharmaceuticals
  • Most Recent Events

    • 09 Mar 2015 Status changed from active, no longer recruiting to completed, according to ClinicalTrials.gov record.
    • 10 Oct 2014 Interim results published in Isis Pharmaceuticals media release, and presented at the 19th International World Muscle Society (WMS) Congress, according to the media release.
    • 04 Aug 2014 According to an Isis Pharmaceuticals media release, data will be reported in an upcoming medical meeting.
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