A Phase 3, Randomized, Double-Blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Infantile-onset Spinal Muscular Atrophy

Trial Profile

A Phase 3, Randomized, Double-Blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Infantile-onset Spinal Muscular Atrophy

Discontinued
Phase of Trial: Phase III

Latest Information Update: 05 Oct 2017

At a glance

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

    • 05 Oct 2017 Results were presented in a Biogen Media Release.
    • 05 Oct 2017 According to a Biogen media release, data were presented at the 22nd International Annual Congress of the World Muscle Society
    • 04 Jul 2017 According to a Biogen Canada media release, the Health Canada has approved SPINRAZA (nusinersen) for the treatment of patients with 5q spinal muscular atrophy (SMA) based on data from this and multiple open-label trials.
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