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: 02 Nov 2017

At a glance

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

    • 02 Nov 2017 Final results published in the New England Journal of Medicine
    • 01 Nov 2017 Results published in a Biogen Media Release.
    • 01 Nov 2017 According to a Biogen media release, results were published in The New England Journal of Medicine.
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