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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: 22 May 2019

At a glance

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

    • 22 May 2019 Results assessing the impact of onasemnogene abeparvovec (AVXS-101) on survival, hospitalization, ventilatory support, and associated costs in SMA1 patients from CL-101 study contrasted with response to nusinersen from ENDEAR study, presented at the 24th Annual International Meeting of the International Society for Pharmacoeconomics and Outcomes Research.
    • 22 May 2019 Results assessing the impact of AVXS-101 on survival and motor milestone achievement in SMA1 patients (CL-101 study) contrasted with response to nusinersen (ENDEAR study) and untreated patients, presented at the 24th Annual International Meeting of the International Society for Pharmacoeconomics and Outcomes Research
    • 16 Mar 2019 Results comparing treatment effects of AVXS-101 relative to nusinersen for the treatment of patients with SMA type 1 in ENDEAR and AVXS-101-CL-101 trials, published in the Advances in Therapy
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